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

Janet Woodcock (AP Images)

Janet Wood­cock to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,000+ biopharma pros reading Endpoints daily — and it's free.

Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,000+ biopharma pros reading Endpoints daily — and it's free.

Steve Harr (L) and Hans Bishop

Paint­ing by the num­bers, Sana founders carve up a gi­ant uni­corn-sized IPO — for a biotech that has­n't quite made it to the clin­ic

Sana Biotechnology is one of those startups that was sketched in on the chalkboard day one in the shape of a unicorn.

A giant unicorn.

And from the numbers the cell therapy 2.0 play spelled out in their S-1 $SANA, it’s clear that the company founders — led by a pair of major VCs aligned with some high-profile industry figures — are hunting a big chunk of that value for themselves.

The raise they penciled in — $150 million — isn’t likely what they actually have in mind, and it doesn’t do justice to the size of their ambitions.

Endpoints Premium

Premium subscription required

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

News brief­ing: Five Prime fi­nal­izes PhI­II plans for gas­tric can­cer; AI di­ag­nos­tics-fo­cused Paige ex­pands staff

Five Prime Therapeutics has finalized a plan to take their comeback gastric cancer drug into late-stage studies.

The South San Francisco-based biotech released full Phase II data for bemarituzumab on Friday, which Five Prime said in November met all of its pre-specified efficacy endpoints in a topline readout. Now, the company is announcing it plans to launch a Phase III trial for the program in 2021. Following November’s readout, the future of bemarituzumab had not yet been finalized.

CEO Brett Monia (Ionis)

Can Brett Mo­nia push Io­n­is be­yond Spin­raza?

For 30 years, Brett Monia struggled as one of Ionis’ top scientists to get their antisense technology to work. Now, as CEO, he’s trying to use it to turn Ionis into one of the industry’s biggest biotechs.

Monia, one of the handful of young scientists who in 1989 followed Stanley Crooke across the country from SmithKline (now GSK) in Philadelphia to found Ionis in Northern California, replaced Crooke as CEO last January. By then, they had proven antisense, an RNA-based method for manipulating gene expression, could work dramatically well in at least some instances, transforming spinal muscular atrophy with the Biogen-partnered blockbuster Spinraza.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,000+ biopharma pros reading Endpoints daily — and it's free.

David Kessler in April 2009 (Eric Risberg/AP Images)

Covid-19 roundup: Hack­ers start re­leas­ing 'ma­nip­u­lat­ed' Covid-19 vac­cine docs; Ex-FDA com­mish David Kessler to re­place Mon­cef Slaoui as Op­er­a­tion Warp Speed chief — re­port

There’s a new twist on the EMA Covid-19 hacking story.

Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

This included internal/confidential email correspondence dating from November, relating to evaluation processes for COVID-19 vaccines. Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,000+ biopharma pros reading Endpoints daily — and it's free.

Terry Rosen, Arcus CEO

Gilead part­ner Ar­cus earns an­a­lyst­s' plau­dits for ear­ly pan­cre­at­ic can­cer da­ta that 'ex­ceed­ed ex­pec­ta­tion­s'

Arcus’ small molecule CD73 inhibitor for pancreatic cancer got a standing ovation from analysts who said preliminary data “exceeded expectations”— making waves in a field that’s seen little progress in several years and proving the candidate could be worth the hundreds of millions Gilead provided upfront in a deal that included more than a billion dollars for opt-in rights and milestones.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,000+ biopharma pros reading Endpoints daily — and it's free.

Peter Thiel, Getty (Photographer: Kiyoshi Ota/Bloomberg)

Pe­ter Thiel's psy­che­delics-fo­cused ATAI ac­quires ma­jor­i­ty stake in Recog­ni­fy and its lead schiz­o­phre­nia can­di­date

Billionaire Peter Thiel has made significant and sometimes controversial pushes into life sciences over the past few years, and one of his startups out of Berlin has made a new acquisition less than two months after achieving unicorn status.

ATAI Life Sciences purchased a majority stake Tuesday in Recognify Life Sciences, a company focused on developing treatments for cognitive impairment associated with schizophrenia. The financial terms of the deal weren’t disclosed, but the acquisition follows up a $125 million Series C in November co-led by Thiel, leading to a post-money valuation of about $1 billion for ATAI.

Cog­nate dou­bles man­u­fac­tur­ing ca­pac­i­ties in Mem­phis, Eu­rope, as de­mand for cell and gene ther­a­pies sky­rock­ets

The marketplace for gene and cell manufacturing therapeutics continues to be scorching.

Cognate BioSciences, a leading CDMO specializing in gene and cell therapy technologies, announced plans Friday that will double its total manufacturing capacities at sites in both the US and Europe — in direct response to a “great demand of commercial capacity within the biologics industry.”

The company provided most details for its US expansion, which will take place at its current headquarters in Memphis, Tennessee near the Memphis International Airport — crucial, it said, as Memphis is one of the world’s busiest cargo airports. Cognate will add two separate facilities totaling 250,000-square-feet: a GMP distribution center to manage global supply chain needs, and a third site for commercial manufacturing.