My­ovant adds re­l­u­golix da­ta in uter­ine fi­broids ahead of June PDU­FA; Mubadala teams up with UK for $1B+ fund

As My­ovant bar­rels to­ward its PDU­FA date for re­l­u­golix in uter­ine fi­broids, the biotech is beef­ing up its da­ta pack­age with new Phase III re­sults.

A Phase III ran­dom­ized with­draw­al study met its pri­ma­ry end­point and all sec­ondary end­points, My­ovant and Pfiz­er an­nounced Wednes­day morn­ing, eval­u­at­ing a once-dai­ly re­l­u­golix com­bo ther­a­py with estra­di­ol and norethin­drone ac­etate. The FDA has marked June 1 down on the cal­en­dar for a reg­u­la­to­ry de­ci­sion.

My­ovant en­rolled 229 el­i­gi­ble women who had pre­vi­ous­ly com­plet­ed a long-term ex­ten­sion study, and sought to ex­am­ine ben­e­fits of the ex­per­i­men­tal drug in con­tin­ued treat­ment for up to two years. All of the pa­tients took the treat­ment for a year be­fore be­ing ran­dom­ized to con­tin­ue the drug or to place­bo for an­oth­er year.

At Week 76, the pri­ma­ry end­point mea­sure­ment date, 78.4% of women in the con­tin­ued treat­ment arm achieved the sus­tained re­spon­der rate com­pared to just 15.1% of those in place­bo. That re­sult­ed in a stel­lar p-val­ue of p<0.0001.

The com­bo ther­a­py al­so hit all three sec­ondary end­points at the same p-val­ue, which in­clud­ed mea­sure­ments of sus­tained re­spon­der rate at two years, time to re­lapse of heavy men­stru­al bleed­ing, and amen­or­rhea rate.

The drug has al­ready been ap­proved as a monother­a­py for ad­vanced prostate can­cer, dubbed Or­govyx, be­com­ing the first once-a-day pill to treat the con­di­tion. It was priced at $2,300 per month, for­mer CEO Lynn Seely told End­points News at the time. Or­govyx us­es the same path­way to low­er testos­terone in men as it does to low­er es­tro­gen and prog­es­terone in women.

UAE state fund teams up with UK for new in­vest­ments

The UAE and the UK have teamed up on a new life sci­ences fund that plans to raise bil­lions for the British biotech sec­tor.

Abu Dhabi’s Mubadala, one of the coun­try’s state-backed funds, is plan­ning to spend £800 mil­lion over the first five years of the deal. The cash will be fund­ed along­side £200 mil­lion from a British gov­ern­ment pro­gram an­nounced last year.

The Fi­nan­cial Times was the first to re­port the news Tues­day.

Clinch­ing the deal with Mubadala was seen in the UK as a ma­jor win for Prime Min­is­ter Boris John­son as he seeks to carve out new trade deals fol­low­ing Brex­it. In ad­di­tion to biotech, the agree­ment will help fund “en­er­gy tran­si­tion and in­fra­struc­ture” pro­grams in both coun­tries.

“This is a ma­jor win for the Of­fice for In­vest­ment and shows how the UK is an in­vest­ment des­ti­na­tion of choice,” UK in­ter­na­tion­al trade sec­re­tary Liz Truss said in a state­ment. “From Liv­er­pool and Ed­in­burgh to Ox­ford and Not­ting­ham, our world class life sci­ences clus­ters and in­no­v­a­tive busi­ness­es will see the ben­e­fits of this part­ner­ship.”

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.