FDA ac­cepts Bris­tol My­er­s' pitch for for­mer MyoKar­dia drug; Clo­vis' Rubra­ca aces ad­vanced ovar­i­an can­cer study

The pro­gram at the cen­ter­piece of Bris­tol My­ers Squibb’s buy­out of MyoKar­dia just got one step clos­er to gain­ing po­ten­tial FDA ap­proval.

BMS an­nounced Fri­day that the agency has ac­cept­ed its NDA pitch, sched­ul­ing a Jan. 28, 2022 PDU­FA date for the ex­per­i­men­tal drug mava­camten. The com­pa­ny is shoot­ing for an ini­tial in­di­ca­tion of symp­to­matic ob­struc­tive hy­per­trophic car­diomy­opa­thy, or oHCM.

In the dis­ease, a mu­ta­tion in pro­teins forces the heart to ex­ert more ef­fort and squeeze more, block­ing the out­flow of blood and thick­en­ing mus­cle. Mava­camten is an oral al­losteric mod­u­la­tor of car­diac myosin, in­tend­ed to re­duce con­trac­tion by the heart mus­cle.

But BMS is bet­ting heav­i­ly that the pro­gram could turn in­to a po­ten­tial block­buster af­ter pony­ing up more than $13 bil­lion in their ac­qui­si­tion of MyoKar­dia. The price per share, $225, rep­re­sent­ed a 61% pre­mi­um.

Fri­day’s NDA ac­cep­tance was based on a Phase III study de­signed to cap­ture both symp­toms and car­diac func­tion. Read­ing out da­ta in May 2020, mava­camten beat place­bo with a p-val­ue of p=0.0005, in ad­di­tion to de­liv­er­ing stel­lar num­bers on a slate of sec­ondary end­points. And lat­er that year, MyoKar­dia re­port­ed that the ben­e­fits ex­tend­ed to all sub­groups.

The ac­qui­si­tion last Oc­to­ber rep­re­sent­ed a move away from on­col­o­gy for BMS, where the com­pa­ny has strug­gled to grow its Op­di­vo fran­chise in the wake of con­tin­ued Keytru­da suc­cess from Mer­ck. — Max Gel­man

Clo­vis’ Rubra­ca hits its marks in PhI­II ovar­i­an can­cer study

Patrick Ma­haffy

Clo­vis On­col­o­gy’s PARP in­hibitor Rubra­ca hit its pri­ma­ry end­point for pro­gres­sion-free sur­vival over chemo in BR­CA-mu­tat­ed, ad­vanced ovar­i­an can­cer pa­tients who have re­ceived two or more pri­or lines of chemo, ac­cord­ing to top-line da­ta pre­sent­ed Fri­day at the vir­tu­al So­ci­ety of Gy­ne­co­log­ic On­col­o­gy meet­ing.

Pa­tients treat­ed with Rubra­ca in the ARIEL4 study post­ed a me­di­an PFS of 7.4 months com­pared with 5.7 months in the chemo arm. The study en­rolled 349 women in Eu­rope, Is­rael and North and South Amer­i­ca.

“The ARIEL4 da­ta add to the grow­ing sci­en­tif­ic un­der­stand­ing about the clin­i­cal uti­liza­tion of Rubra­ca com­pared to chemother­a­py, in­clud­ing plat­inum-based chemother­a­py, for women di­ag­nosed with BR­CA mu­ta­tion-pos­i­tive ad­vanced ovar­i­an can­cer,” Clo­vis CEO Patrick Ma­haffy said in a state­ment.

In terms of safe­ty, Rubra­ca post­ed sim­i­lar side ef­fect rates as in pre­vi­ous stud­ies, Clo­vis said, with ane­mia the most com­mon­ly re­port­ed Grade 3 and high­er event. — Kyle Blanken­ship

Sanofi wants out of gene ther­a­py deal with Ox­ford Bio­Med­ica

Gene ther­a­py play­er Biover­a­tiv inked a $100 mil­lion deal for ac­cess to Ox­ford Bio­Med­ica’s lentivirus­es to treat he­mo­phil­ia back in 2018 — just a month af­ter the Bio­gen spin­off was snatched by Sanofi for $11.6 bil­lion. Now, Sanofi says it wants out.

Sanofi has giv­en no­tice that it in­tends to ter­mi­nate the col­lab­o­ra­tion with Ox­ford Bio­Med­ica, ac­cord­ing to a state­ment.

Ox­ford Bio­Med­ica got $5 mil­lion up­front, and was el­i­gi­ble for mile­stones up to $100 mil­lion, along with roy­al­ties on sales of the he­mo­phil­ia treat­ments Biover­a­tiv was de­vel­op­ing.

In 2019, Sanofi watched the mar­ket for one of those drugs — the he­mo­phil­ia treat­ment Eloc­tate — shriv­el in the face of Roche’s star, Hem­li­bra.

“It is a non-cash event of course but it is clear that the com­pe­ti­tion from Hem­li­bra was a sur­prise to every­one and it is in­deed putting pres­sure,” Sanofi chief fi­nan­cial of­fi­cer Jean-Bap­tiste de Chatil­lon told re­porters, ac­cord­ing to a re­port in Reuters. — Nicole De­Feud­is 

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lymphoma Society has tapped 5 new early-stage projects to back with up to $10 million each in fresh investments. The 5 biotechs are:

— Caribou, headed by Rachel Haurwitz and co-founded by Jennifer Doudna, is working on next-gen, off-the-shelf CAR-Ts to replace the patient-derived cells now in use.

— The LLS supported NexImmune’s IPO, helping fund its work on nanoparticles that can gin up an immune response directed at cancer cells. The biotech has 2 projects now in Phase I trials.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.