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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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