Omeros holds big potential - 500 Beiträge pro Seite

[Shanghai2006]

Brian Feroldi (Omeros Corporation): Omeros Corporation has already crossed the finish line of FDA approval with a pupil-dilating drug called Omidria, which helps eye surgeons perform cataract removal and lens replacement procedures. While the initial ramp-up was slow, Omidria's sales growth exploded by 212% last year to $41.4 million -- and that could be just the beginning. Since millions of eye surgeries are performed every year, it's possible that Omeros could have a blockbuster on its hands. If true, then Omeros could easily turn profitable from Omidra sales alone.
However, the real reason I think investors should pay attention to Omeros is a compound drug called OMS721. This product candidate is being researched as a treatment option for a number of disorders that includes atypical hemolytic-uremic syndrome (aHUS). There is currently only one treatment option for this ultra-rare condition: Alexion Pharmaceuticals' Soliris. Alexion rang up more than $2.8 billion in total Soliris sales last year in part because of its aHUS monopoly. That hints that OMS721 could potentially ring up nine figures in sales on its aHUS opportunity alone. That number could easily cross into blockbuster status if it proves to be helpful in treating other rare disorders, too.
Omeros offers investors profit potential with Omidra and the chance at lottery ticket-type winnings from OMS721. That's an interesting combination for a company with a sub-$700 million market cap, which is why I think this stock should appeal to any risk-loving biotech investor.

https://www.fool.com/investing/2017/06/05/3-stocks-with-exel…

[Shanghai2006]

Omeros shares surge 10% after settlement with Endo delays generic to 2032

Published: Oct 5, 2017 11:31 a.m. ET

Omeros Corporation OMER, -6.04% shares surged as much as 10% in morning trade Thursday, before paring some gains, after the company announced a settlement with Endo International PLC's Par Pharmaceutical Inc. ENDP, -0.92% that prevents Par from launching a generic of Omeros' Omidria until April 1, 2032. Under the agreement, Par, if it enters the market, will pay a 15% royalty on net sales of a generic Omidria until the last patents expire on October 23, 2033. Endo has also filed pending patent applications that would expire as late as November 30, 2035, according to the company's late-April annual report. Omidria is Omeros' only FDA-approved product and brought in net revenue of $41.4 million last year, according to the annual report. Par, which had filed a new drug application with the Food and Drug Administration for its generic Omidria, said that all Omidria patents are valid as part of the settlement, according to Omeros. Omidria, which was approved by the FDA in June 2014, is used during cataract surgery or intraocular lens replacement. Omeros shares have surged nearly 15% over the last three months, and Endo shares have dropped 25%.

http://www.marketwatch.com/story/omeros-shares-surge-10-afte…

[Shanghai2006]

Report of Successful OMS721 Treatment of Patient with Both Stem Cell Transplant-Associated Thrombotic Microangiopathy and Graft-versus-Host Disease Presented at EBMT Annual Meeting
-- Results May Expand Target for Omeros’ Planned Phase 3 Program in HCT-TMA --

SEATTLE--(BUSINESS WIRE)--Oct. 20, 2017-- Omeros Corporation (NASDAQ: OMER) today announced the presentation of a case report of a patient having co-existing hematopoietic stem cell transplant-associated thrombotic microangiopathy (HCT-TMA) and graft-versus-host disease (GvHD), which both resolved following OMS721 treatment. This case was presented this morning at the European Society for Blood and Marrow Transplantation (EBMT) Crash Course on Diagnosis and Treatment of Noninfectious Complications after HCT in Granada, Spain. The presentation, “Effective Treatment of GvHD-Associated Transplant-Associated Microangiopathy with a Novel Inhibitor of the Lectin Pathway of Complement,” was made by Chiara Caprioli, M.D. of the Hematology and Bone Marrow Transplant Unit, Azienda Ospedaliera Papa Giovanni XXIII, in Bergamo, Italy.

The patient was a participant in Omeros’ ongoing Phase 2 uncontrolled clinical trial of thrombotic microangiopathies, including HCT-TMA. To be eligible for enrollment, HCT-TMA patients are required to be adults with post-transplant TMA persisting at least two weeks following calcineurin inhibitor modification (conservative treatment). This population is considered to be at high risk for poor outcomes, including mortality.

Prior to enrollment in the OMS721 trial, the patient had undergone HCT for T-cell acute lymphoblastic leukemia. His post-transplant course was complicated by multiple episodes of steroid-refractory grade IV (life-threatening) GvHD, cytomegalovirus infection, and HCT-TMA. After two prior episodes of GvHD, the patient presented with bloody diarrhea. Intestinal biopsy demonstrated both HCT-TMA and GvHD. No infections were identified. Notably, the patient also had new onset neurological symptoms of paresthesias, tetraparesis, and a neurogenic bladder, which have been reported as neurological manifestations of GvHD and TMA. The patient was unable to walk due to the tetraparesis and required blood transfusions at least once daily. Hematological markers demonstrated HCT-TMA with thrombocytopenia, elevated lactate dehydrogenase (LDH), and schistocytes. Two weeks prior to initiation of treatment with OMS721, his immunosuppression (cyclosporine) had been decreased and, given his history of steroid-refractory GvHD, he was receiving only low-dose corticosteroids. He received no other GvHD treatment.

After two OMS721 doses, his bloody diarrhea resolved and his hematological markers improved. After four OMS721 doses, he was able to walk with help. He completed eight weeks of OMS721 treatment and has been doing well at home. All signs and symptoms of HCT-TMA and all clinical symptoms of GvHD have resolved. His neurological symptoms have continued to improve.

“This patient’s marked response to OMS721 treatment was very gratifying,” stated Anna Grassi, M.D., Director of the Bone Marrow Transplant Unit at the Azienda Ospedaliera Papa Giovanni XXIII. “The cause of his neurological symptoms is not clear, but may be a manifestation of GvHD or other endothelial injury. Prior to OMS721 treatment, this patient was deteriorating and at high risk for early death. The improvement of GvHD, HCT-TMA, and the neurological symptoms following OMS721 treatment is promising.”

“This case reinforces recent thinking that endothelial injury is a common cause of post-transplant complications in our patients,” stated Professor Alessandro Rambaldi, Director of Department of Hematology and Oncology, Azienda Ospedaliera Papa Giovanni XXIII. “This patient’s course suggests that inhibition of the lectin pathway may be beneficial in the treatment of HCT-complicating endothelial syndromes beyond TMA, including GvHD. I look forward to further study of this pathway and treatment.”

Thrombotic microangiopathy is a potentially life-threatening complication of HCT. Approximately 20,000 HCT procedures are performed in the U.S. annually, and TMA is reported to occur in approximately 10 to 25 percent of HCT patients. Although the kidney is the most commonly affected organ, HCT-TMA is a multi-system disorder and can also manifest clinically in the lungs, gastrointestinal tract and central nervous system. Reported mortality in patients with multi-organ involvement is greater than 90%. Even in patients who survive acute episodes, HCT-TMA increases the risk for chronic kidney disease and end-stage renal disease.

Graft-versus-host disease is a common complication of HCT. Both acute and chronic forms exist and result from donor immune cells recognizing the recipient patient as foreign tissue. This triggers an immune response against the recipient patient. Acute GvHD occurs in up to 50% or more of patients who receive allogeneic transplants. Acute GvHD most commonly targets the skin, gastrointestinal tract, and liver, but can also affect the kidney, eye, lung, and blood cells. Chronic GvHD occurs in approximately 40% of patients who receive allogeneic transplants and most commonly affects the skin, liver, eye, gastrointestinal tract and lungs. Both acute and chronic GvHD are related to significant morbidity and mortality.

The most frequently reported adverse events in HCT patients treated with OMS721 have been nausea and diarrhea, neither of which investigators have deemed “drug-related.” One possibly treatment-related serious adverse event has been reported because the investigator could not rule out a contribution from OMS721. This patient was neutropenic, developed sepsis and subsequently died. Neutropenic sepsis is a common complication of HCT. Other infection-related deaths have occurred in the trial at times when the patients were not receiving OMS721 treatment (i.e., in the screening or follow-up periods).

“Our OMS721 clinical trials continue to yield exciting data,” stated Gregory A. Demopulos M.D., chairman and chief executive officer of Omeros. “Potential treatment of both HCT-TMA and GvHD is consistent with our understanding of lectin pathway activation in the setting of endothelial injury. We continue to treat patients with OMS721 who have a high likelihood of mortality and the results to date have been impressive. We plan to initiate later this year a Phase 3 program in HCT-TMA and will discuss with regulators approaches to expand the program to assess GvHD as well.”
In addition to its clinical program in HCT-TMA, OMS721 is being evaluated in Phase 3 clinical programs for atypical hemolytic uremic syndrome and for immunoglobulin A nephropathy (IgAN). OMS721 has received breakthrough therapy designation from FDA for IgAN and Omeros is in discussions with FDA for breakthrough designation for the drug in HCT-TMA. Omeros also is applying for the European Medicines Agency’s Priority Medicines (PRIME) program for OMS721 in each of IgAN and HCT-TMA.

http://investor.omeros.com/phoenix.zhtml?c=219263&p=irol-new…

[Shanghai2006]

Omeros Corporation Announces FDA Approval of OMIDRIA® for Use in Pediatric Patients

-- FDA Also Grants Additional Market Exclusivity --

SEATTLE--(BUSINESS WIRE)--Dec. 12, 2017-- Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases and disorders of the central nervous system, today announced that the U.S. Food and Drug Administration (FDA) approved Omeros’ supplemental new drug application (sNDA) following review of efficacy and safety data from a pediatric clinical trial, expanding the indication for OMIDRIA (phenylephrine and ketorolac intraocular solution) 1% / 0.3% to include use in pediatric patients (ages birth through 17 years old). OMIDRIA, used during cataract surgery or intraocular lens (IOL) replacement, prevents intraoperative miosis (pupil constriction) and reduces postoperative pain. FDA approved the sNDA for OMIDRIA under priority review.

The successful clinical trial was conducted in 78 pediatric patients randomized to either OMIDRIA or phenylephrine administered intraoperatively. Together with the label expansion now including both pediatric and adult patients, FDA also granted OMIDRIA an additional six months of U.S. market exclusivity. Under section 505A of the Federal Food, Drug, and Cosmetic Act, this six-month extension of market exclusivity is attached to the term of the drug’s patents listed in FDA’s Orange Book.

“We are pleased that FDA has approved the use of OMIDRIA in pediatric patients and granted additional market exclusivity,” said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. “Now all patients undergoing cataract or other lens replacement surgery can receive the benefits of OMIDRIA. These procedures are more complex in pediatric patients than in adults, and it’s gratifying to know that our drug will be helping children.”

Omeros previously announced a favorable settlement of its patent infringement lawsuit against Par Pharmaceutical, Inc. and its subsidiary Par Sterile Products, LLC (Par) in which Par acknowledged and confirmed the validity of the Orange Book-listed patents for OMIDRIA. Unless subsequently authorized pursuant to terms in the settlement agreement, Par is prohibited from launching a generic version of OMIDRIA until April 1, 2032. These same patents are similarly being asserted against Sandoz Inc. and Lupin Ltd. in Hatch-Waxman litigation planned for trial in mid-2019 in the U.S. District Court for the District of Delaware, the same court that presided over the Par case. If that court upholds and finds infringement of any claim in the Orange Book-listed patents for OMIDRIA that expire in October 2033, Sandoz and Lupin, as a result of the newly granted market exclusivity, will be precluded from launching a generic version of OMIDRIA until April 2034.

http://investor.omeros.com/phoenix.zhtml?c=219263&p=irol-new…

[Shanghai2006]

Omeros Corporation Reaches Agreement with FDA on OMS721 Phase 3 Trial Protocol for IgA Nephropathy

-- Also Initiates OMS721 Phase 3 Program in HCT-TMA --

SEATTLE--(BUSINESS WIRE)--Jan. 3, 2018-- Omeros Corporation (Nasdaq: OMER) today announced that it has reached agreement with the US Food and Drug Administration (FDA) on Omeros’ protocol for its Phase 3 clinical trial evaluating OMS721 in patients with IgA nephropathy (IgAN). Patient enrollment is expected to begin early next month.

The single Phase 3 trial is a randomized, double-blind, placebo-controlled trial in patients at least 18 years of age with biopsy-confirmed IgAN and with 24-hour urine protein excretion greater than 1 g/day at baseline on optimized renin-angiotensin system (RAS) blockade. Patients will receive an initial 12 weekly intravenous doses of study drug; additional weekly dosing can be administered for partial responders and relapsers. The primary endpoint, which could suffice for full approval, is reduction in proteinuria at 24 weeks after the start of dosing. The trial will employ an adaptive design that will allow intra-trial adjustment in sample size. For purposes of safety and efficacy assessments, the initial sample size for the proteinuria endpoint is estimated at 140 patients in each of the treatment and placebo groups. This will include a subset of patients with high levels of proteinuria (i.e., equal to or greater than 2 g/day) at baseline, and full approval could also be obtained if a substantial improvement is seen at 24 weeks in this subset of patients alone. The trial design will allow assessment for either full or accelerated approval at 24 weeks based on proteinuria results either (1) across the general population of study patients or (2) in the high-proteinuria subset of patients. In the event that the primary endpoint at 24 weeks results in accelerated approval from FDA, change in estimated glomerular filtration rate (eGFR) will be assessed at approximately three years after the start of dosing. The initial sample size estimate for the eGFR endpoint is approximately 160 patients per group and also will be adjustable under the study’s adaptive design.

FDA granted both breakthrough therapy designation and orphan drug designation for OMS721 in IgAN. Omeros’ applications to the European Medicines Agency (EMA) for orphan drug status and for eligibility to the Priority Medicines (PRIME) program for OMS721 in IgAN are pending.

Omeros has also initiated its Phase 3 program for OMS721 in hematopoietic stem cell-associated thrombotic microangiopathy (HCT-TMA) and intends to amend the ongoing Phase 2 protocol following discussion with FDA and/or EMA to transition the protocol into a Phase 3/pivotal trial. In the third quarter of 2017, Omeros submitted a preliminary breakthrough therapy designation request to FDA for OMS721 in HCT-TMA. Based on the data in that submission, FDA requested that Omeros submit a full application for breakthrough therapy designation for OMS721 in HCT-TMA following collection of additional information on the patients in the Phase 2 clinical trial. Omeros intends to submit the full application for breakthrough therapy designation by the end of this month. Enrollment in the Phase 2 trial has continued pending initiation of/conversion to the pivotal trial. Omeros also recently applied for eligibility to EMA’s PRIME program for OMS721 in HCT-TMA. FDA has already granted orphan designation for OMS721 in the prevention (inhibition) of complement-mediated TMAs, which includes HCT-TMA.

The company’s Phase 3 program in aHUS is also ongoing in both the US and Europe. The single-arm, open label, Phase 3 trial is targeting approximately 40 patients for EMA approval and US accelerated approval with 80 patients required for full approval in the US. Dosing consists of an initial IV loading followed by daily subcutaneous dosing. The FDA has granted OMS721 fast track designation in aHUS as well as orphan designation for OMS721 in aHUS and other complement-mediated TMAs.

“We’re pleased to have received agreement from FDA for our OMS721 Phase 3 protocol in IgA nephropathy,” said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. “Final preparations to begin the trial can now be completed and enrollment is expected to open in early February. To our OMS721 Phase 3 programs in IgA nephropathy and aHUS, we have recently added a third Phase 3 program in stem cell transplant-associated TMA. We look forward to ongoing discussions with both FDA and EMA regarding the HCT-TMA Phase 3 trial as well as breakthrough and PRIME designations. In the meantime, enrollment in the Phase 2 HCT-TMA trial has progressed with patients expected to have a very high mortality rate doing well on OMS721.”

http://investor.omeros.com/phoenix.zhtml?c=219263&p=irol-new…

[Shanghai2006]

This Is Why Omeros Jumped 95.8% in 2017

Rising sales and upbeat clinical news have left investors feeling optimistic.

Brian Feroldi (TMFTypeoh)
Jan 10, 2018 at 4:22PM

What happened
Shares of Omeros (NASDAQ:OMER), a commercial-stage biotech, rose 96% in 2017, according to data from S&P Global Market Intelligence. Investors can thank soaring Omidria sales and a handful of positive clinical updates for the big jump.

So what
Here's a review of the key announcements from the year:
•In May, investors cheered after the company reported data from a phase 2 study showing that OMS721 -- which is Omeros' lead pipeline candidate -- reduced urine protein levels in patients with Immunoglobulin A (IgA) nephropathy.
•In June, Omeros' shares soared after the company announced that OMS721 was granted breakthrough therapy designation for the treatment of IgA nephropathy.
•In November, shares took off after Omeros reported stronger-than-expect Omidria sales growth. Omidria is a drug that is used during eye surgery to help lower the risk of complications and is the company's primary moneymaker.
•In December, Omidria won Food and Drug Administration approval for a label expansion into the pediatric population. The regulatory win expanded the drug's addressable market and also extended its period of market exclusivity out until 2034.

Given the flurry of upbeat news, it is understandable why shares enjoyed such a positive run.

Now what
Omeros appears to be in great shape today. Omidria sales are growing rapidly and reaching the $1 billion sales mark isn't out of the question. OMS721 is also progressing nicely in the clinic and could turn into a megablockbuster compound if everything works out. That represents a lot of potential for a company that is currently worth less than $1 billion.

https://www.fool.com/investing/2018/01/10/why-omeros-jumped-…

All in all, I think that risk-loving investors have plenty of reasons to put this small-cap biotech on their radar.

[Shanghai2006]

Omeros: Competition In IgAN, But OMS721 Has The Lead

Jan. 11, 2018 5:42 AM ET

Summary

•OMS721 is well-advanced in IgAN and we are bullish on OMER based on that.

•However, we shouldn’t neglect a study of competition.

•There’s quite a bit of competition, as we will see here.

•We may update our valuation based on this data.

•Only members wishing to do more DD should read this article in its entirety. Others can skip to the last paragraph.

• This idea was discussed in more depth with members of my private investing community, The Total Pharma Tracker.


Omeros Corporation’s (OMER) lead drug candidate is OMS721, which is going through trials in IgA Nephropathy. We have covered the stock extensively, and readers know we are bullish OMER. However, we would be remiss in our due diligence if we did not discuss some of the main competition for OMS721. In this article, we will focus on competition in IgAN, and try and do some quick comparison.

OMS721 Phase 2 results in IgAN

Data can be found in this press release. Broadly, this was a complex trial involving four different complement-associated kidney diseases: IgA nephropathy, membranous nephropathy, lupus nephritis, and complement component 3 (C3) glomerulopathy. There were only four (4) patients in the first part of the IgAN study, which involved patients on at least three months of ongoing corticosteroid treatment. There is a second part, where patients are non-steroid-treated - this could possibly mean the part 1 patients were more seriously ill. We do not have data from the part 2 yet.

Part 1 patient data was excellent. To quote from our previous article:

“Omeros recently announced follow-up data on the four IgA nephropathy patients in the open-label portion of the Phase 2 trial. All four patients demonstrated a substantial reduction in proteinuria during the clinical trial. “In the extended (up to one year) follow-up after completion, proteinuria reduction was maintained in three of the four patients. Specifically, those three patients maintained partial remission relative to baseline (76 percent to 86 percent decrease in albumin/creatinine ratios (uACRs)) during extended follow-up.” After a substantial drop in uACR during the trial, the fourth patient’s uACR returned to 88 percent of baseline at four months post-treatment. eGFR improved in three of the four patients during the extended follow-up, with increases ranging from 7 to 17 mL/min/1.73 m2 (up to 57 percent improvement) relative to baseline. The fourth patient demonstrated stable eGFR relative to baseline. OMS721 was well-tolerated.”

“I have never seen the clinical responses that I’ve observed in IgA nephropathy patients treated with OMS721,” said Geoffrey Block, M.D., Director of Clinical Research at Denver Nephrology and Principal Investigator of the trial. “All of these patients had significant renal impairment when they entered the trial and each patient dramatically improved. The improvements in these patients continued to increase after the end of treatment and persisted following completion of the trial.”

On another matter, I discussed how OMER received what amounts to an SPA or Special Protocol Assessment from the FDA for the Phase 3 trial in IgAN. This trial will begin enrollment in February, and we expect results no later than end-2017.

The main competition
As against the backdrop of those excellent results from what was, after all, a pretty small trial, we have a number of companies in various stages of developing therapies for IgAN.

Probably the most advanced among them is actually a private European company called Calliditas Therapeutics AB. They are developing a product for IgAN, which completed Phase 2, then got the same sort of deal from the FDA for the endpoint as OMER, and seem to have a couple of months' lead over OMS721 in the Phase 2 trial, at least. However, Phase 3 may have lost that lead. The Lancet article where Phase 2 data was published is here.

Most important to note right off is that budesonide is a very old medicine of the corticosteroid type - meaning, there’s not a lot new here. Corticosteroids have been used against all sorts of inflammatory diseases for generations - and they come with their usual problems. To avoid those, this was made as a targeted-release formulation; however, I am not impressed with the innovation.

As to the trial, this was a much larger trial of 150 or so patients; however, it continued on a parallel optimised renin-angiotensin system or RAS blockade throughout the trial. The drug was not very safe, with quite a bit of AEs and SAEs (adverse events and serious adverse events), much of which were treatment emergent.

As to efficacy, I quote: “24·4% (SEM 7·7%) decrease from baseline in mean UPCR (change in UPCR vs. placebo 0·74; 95% CI 0·59–0·94; p=0·0066). At 9 months, mean UPCR had decreased by 27·3% in 48 patients who received 16 mg/day (0·71; 0·53–0·94; p=0·0092) and 21·5% in the 51 patients who received 8 mg/day (0·76; 0·58–1·01; p=0·0290); 50 patients who received placebo had an increase in mean UPCR of 2·7%.” (urine protein creatinine ratio = (UPCR)).


Now, while UACR and UPCR have strong correlation, the Phase 3 trial for OMS721 is about proteinuria, as is this Phase 2 trial from Calliditas. So, we would have liked to see data for that from Omeros. “In post-trial follow-up, urine protein/creatinine ratios (uPCR) were measured by the investigator according to his practice standard. For purposes of post-hoc comparisons of proteinuria during and after the clinical trial, each post-trial uPCR value was converted to uACR (Zhao, Clin J Am Soc Nephrol 2016;11:947-55).”

Lacking such data at hand, it is difficult to do a proper cross trial comparison. However, very broadly, it appears that OMS721 does not have very strong competition in this refurbished corticosteroid drug. I see no press release on the company website, so I am assuming enrollment has not yet started. However, the ASN data is here, and the multiplying factor between uACR and uPCR is 0.64, which adds up to a much more significant and sustained proteinuria reduction than the other trials discussed in this article. For convenience of follow up, here’s the actual data from the latest follow up trial:

RESULTS
All patients achieved partial remission following OMS721 treatment. The mean age of the 3 females and 1 male was 42 years, 3 are Caucasian and one is Asian, the mean eGFR was 41 mL/min/1.73 m2, and the mean entry steroid dose was 55 mg. Follow-up ranged from 2-9 months after the last OMS721 dose. During the trial the mean uACR decreased 77% (p = 0.026). Three patients maintained partial remission during available follow-up (54%, 79%, and 88% uACR decreases at 12, 12, and 5 months, respectively). One patient had 90% of baseline uACR at 7 months. Three patients also demonstrated improved eGFR by 7, 13, and 7 ml/min/1.73 m2 during follow-up. The fourth patient’s eGFR was stable. All patients discontinued steroids. OMS721 was well tolerated. All adverse events were mild with headache and sinus congestion considered possibly related by the investigator.”

Other competition
I will more or less simply list the ones I could find. This is just a listing of pipeline drugs. I cannot comment on current stages of development in some cases, except to say that none appear in the same timeline as OMS721.

Anthera Pharma (ANTH) has a Phase 2 program of a drug candidate named blisbimod. This company announced Phase 2 top line data in August, and we need to do a more thorough follow-up comparison. Briefly, material is available here, and data is here. From a quick look, the drug was able to halt increase in proteinuria. This is against the “substantial reduction in proteinuria” claimed by Omeros.

Rigel’s fostamitinib is being studied in IgAN. Fostamitinib is an oral SYK inhibitor. From their website: “Pre-clinical data show that fostamatinib, Rigel's oral SYK inhibitor, may decrease SYK activation in the kidney, resulting in the reversal of the inflammation in the glomeruli and improvement in kidney function. Rigel announced results of the first cohort (100mg BID, n=26; placebo n=12) of a Phase 2 study of fostamatinib in IgAN in January 2017. Rigel expects that the second cohort, evaluating a higher dose of fostamatinib (150mg BID), will finish enrollment in 2017 with full results in 2018.”

Merck Serono has a Phase 2 product candidate named atacicept, an anti-Blys/anti-APRIL fusion protein targeting IgA nephropathy.

Baxalta, owned now by Shire Plc (SHPG), has a program in IgAN, which initiated a Phase 2 in 2015. This may have been discontinued because I can find no new data beyond 2015. This one, called SM101, is a “IgG Immune Complex Scavenger Receptor fragment protein which binds to circulating immune complexes Immune complex mediated diseases (e.g. SLE, IgA Nephropathy) and completed Phase IIa studies in SLE.”

BioMarin (BMRN) partnered with private player IGAN Biosciences to develop a treatment for IgAN. However, their website doesn’t list anything, so I am assuming this too is a dud.

Finally, Chemocentryx’s (CCXI) CCX168 once had a program in IgAN. However, it's not mentioned in their website right now.

So, broadly, that is the state of competition for Omeros in this one indication. Competition exists in the other disease areas as well. However, it seems that not only does oMS721 have a lead, but it also has some efficacy edge and a strong edge in safety. Now, the drug has been - or will be - declared an orphan drug in these indications.

Some of these other drugs are, too. However, and although I am not absolutely sure of the legal implications, but if OMS721 comes to market first and as an orphan drug, then it should have 7-year exclusivity in this target area as long as the competition does not prove superiority. That is my understanding, and I look forward to your comments in the discussion section below.

https://seekingalpha.com/article/4136817-omeros-competition-…

[Shanghai2006]

Omeros: OMS721 Updates Are Very Positive, But Where's The Update On Omidria?

Jan. 11, 2018 7:43 AM ET
ONeil Trader

Summary

•Omeros surged last week after providing an update on two OMS721 programs, but the stock gave back all of its gains.

•The OMS721 updates are very positive for the company but the lack of any updates on Omidria and its pricing status in 2018 is keeping the stock down.

•I think the IgA nephropathy trial could report results as soon as mid-2019, but that's later than some Omeros bulls expect.

•The HCT-TMA may become the first approved indication for OMS721 - the phase 2 protocol will transition to a phase 3 trial.

• This idea was discussed in more depth with members of my private investing community, Growth Stock Forum.

It’s hard being an Omeros (OMER) shareholder if you look at and give significance to daily share price movements. The stock surged last week after the company provided an update on two of its three phase 3 programs of OMS721 but gave back all the gains and closed with a 2% loss the same day. The main issue, as I see it and as I covered extensively over the last couple of months is Omidria’s pricing and reimbursement status in 2018. I see many investors blaming shorts for these kinds of selloffs, but the company itself should take some of that heat (and perhaps all of it in this particular case) due to very poor transparency and the lack of updates, which just gives Omeros bears ammunition. This could easily be resolved with a simple press release and the stock will do what it will do in the short run and resume its trend along with fundamental changes that happen along the way – Omidria’s quarterly net sales, and more importantly, the progress of OMS721 in several late-stage trials, as well as the progress of other pipeline candidates.

I remain as bullish on Omeros as I was previously (probably more so on OMS721 after this week’s update), but must say I am not happy with the company failing to communicate its pricing and access strategy for Omidria in 2018. With that in mind, the rest of the article will focus on last week’s update on the two OMS721 phase 3 programs.

IgA nephropathy protocol – all we wanted to see, and then some

Omeros and the FDA reached an agreement on the phase 3 protocol for OMS721 in IgA nephropathy. The single phase 3 trial is a randomized, double-blind, placebo-controlled trial in patients at least 18 years of age with biopsy-confirmed IgAN and with 24-hour urine protein excretion greater than 1 g/day at baseline on optimized renin-angiotensin system (NYSE:RAS) blockade. Patients will receive OMS721 once-a-week for 12 weeks intravenously and partial responders and relapsers can receive additional weekly doses. The primary endpoint for either accelerated or full approval is reduction in proteinuria at 24 weeks after the start of dosing. The trial will enroll approximately 140 patients in each of the treatment arm and placebo groups and will include a subset of patients with high levels of proteinuria (equal to or greater than 2 g/day) at baseline, and full approval can be obtained if a substantial improvement is seen at 24 weeks for this subset of patients alone. If accelerated approval is granted, change in eGFR will be assessed at approximately three years after the start of dosing.

This protocol is roughly in line with what CEO Demopulos shared on the Q3 earnings call. The new details are:
•The number of patients. I thought the number of patients could be somewhat lower than 140 per arm. This will lengthen the enrollment process.
•The subset of patients was not clearly defined. Dr. Demopulos only mentioned this as a possibility, and this remains a very positive development as it provides two shots on goal for OMS721 in this trial.
•The design is adaptive, meaning changes could be made during the trial. This allows for a potential reduction in patient numbers and faster enrollment, though I wouldn’t count on that.

Enrollment will start in early February instead of late 2017, but everything looks pretty good to me. Now, let’s talk timelines. If enrollment starts next month, I think we won’t see the results of this trial before mid-2019. I think it will take at least 9 and up to 12 or 15 months to enroll close to 300 patients. To that, we should add roughly 6 months of treatment for the last patient enrolled and 1-3 months to collect and analyze data before announcing the results. Adding this up, we get 16 to 24 months from the start of enrollment in February, which translates to a trial readout in June/July 2019 or Q1 2020. If the company can do it sooner, that would be great, but I am fairly certain that we won’t see the results before mid-2019 and that’s the best-case scenario for me. I see fellow Seeking Alpha Avisol Capital Partners expects to see results by end of 2018 or early 2019. I certainly hope they are right but I am sticking with my timeline estimate.

If you want an example, there is a similar trial, targeting a similar disease. ChemoCentryx (CCXI) is conducting a phase 3 trial for avacopan in ANCA-associated vasculitis, or AAV. ChemoCentryx needs to enroll 300 patients – very similar to OMS721’s target of 280 patients and below is the trial’s enrollment curve. As you can see, it takes time to open up sites and to word to spread from physicians to patients, and eventually, to get patients screened and enrolled in the trial. ChemoCentryx expects to complete enrollment by mid-2018, which translates to roughly 18 months from the start of enrollment in late 2016.

So, it looks I am being quite generous with my 9-15 month enrollment completion estimate for OMS721’s IgAN trial, but it’s also worth noting that the number of IgAN patients is between 2x and 4x the number of AAV patients in the U.S. However, that by itself does not translate to a significantly increased pace of enrollment, while the subset of IgAN patients with very high proteinuria limits the addressable patient pool, probably bringing it closer to avacopan’s AAV patient pool. But overall, I think Omeros could do it's trial somewhat faster as I noted with the 9-15 month timeline instead of an 18-month enrollment timeline for avacopan. I would be more than happy to be proven wrong (on the positive side, of course), meaning the company can enroll patients faster than I anticipated.

Another example in a similar disease setting with a more prevalent population is Aurinia's (AUPH) phase 3 trial of voclosporin in lupus nephritis (at least 50% higher prevalence in the U.S. than IgAN). Aurinia dosed its first patient in May 2017 and expects to complete enrollment of approximately 320 patients in 2H 2018 - that's at least 15 months from start to finish just for enrollment.

And finally, we have Rigel (RIGL) conducting a phase 2 trial in IgAN. This trial had two cohorts and enrollment for the first cohort opened in October 2014, and the company reported results in early 2017 (approximately 40 patients). The second, higher dose cohort started enrolling in mid-2016 end completed enrollment in August 2017 with results expected in the following months (see study changes/updates here).

I am writing about this because we as investors need to temper our expectations about trial completion timelines – this one and every other. Having too aggressive trial completion timelines often leads to disappointment. Do we have evidence that Omeros can execute a trial better than ChemoCentryx, Aurinia or Rigel? No. In fact, remember the placebo-controlled phase 2 trial of OMS721 in IgAN with just 10 patients (5 OMS721-treated and 5 placebo patients) - the company changed the protocol to include this small trial in February 2017 and expects to report results from this small trial by mid-2018. That's 15 months for 10 patients from protocol change to data release, unless they report results sooner than mid-2018, but we are already 11 months from the protocol change. The aHUS phase 3 trial is also looking slow with no updates 9-10 months after the start of enrollment and it took Omeros 18 months from positive phase 2 data in August 2015 to finally start enrolling patients in the phase 3 trial.

So, we need to keep our expectations in check. Omeros will get to the finish line in IgAN, but probably at a slower pace than most expect. And that’s fine, drug development is a marathon, not a sprint.

HCT-TMA phase 2 protocol to transition to a phase 3/pivotal trial

I think the update on the HCT-TMA program was more bullish than the IgAN program update. Omeros intends to amend the ongoing phase 2 protocol following discussion with FDA and/or EMA to transition the protocol into a phase 3/pivotal trial. Omeros intends to submit the full application for breakthrough therapy designation by the end of January and the company also recently applied for eligibility to EMA’s PRIME program in HCT-TMA. Enrollment in the trial has continued pending initiation of/conversion to the pivotal trial.

This is what Dr. Demopulos meant when he said the HCT-TMA program may leapfrog both the aHUS and IgAN phase 3 trials in terms of completion. We don’t know the full details yet, mainly the estimated number of patients needed to obtain full approval which is the largest variable, but it is possible that the number will not be higher than 30-40 patients, similar to the aHUS trial, which requires 40 patients for accelerated approval and 80 patients for full approval. If the number of patients required for approval is closer to 30, I think this trial should enroll enough patients fairly quickly and that we may see the results in late 2018, and definitely in 1H 2019. But let’s wait until we see the additional details before making any estimates.

The super-bullish scenario was not achieved here – the submission for accelerated approval based on existing phase 2 data in HCT-TMA. But this is the second best thing to have occurred and I am very pleased with this update, more so than with the IgAN program update.

Conclusion

Omeros’ failure to communicate Omidria’s 2018 pricing and access strategy has most likely driven the stock down following the good news on OMS721 in IgAN and HCT-TMA. Once we get Omidria’s pricing over with, we can focus on other, more important things and the market will adjust to the new reality. Uncertainty can sometimes be worse than actual bad news and even if the stock drops on the anticipated update on Omidria, I think we will see it trading higher in due time. The company made significant progress with OMS721 in IgAN and HCT-TMA, and this progress will eventually get investor recognition.


The main risks to the thesis include Omidria not generating enough revenues to cover expenses and/or the company being unable to raise enough capital to complete the clinical trials of OMS721 (Omeros had $87 million in cash and equivalents at the end of Q3 2017 and access to $45 million in additional funding under the CRG loan) and OMS721 and other pipeline candidates failing in the clinic.

Below are the potential catalysts/developments throughout 2018:
•Omidria’s pricing/access status in the following weeks.
•Start of enrollment in the OMS721 phase 3 trial in February.
•Protocol change of OMS721 phase 2 trial in HCT-TMA to a phase 3 trial in the following months and continued enrollment of patients in this trial. Update on the phase 3 protocol itself – the number of patients needed for approval and the endpoints of the trial, is also due in the following months. It’s also possible for the company to report additional phase 2 data in HCT-TMA, though that appears less likely given the expected protocol change.
•Phase 2 results of OMS721 in IgA nephropathy by mid-2018 – this is a placebo controlled trial with 5 patients in each cohort.
•Start of a phase 2 trial of OMS721 in IgAN patients with eGFR below 30 mL/min/1.73 m2 in 1H 2018.
•Potential Breakthrough Therapy Designation for OMS721 in HCT-TMA in the U.S. and PRIME designation in the EU within the next few months.
•Ex-U.S. partnership for Omidria needs to happen soon if the company is to retain the EU marketing authorization (it expires in July 2018). If not, we will need to write off ex-U.S. contribution.
•Update on OMS721 in lupus nephritis and the path forward for this indication (if there is a path forward), as well as potential updates on other indications OMS721 may target, like C3 glomerulopathy.
•Start of a phase 1 trial of OMS527 in 1H 2018 – this candidate is being developed for addiction disorders.
•Other pipeline updates, including OMS824, OMS405, OMS906 (though OMS906 is expected to enter the clinic in 2019 or 2020), and the GPCR platform.
•There is also a scenario where Omeros could partner OMS721 ex-U.S. This doesn’t seem likely to me at this point but is something the company might consider, especially if Omidria’s uptake is not satisfactory within the bundle and if Omeros decides to raise non-dilutive capital.
•And finally, there is a possibility for Omeros to obtain separate reimbursement for Omidria in the following months/quarters.

https://seekingalpha.com/article/4136848-omeros-oms721-updat…

[paul81]

-50% seit Anfang 2018... Zeit zum einsteigen oder in meinem Fall zum nachkaufen

[Shanghai2006]

Press Release
26. April 2018

FDA Grants Breakthrough Therapy Designation to Omeros’ MASP-2 Inhibitor OMS721 for the Treatment of High-Risk Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy
– Discussions Ongoing with FDA and European Regulators for Expedited Approval –

SEATTLE--(BUSINESS WIRE)--Apr. 26, 2018-- Omeros Corporation (NASDAQ: OMER) today announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to OMS721 for the treatment of patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patients who have persistent TMA despite modification of immunosuppressive therapy. This is the second breakthrough therapy drug designation for OMS721, which last year received the designation from FDA for the treatment of Immunoglobulin A (IgA) nephropathy. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

“TMA is an increasingly common complication following stem cell transplantation and is devastating when conservative treatment of immunosuppressive modification fails,” stated Rafael Duarte, M.D., Ph.D., F.R.C.P.(Lon), Associate Professor, Head of Hematopoietic Transplantation and Hemato-oncology Section, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain, and Secretary of the European Society for Blood and Marrow Transplantation. “Patients with HSCT-TMA who cannot undergo or do not respond to modification of immunosuppressive therapy are at very high risk of death from this complication. Currently, there is no approved treatment for HSCT-TMA, which remains one of the most pressing therapeutic needs in the field of HSCT. The impressive effect on survival and other results seen in OMS721-treated patients are quickly apparent following initiation of OMS721 therapy and can’t be explained by other factors. The HSCT community looks forward to the drug’s broad availability for our patients.”

Breakthrough therapy designation was granted based on data from Omeros’ Phase 2 clinical trial evaluating OMS721 in patients with high-risk HSCT-TMA. To be eligible for enrollment in the clinical trial, HSCT-TMA patients are required to be adults with post-transplant TMA persisting for at least two weeks following immunosuppressive regimen modification (conservative treatment) or more than 30 days post-transplant. This population was chosen to represent a population at risk for poor outcomes, including mortality. These patients often have serious, life threatening co-existing conditions, and mortality rates have been reported to be as high as 100 percent. As reported previously, the estimated median survival for OMS721-treated patients was an order of magnitude greater than that for a matched historical control (p<0.0001). Further analysis of the data examined 100-day mortality, an important endpoint previously used as an approval endpoint in HSCT. That analysis also showed that OMS721-treated patients had improved survival relative to the historical control (53% vs 10%; p = 0.0002). As previously reported, biomarkers of disease (i.e., mean platelet count and mean levels of lactate dehydrogenase and haptoglobin) demonstrated statistically significant improvement. Study patients also showed substantial improvement in red blood cell and platelet transfusion requirements. Other serious co-existing conditions in the patients treated with OMS721 included graft versus host disease (GvHD), cytomegalovirus and human herpes virus 6 infections, prior sepsis, diffuse alveolar hemorrhage, and residual underlying malignancies.

“I have treated several stem-cell TMA patients with OMS721 and seen marked and unexpected improvements that can’t be otherwise explained,” stated Professor Alessandro Rambaldi, from the University of Milan and Director of Department of Hematology and Oncology, Azienda Ospedaliera Papa Giovanni XXIII. “Most notable was a deteriorating patient with co-existing GVHD, TMA and neurological disability that confined him to bedridden hospitalization. Following treatment with OMS721, his TMA resolved quickly and his neurological status progressively improved, allowing him to leave the hospital and return to part-time work. This effect on TMA was observed in the absence of other specific treatments. The improvement seen in my patients has convinced me that lectin pathway inhibition by OMS721 is a scientifically sound and highly promising treatment strategy for a range of disorders associated with endothelial cell injury that commonly occur following stem cell transplantation.”

FDA’s breakthrough therapy designation enables expedited development and review of a drug candidate for the treatment of a serious or life-threatening disease. Preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies is required. Benefits of breakthrough therapy designation include the eligibility for priority review of the application and rolling submission of portions of the application. FDA works closely with the company to provide guidance to determine the most efficient route to approval.

“High-risk TMA following hematopoietic stem cell transplant carries an extremely high mortality rate, and no treatments are approved for this devastating disorder,” stated Gregory A. Demopulos, chairman and chief executive officer of Omeros. “We appreciate FDA’s recognition of the potential for OMS721 to improve outcomes – most importantly survival – for these patients, and we look forward to working closely with the Agency to accelerate the development and approval of OMS721.”

Persistent thrombotic microangiopathy is a life-threatening complication of HSCT. Approximately 20,000 HSCT procedures are performed in the U.S. annually, and TMA is reported to occur in approximately 10 to 25 percent of HSCT patients. Reported mortality in high-risk patients is greater than 90%.

OMS721 is also being evaluated in ongoing Phase 3 clinical trials in IgA nephropathy and atypical hemolytic uremic syndrome. Across all clinical trials with OMS721, the drug has been well tolerated and no safety concerns have been identified.

http://investor.omeros.com/phoenix.zhtml?c=219263&p=irol-new…

[Shanghai2006]

Press Release

Additional Clinical Studies Supporting the Benefits of OMIDRIA® Presented at the American Society of Cataract and Refractive Surgery and American Society of Ophthalmic Administrators Annual Meeting

--OMIDRIA demonstrates clear benefits across traditional and femtosecond laser-assisted cataract surgery and in IFIS cases--

SEATTLE--(BUSINESS WIRE)--Apr. 19, 2018-- Omeros Corporation (NASDAQ: OMER) announced that the results of four real-world clinical studies evaluating the benefits of OMIDRIA® (phenylephrine and ketorolac intraocular solution) 1% / 0.3% were presented at the American Society of Cataract and Refractive Surgery and American Society of Ophthalmic Administrators Annual Meeting held in Washington, D.C., April 13-17, 2018. The studies examined the use of OMIDRIA in both routine and complex cataract surgery cases performed in high-volume surgery centers, with and without femtosecond laser.

The first presentation, a retrospective analysis, assessed whether the use of OMIDRIA in femtosecond laser-assisted cataract surgery (FLACS) – a procedure that is known to cause miosis (pupil constriction) due to the increased inflammation generated by the laser – leads to a reduction in the need for pupil expansion devices and in surgical time. Investigators compared 100 consecutive femtosecond-assisted cataract procedures in which epinephrine was used in the irrigation solution to 100 consecutive femtosecond-assisted procedures performed with OMIDRIA. In the epinephrine group, 12 eyes (12 percent) required a Malyugin Ring® to maintain or improve pupil size versus 2 eyes (2 percent) in the OMIDRIA group (p=0.009). Surgical times in the OMIDRIA group were reduced by an average of 78 seconds compared to those in the epinephrine group (p = 0.007). The authors conclude that use of OMIDRIA achieves faster surgery and a more manageable pupil.1
The second clinical study assessed the effects of OMIDRIA on pupil dilation when used early in patients undergoing both FLACS and traditional cataract surgery. A total of 77 patients underwent either traditional phacoemulsification (n=57) or FLACS (n=20), all of whom received OMIDRIA in the irrigation solution and administered through the side-port incision at the beginning of the case. Pupil diameter was measured before and after OMIDRIA administration as well as prior to intraocular lens (IOL) insertion. In the traditional phacoemulsification cases, the mean pupil diameter was 6.65 mm at baseline and 7.42 mm intraoperatively before IOL insertion. Similarly, in the FLACS group, the mean pupil diameter was 6.69 mm at baseline and 7.65 mm prior to IOL insertion. The study demonstrates that OMIDRIA is effective in maintaining and even increasing pupil size in traditional as well as in femtosecond laser-assisted cataract procedures, which, without OMIDRIA, frequently induce miosis.2

A third study evaluated the effect of OMIDRIA on pupil dilation, iris billowing and iris prolapse in patients at high risk for intraoperative floppy iris syndrome (IFIS). The randomized, double-masked, investigator-initiated study enrolled 50 male subjects (50 eyes) who had been exposed to tamsulosin (Flomax®), a prostate drug that causes IFIS, prior to cataract surgery. The 50 patients were randomized 1:1 to receive either OMIDRIA or control. All subjects underwent routine cataract surgery recorded endoscopically from the perspective of the ophthalmic microscope. Investigators assessed pupil diameter and IFIS symptoms in all 50 subjects (50 eyes). Mean pupil diameter during surgery for the control group was 5.92mm and was 7.08mm for the OMIDRIA-treated group (p < 0.001). Symptoms of IFIS were observed in all 25 (100 percent) patients in the control group and in only 3 (12 percent) of patients who received OMIDRIA. Iris prolapse occurred in 14 (56 percent) subjects in the control group versus 3 (12 percent) in the OMIDRIA group. Iris billowing, graded according to a new grading scale for intraoperative iris abnormalities, averaged 2.36 in the control group and 1.68 in the treatment group, with Stage 3 (severe) billowing seen in 10 (40 percent) vehicle subjects and 1 (4 percent) treatment subject. All of the comparisons related to IFIS symptoms were statistically significant with p < 0.001. The authors conclude that OMIDRIA offers significant benefits in IFIS cases and could reduce the complexity and unpredictability of such cases.3

A fourth study assessed clinical outcomes and surgical practice patterns for cataract patients treated with either OMIDRIA or epinephrine. This retrospective chart review included patients who received treatment at one of two clinics by a single surgeon practicing at both locations. A total of 635 eyes in 375 patients underwent cataract surgery with either OMIDRIA (n=275) or epinephrine (n=360) in the irrigation solution. Mean surgical time was significantly shorter with OMIDRIA versus epinephrine (16.5 min vs 17.8 min; p = 0.006), and only 6 (2.2 percent) pupil expansion devices were required in the OMIDRIA group vs 24 (6.7 percent) in the epinephrine group (p = 0.008). Investigators conclude that OMIDRIA reduced surgical time and decreased the need for pupil expansion devices compared to the use of intracameral epinephrine.4

Detailed descriptions of all four clinical studies have been accepted for publication by, or are planned for submission to, peer-reviewed journals.

http://investor.omeros.com/phoenix.zhtml?c=219263&p=irol-new…

[Shanghai2006]

Here's Why Omeros Corporation Stock Is Surging Today
Pipeline progress outlined during the company's first-quarter report lifted spirits.

Cory Renauer
May 11, 2018 at 1:29PM

What happened
Shares of Omeros Corporation (NASDAQ:OMER), a commercial-stage biopharmaceutical company, are on the move following the company's first-quarter earnings report. Although revenue fell off a cliff, investors liked what they heard about this biotech's lead candidate enough to drive the stock 23.4% higher as of 12:48 p.m. EDT on Friday.

So what
Omeros Corporation's first-quarter haul was decimated by the temporary loss of Omidria's favorable reimbursement status with the Centers for Medicare and Medicaid Services. Sales fell to just $1.6 million from $12.3 million during the same period last year. Despite the revenue dip, news concerning the company's lead candidate encouraged investors to push up the stock.

Earlier this year, it reported impressive results from a study with its lead candidate OMS721 as a treatment for a rare but dangerous condition that affects patients who undergo stem cell transplants. Transplant-associated thrombotic microangiopathy is so lethal that historical data projects a dismal 10% survival rate at 100 days for high-risk patients similar to those Omeros enrolled into a single-arm study with its candidate.

The company doesn't have any comparison data for OMS721 yet, but a 53% survival rate at 100 days looked impressive. Investors are feeling enthusiastic today because management dropped hints that the Food and Drug Administration might be willing to grant the experimental therapy an accelerated approval based on single-arm data. Omeros would need to run a post-approval confirmatory study, but speeding the therapy to a commercial launch could send the stock screaming higher.

Now what
Omeros has started enrolling immunoglobulin A nephropathy patients in a phase 3 trial and another late-stage study with atypical hemolytic uremic syndrome patients is forging ahead as well. Before getting too excited, investors need to remember a couple things about this biotech stock. First, communications with the FDA aren't subject to full disclosure. The FDA could have discouraged the company from submitting an application without running expensive comparison studies and Omeros really isn't required to tell us.

I'm more concerned about the fact that we only have OMS721 data from a small number of single-arm studies. Using historical data to argue for efficacy has misled plenty of research efforts in the past. That said, the magnitude of the difference is hard to ignore. A majority of 19 patients treated with the experimental drug survived 347 days versus expectations of just 21 days based on past observations of patients with the lethal condition.

https://www.fool.com/investing/2018/05/11/heres-why-omeros-c…

[Shanghai2006]

Omeros up 23% on preparations for BLA for OMS721

May 11, 2018 1:36 PM ET|About: Omeros Corporation (OMER)|
By: Douglas W. House

Ignoring tepid Q1 results, investors are moving into Omeros (OMER +23.4%) after the company announced initial preparations for a U.S. marketing application for OMS721 for HSCT-TMA.
The FDA has requested a further characterization of patients treated with OMS721, additional information on the historical control population and an analysis plan to assess biomarker data. Consistent with accelerated approval, the company would concurrently conduct a confirmatory study prior to full approval. It expects to commence a rolling BLA submission later this year.

Previously: Omeros misses by $0.05, misses on revenue (May 10)

Previously: FDA designates Omeros' OMS721 for accelerated review for stem cell transplant-associated clotting disorder; shares up 9% (April 26)

https://seekingalpha.com/news/3355953-omeros-23-percent-prep…

[Shanghai2006]

Omeros Announces Settlement of Infringement Suit Against ANDA Filer Lupin

Business WireMay 24, 2018

SEATTLE--(BUSINESS WIRE)--
Omeros Corporation (OMER) today announced that it has entered into a settlement agreement with Lupin Ltd. and its subsidiary Lupin Pharmaceuticals, Inc. (Lupin), resolving Omeros’ patent litigation against Lupin. The litigation concerned Lupin’s filing of an Abbreviated New Drug Application (ANDA) seeking approval from the U.S. Food and Drug Administration (FDA) to market a generic version of Omeros’ commercial drug OMIDRIA® (phenylephrine and ketorolac intraocular solution) 1% / 0.3%.

Last year, Omeros announced that it had settled litigation directed to an ANDA filing by Par Sterile Products, LLC and Par Pharmaceutical, Inc. (Par). As in the settlement with Par, this agreement with Lupin includes Lupin’s acknowledgment and confirmation of the validity of all asserted patents for OMIDRIA as well as overall terms and market entry date similar to those set forth in the Par agreement. The expiration date of the last-to-expire of Omeros’ asserted patents for OMIDRIA is October 23, 2033.

The litigation against Lupin began in 2017 after Omeros received a Paragraph IV certification from Lupin in connection with Lupin’s filing of an ANDA seeking the FDA’s approval to market a generic version of OMIDRIA. As part of the agreement, Lupin acknowledges and confirms the validity of each of the patents listed in the Orange Book for OMIDRIA, namely U.S. Patent No. 8,173,707, U.S. Patent No. 8,586,633, U.S. Patent No. 9,066,856, U.S. Patent No. 9,278,101, U.S. Patent No. 9,399,040, U.S. Patent No. 9,486,406, and U.S. Patent No. 9,855,246.

“We are pleased with the Lupin settlement agreement,” stated Gregory A. Demopulos M.D., chairman and chief executive officer of Omeros. “With both the Par and Lupin litigation now settled, we remain focused on the near-term objectives for OMIDRIA – preparing for resumption of CMS separate payment on October 1, building utilization within the VA system, growing our customer base and expanding the drug’s Medicare Advantage and commercial reimbursement – all of which we expect will increase access to OMIDRIA for ophthalmic surgeons and their cataract surgery patients, improving outcomes and decreasing safety risks.”

https://finance.yahoo.com/news/omeros-announces-settlement-i…

[Shanghai2006]

Recent Developments in the Stem Cell Market

Published: May 22, 2018 9:00 a.m. ET
FinancialBuzz.com News Commentary

NEW YORK, May 22, 2018 /PRNewswire via COMTEX/ -- NEW YORK, May 22, 2018 /PRNewswire/ --
According to Grand View Research, Inc., the global stem cell market is projected to reach a value of USD 15.63 billion by 2025, while growing at a CAGR of 9.2%. The increasing number of research studies that aim at broadening the scope of stem cells associated products is on the strongest drivers of this market. The report also indicates that scientists are engaged in discovering novel methods to create human stem cells. This is to address the increasing demand for stem cell production for potential investigation in disease management. Experts in the field recognize this new field as the possible next major advancement in healthcare, one that can transform the industry. Some of the possible treatments of stem cell products could be of Parkinson's disease, type 1 diabetes, spinal cord injury, Alzheimer's disease, and others. International Stem Cell Corp. ISCO, +2.99% Intellia Therapeutics, Inc. NTLA, -6.09% Omeros Corporation OMER, +2.01% BrainStorm Cell Therapeutics Inc. BCLI, +3.80% Gilead Sciences, Inc. GILD, +1.34%

Recent results of an international trial of a stem cell transplant treatment shows that it could be a "game changer" for many patients with multiple sclerosis. According to Fortune, "Interim results from an international trial of a stem cell transplant treatment showed a much higher success rate than the control group, which received a drug treatment. MS is a disease that affects the brain, spinal cord, and immune system, leading to a number of disabilities. The stem cell treatment uses chemotherapy to wipe out a patient's immune system and then "re-boots" it using stem cells from the patient's blood and bone marrow that are unaffected by the disease."

International Stem Cell Corp. (otcqb:ISCO) a California-based clinical stage biotechnology company developing novel stem cell-based therapies and biomedical products, today provided a business update announcing operating results for the three months ended March 31, 2018.

"This was a successful quarter for us both operationally and financially. We were able to decrease net loss by more than 70% compared to the prior year quarter, as well as successfully move forward with our research and development activities. As a result, we have significantly decreased our cash burn while moving forward with our clinical trial. These outcomes give us confidence in the Company's future," said Andrey Semechkin, PhD., CEO and Co-Chairman of ISCO.

Q1 2018 Financial Highlights

Consolidated revenue for the first quarter of 2018 was $2.6 million, an increase of 30% compared to the consolidated revenue of $2.0 million for the first quarter of 2017.

Combined operating income for the quarter ended March 31, 2018 from our two wholly owned revenue generating subsidiaries was $631,000, an increase of 10% compared to $572,000 in the first quarter of 2017.

Average net cash used in operating activities, excluding capital expenditures and patent costs, was approximately $16,000 per month during the three months ended March 31, 2018, a decrease of 92%, compared to $190,000 per month for the same period in 2017.

Net loss for ISCO was $830,000 for the first quarter of 2018 compared to a net loss of $3.1 million for the first quarter in 2017, a decrease of 73%.

Recent Clinical Trial Highlights
The 2nd cohort of patients in the clinical trial for Parkinson's disease was successfully transplanted with 50,000,000 ISC-hpNSC® cells.

Data Safety Monitoring Board (DSMB), a group of experts monitoring the clinical trial of ISC-hpNSC®, has reviewed the safety data from the 2nd cohort in the clinical trial. The DSMB has authorized the clinical trial to move forward with accelerated enrollment and dose escalation to the 3rd cohort. Recruitment for the 3rd cohort will begin immediately, with patients receiving a higher dose of 70,000,000 ISC-hpNSC®.

United States Patent and Trademark Office (USPTO) has granted the Company a key patent on the method used to manufacture ISC-hpNSC, which are administered in ISCO's ongoing Parkinson's disease clinical trial, and which can potentially be utilized in therapies to treat traumatic brain injury and stroke.

Presented Interim Clinical Results at the American Academy of Neurology in Los Angeles, CA.

About International Stem Cell Corporation - International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (http://www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (http://www.lifelineskincare.com). More information is available at http://www.internationalstemcell.com."

Intellia Therapeutics, Inc. NTLA, -6.09% is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through optimized cell therapies that can treat cancer and immunological diseases by replacing patients' diseased cells. Recently, the company new scientific advisors in immuno-oncology and autoimmunity. The advisors hail from prestigious international institutions and collectively have both scientific and clinical expertise in cell therapies in these areas.

Omeros Corporation OMER, +2.01% is a Seattle-based commercial-stage biopharmaceutical company committed to discovering, developing, and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases and disorders of the central nervous system. On April 26, 2018, the company announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to OMS721 for the treatment of patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patients who have persistent TMA despite modification of immunosuppressive therapy. This is the second breakthrough therapy drug designation for OMS721, which last year received the designation from FDA for the treatment of Immunoglobulin A (IgA) nephropathy. OMS721 is Omeros' lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

BrainStorm Cell Therapeutics Inc. BCLI, +3.80% is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. On March 28, 2018, the company announced that it has amended the protocol of its Phase 3 clinical trial of NurOwn® for the treatment of amyotrophic lateral sclerosis (ALS). Per the protocol amendment, Canada-based ALS patients may now enroll in the ongoing Phase 3 NurOwn clinical trial. NurOwn utilizes a patient's own cells which have been engineered outside the body, to produce and secrete factors known to promote neuronal survival. NurOwn has the potential to be the first ALS treatment to improve patient functioning as a regenerative medicine.

Gilead Sciences, Inc. GILD, +1.34% is a biopharmaceutical company that discovers, develops and commercializes innovative therapeutics in areas of unmet medical need. Recently, Kite, a Gilead Company, announced it has leased a new facility in the Netherlands to engineer cell therapies in Europe. The 117,000 square-foot site in Hoofddorp (SEGRO Park Amsterdam Airport) will enable Kite to efficiently manufacture and deliver its cell therapies to people living with cancer in Europe and will provide more than 300 new jobs when fully operational in 2020. The facility will engineer and produce innovative cell therapies, including axicabtagene ciloleucel, a Chimeric Antigen Receptor T cell (CAR T) therapy that is currently under review by the European Medicines Agency and which is approved in the United Statesas Yescarta®. In addition to the Netherlands facility, Kite has recently purchased a new building in Santa Monica from Astellas Pharma Inc. that will be used for cell therapy research, development and the expansion of clinical manufacturing capabilities, and has leased a 26,000 square-foot facility in Gaithersburg, Maryland. The Maryland site will support the work of a new Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) to develop adoptive cell therapies targeting patient-specific tumor neoantigens.

https://www.marketwatch.com/story/recent-developments-in-the…

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