Re­gen­eron ditch­es high­er dose of pain drug fas­inum­ab in PhI­II, trig­ger­ing safe­ty scare; In a small win for Al­ny­lam, FDA de­lays re­view for Ion­is' in­ot­ersen

→ Re­gen­eron $REGN has canned its high dose arm of fas­inum­ab, an os­teoarthri­tis and back pain drug be­ing test­ed in Phase III tri­als, fol­low­ing rec­om­men­da­tions from an in­de­pen­dent da­ta mon­i­tor­ing com­mit­tee. A risk-ben­e­fit as­sess­ment was done on the high­er dose of the drug, and the com­mit­tee found it want­i­ng.

Re­gen­eron hasn’t yet re­leased any de­tails about the as­sess­ment, but this isn’t the first time fas­inum­ab has come un­der fire. In 2016, the FDA slapped a clin­i­cal hold on the com­pa­ny’s Phase IIb study of the drug in low­er back pain. The hold was trig­gered af­ter a pa­tient in the study suf­fer­ing from ad­vanced os­teoarthri­tis de­vel­oped a case of ad­ju­di­cat­ed arthropa­thy – joint dis­ease.

Drop­ping the high dose is sur­fac­ing con­cerns about drugs like fas­inum­ab, which tar­get nerve growth fac­tor (NGF) to si­lence pain. NGF drugs were all the rage un­til a few years ago, when some of the pa­tients in clin­i­cal stud­ies be­gan to blow out their joints with meds de­signed to si­lence nerve growth fac­tors. The FDA hit J&J, Re­gen­eron, and Pfiz­er with clin­i­cal holds, which were fol­lowed by re­ports of joint re­place­ments in pa­tients tak­ing the drugs. Some spec­u­late that NGF drugs work so well at si­lenc­ing pain, that peo­ple be­come more ac­tive — ac­cel­er­at­ing the pace of car­ti­lage de­te­ri­o­ra­tion at the joints.

The safe­ty is­sues side­lined the drugs, once tapped as like­ly block­busters, but de­vel­op­ers slow­ly worked out a plan to pro­tect pa­tients, and back in­to the clin­ic. Re­gen­eron’s in­vestors will be in­ter­est­ed to see how the low­er dose of fas­inum­ab moves for­ward.

→ As Al­ny­lam $AL­NY pre­pares to hear back from the FDA on its lead­ing drug patisir­an, it can now be sure that it’s in the lead. The FDA has ex­tend­ed the re­view pe­ri­od for in­ot­ersen — a ri­val hered­i­tary TTR amy­loi­do­sis drug de­vel­oped by Io­n­is $IONS — and pushed the PDU­FA date back to Oc­to­ber 6. That’s 90 days lat­er than the orig­i­nal date, and two months af­ter Al­ny­lam’s drug will like­ly have been ap­proved. “The FDA de­ter­mined that they need ad­di­tion­al time to re­view our re­spons­es to their stan­dard in­for­ma­tion re­quests and we are work­ing close­ly with them to ad­vance the re­view of our fil­ing as quick­ly as pos­si­ble,” said Sarah Boyce, pres­i­dent of Akcea $AK­CA, the Io­n­is spin­off now han­dling in­ot­ersen. That slight edge can be key in the in­tense ri­val­ry be­tween the an­ti­sense ex­perts at Al­ny­lam and Io­n­is over a block­buster mar­ket.

→ Un­der the spot­light for an ex­pect­ed surge of biotech list­ings, Hong Kong Stock Ex­change un­veiled a pan­el of biotech in­dus­try vet­er­ans it’s as­sem­bled to re­view list­ing ap­pli­ca­tions. Giv­en the high hopes and se­ri­ous skep­ti­cism sur­round­ing the mar­ket’s abil­i­ty to eval­u­ate pre-rev­enue biotech com­pa­nies — which be­fore this week weren’t al­lowed to list on the ex­change — HKEX has as­sem­bled a star-stud­ded group. The 13-strong pan­el fea­tures Zai Lab founder Saman­tha Du as well as promi­nent VCs Jonathan Wang (of Or­biMed Asia) and Ling Su (of Lil­ly Asia Ven­tures). Oth­er mem­bers in­clude biotech ex­ecs, an­a­lysts and reg­u­la­to­ry pro­fes­sion­als, all of whom have been ap­point­ed for an ini­tial term of two years. In ad­di­tion to ad­vis­ing on ques­tions about prospec­tus dis­clo­sure, the pan­el will al­so serve as a sound­ing board for pol­i­cy rec­om­men­da­tions.

With con­tri­bu­tion by Am­ber Tong

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Bay­er plots a ma­jor facelift at Berke­ley cam­pus, un­cork­ing a 30-year, $1.2B plan to dri­ve cell and gene ther­a­pies

Bayer first set roots in Berkeley back in 1974, when it was still operating as Miles Labs. The site has pumped out three hemophilia A treatments for distribution worldwide; but now, as the pharma continues its cell and gene therapy push, it has something bigger in mind.

Bayer is planning a 30-year revamp at the campus, which includes 918,000 square feet in new buildings and double the jobs, according to a report by the Bay Area Council Economic Institute.

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:

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Steffen Schuster, ITM CEO

Ra­dio­phar­ma re­mains hot as Ger­many's ITM rais­es $109M to ad­vance neu­roen­docrine can­cer pro­gram

The world of radiopharmaceuticals has been heating up over the last few years, and Thursday saw another company focused on the field pull in a new nine-figure raise.

Germany’s ITM, or Isotopen Technologien München, scored a $109 million round of loan financing to push forward its precision oncology pipeline and fund late-stage development for its lead program. As part of the agreement, the loan will convert to shares in the event of future financial or corporate transactions, ITM said.

Noubar Afeyan (Sebastien Micke/Paris Match/Contour by Getty Images)

As Mod­er­na rose, Flag­ship cashed in for $1.4B — with a lot more wealth still re­main­ing

For nearly a decade, Flagship poured record-setting levels of cash into Moderna, even as they faced setbacks on early programs and skeptics wondered whether the company’s science could ever match its hype.

Now that the science has delivered, Flagship is cashing in.

Over the last 13 months, since the World Health Organization declared a pandemic, Flagship has sold off Moderna shares worth $1.4 billion. The sales, first reported by Forbes, came as the Cambridge biotech’s shares soared from just under $20 per share on Jan. 3, 2020, to $169.50 when markets opened Thursday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 107,400+ biopharma pros reading Endpoints daily — and it's free.