Ea­ger to tout piv­otal win, Omeros how­ev­er keeps key pa­ra­me­ters shroud­ed

Omeros has brought some stel­lar piv­otal da­ta to the FDA to build a case for its blood clot drug, nar­so­plimab. They just can’t say ex­act­ly how they got to the con­clu­sion yet.

Gre­go­ry De­mop­u­los

Re­port­ing pre­lim­i­nary da­ta from its sin­gle-arm, open-la­bel tri­al, the Seat­tle-based biotech said 56% of the pa­tients — who were ex­pe­ri­enc­ing throm­bot­ic mi­croan­giopa­thy fol­low­ing hematopoi­et­ic stem cell trans­plants — re­ceiv­ing at least one dose of nar­so­plimab achieved com­plete re­spon­der sta­tus. Among the sub­set who re­ceived at least four weeks of dos­ing, 68% met the cri­te­ria.

So what sep­a­rates a re­spon­der from a non-re­spon­der?

That will have to “re­main con­fi­den­tial for com­pet­i­tive busi­ness rea­sons,” the com­pa­ny said in a state­ment, stress­ing that FDA reg­u­la­tors agreed to the “high­ly rig­or­ous” set of re­sponse cri­te­ria.”

Omeros shares $OMER trad­ed up 7.65% to $15.47.

While Omeros had orig­i­nal­ly des­ig­nat­ed “clin­i­cal ac­tiv­i­ty as as­sessed by platelet count” as the pri­ma­ry end­point, the com­pa­ny changed it at the re­quest of the FDA, a spokesper­son told End­points News. The sec­ondary end­points were al­so mod­i­fied.

Rafael Duarte

They in­clude: 100-day sur­vival rates of 65% among those re­ceiv­ing at least one dose, 81% among those who re­ceived at least four weeks of treat­ment, and 93% among the com­plete re­spon­der group; “sub­stan­tial and sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in platelet count, LDH and hap­to­glo­bin”; and in­creased he­mo­glo­bin across all groups.

“The re­sponse rate in this high-risk pop­u­la­tion would be ex­pect­ed to be 10 to 15 per­cent with a 100-day sur­vival rate of less than 20 per­cent,” said Rafael Duarte, who heads the hematopoi­et­ic pro­gram at Uni­ver­si­ty Hos­pi­tal Puer­ta de Hi­er­ro Ma­jada­hon­da in Madrid, in a glow­ing state­ment. “The re­sponse rate and 100-day sur­vival achieved with nar­so­plimab in this tri­al demon­strate an un­prece­dent­ed ef­fect in this con­di­tion.”

Omeros dis­closed that 21% of pa­tients died dur­ing the tri­al “due to caus­es com­mon in stem cell trans­plant” but no oth­ers dis­con­tin­ued for ad­verse events, which ranged from nau­sea, vom­it­ing and di­ar­rhea to hy­pokalemia, neu­trope­nia and fever.

The tri­al was de­signed to en­roll 89 pa­tients, al­though the com­pa­ny didn’t spec­i­fy the fi­nal num­ber in­clud­ed for the analy­sis.

In a pre­vi­ous da­ta cut in­volv­ing 19 pa­tients, Omeros high­light­ed that the drug helped ex­tend me­di­an over­all sur­vival to 347 days, a big im­prove­ment com­pared to the 21 days record­ed in lit­er­a­ture.

The rolling BLA that they ini­ti­at­ed in Oc­to­ber is ex­pect­ed to be com­plet­ed by the first half of next year, ac­cord­ing to CEO Gre­go­ry De­mop­u­los: “the non­clin­i­cal sec­tions have been sub­mit­ted and the da­ta from this tri­al form the ef­fi­ca­cy ba­sis of the ap­pli­ca­tion.”

Pre­vi­ous­ly known as OMS721, nar­so­plimab is dec­o­rat­ed with or­phan drug and break­through ther­a­py des­ig­na­tions from the FDA. If ap­proved, the MASP-2 in­hibitor would be Omeros’ sec­ond drug on the mar­ket.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Ed Kaye, Stoke Therapeutics CEO

Stoke touts ear­ly signs of ef­fi­ca­cy for Dravet syn­drome drug

Two and a half years after driving his antisense oligonucleotide platform to Wall Street, Stoke Therapeutics CEO Ed Kaye is painting a fuller picture of the company’s first clinical data. And though the trial wasn’t powered to detect statistical significance, Kaye says the readout shows early signs of efficacy in kids with a rare, drug-resistant form of epilepsy

STK-001 was well-tolerated in single and multiple doses in 22 Dravet syndrome patients between the ages of 2 and 18 years old, Stoke announced on Friday. What’s more, 12 of the 17 evaluable patients at the time (70.6%) saw reductions from baseline in convulsive seizure frequency, according to the company.