Omeros plunges deep­er af­ter FDA re­jects rare dis­ease drug, ask­ing for more in­for­ma­tion

Omeros prac­ti­cal­ly warned in­vestors that a com­plete re­sponse let­ter was com­ing when it dis­closed that the FDA found de­fi­cien­cies in its BLA for nar­so­plimab. But the agency did not elab­o­rate on the specifics of those de­fi­cien­cies for the drug, which was be­ing po­si­tioned as a treat­ment for the rare but se­ri­ous blood clot­ting dis­ease known as hematopoi­et­ic stem cell trans­plant-as­so­ci­at­ed throm­bot­ic mi­croan­giopa­thy (HSCT-TMA).

The oth­er shoe has now dropped.

In the CRL, the FDA “ex­pressed dif­fi­cul­ty in es­ti­mat­ing the treat­ment ef­fect of nar­so­plimab in HSCT-TMA and as­sert­ed that ad­di­tion­al in­for­ma­tion will be need­ed to sup­port reg­u­la­to­ry ap­proval,” ac­cord­ing to Omeros.

While it wouldn’t be en­tire­ly sur­pris­ing to some, the re­jec­tion still star­tled oth­er in­vestors, who sent shares $OMER down 27.04% to $5.64.

TMA — a con­stel­la­tion of symp­toms caused by the for­ma­tion of tiny blood clots in the body — is one of the con­di­tions that can plague pa­tients who have gone through an HSCT. Omeros be­lieves it’s large­ly be­cause stem cell trans­plants trig­ger en­dothe­lial dam­age, which in turns ac­ti­vates the com­ple­ment sys­tem, pri­mar­i­ly through the lectin com­ple­ment path­way.

By block­ing an en­zyme on the lectin path­way dubbed MASP-2, nar­so­plimab was de­signed to ad­dress that ex­act cause.

It’s not ex­act­ly alone in pin­ning the blame on the com­ple­ment sys­tem. Alex­ion’s C5 in­hibitor Soliris is of­ten pre­scribed off-la­bel for this use as well as trans­plant-as­so­ci­at­ed TMA; in fact, some of the in­di­ca­tions it’s ap­proved in can be char­ac­ter­ized as TMA. But nar­so­plimab was the first can­di­date to be sub­mit­ted specif­i­cal­ly for HSCT-TMA.

For its part, Omeros says it re­mains con­fi­dent about its drugs’ ef­fi­ca­cy and safe­ty da­ta — from a sin­gle-arm piv­otal tri­al that was com­pared to his­tor­i­cal con­trols, and em­ployed clin­i­cal re­sponse as the pri­ma­ry end­point — and plans to re­quest a Type A meet­ing with reg­u­la­tors.

“There were no chem­istry, man­u­fac­tur­ing and con­trols (CMC), safe­ty, or non-clin­i­cal is­sues pre­clud­ing ap­proval raised in the CRL,” the com­pa­ny added, while not­ing the drug is adorned with break­through and or­phan des­ig­na­tions.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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Elcin Barker Ergun, Menarini Group CEO

Amid Roche and Sanofi's oral SERD set­backs, Menar­i­ni gets speedy re­view at FDA

Menarini and Radius Health are getting a speedy review at the FDA for their oral SERD breast cancer drug months after the field opened up with competitors failing and fleeing.

It was a one-two-three punch in March, April and May as Sanofi flunked its first big test for its oral selective estrogen receptor degrader (SERD), Roche also flamed out in a Phase II and G1 Therapeutics ended its program after scoping out the data and potential partners.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.