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

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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