EMA en­dors­es Teva's mi­graine drug; Dis­as­ter prone CTI Bio­Phar­ma re­scinds EU ap­pli­ca­tion for pa­cri­tinib

→ The EMA was busy on Fri­day. The Eu­ro­pean drug reg­u­la­tor, which is cur­rent­ly in the process of shift­ing its head­quar­ters to Am­s­ter­dam from Lon­don, rec­om­mend­ed the ap­proval of Te­va’s mi­graine-pre­ven­tion drug, Ajovy. A fi­nal de­ci­sion is ex­pect­ed in the first half of this year, and if ap­proved the treat­ment drug will be the first and on­ly an­ti-CGRP med­i­cine in the EU with both quar­ter­ly and month­ly dos­ing op­tions, the Is­raeli drug­mak­er said. CTI Bio­Phar­ma, how­ev­er, had bad news to re­port on the Eu­ro­pean front for its myelofi­bro­sis drug pa­cri­tinib. The no­to­ri­ous­ly fail­ure-prone drug de­vel­op­er an­nounced it was with­draw­ing an ap­pli­ca­tion to mar­ket the drug in the EU, fol­low­ing dis­cus­sions with the EMA. Last year, Shire backed away from a part­ner­ship with the ill-fat­ed com­pa­ny, fol­low­ing a mixed da­ta read­out from a piv­otal study that was pre­vi­ous­ly put on a full clin­i­cal hold — un­der the be­hest of the FDA — af­ter the death of sev­er­al pa­tients. The com­pa­ny’s shares $CTIC tum­bled more than 15% in ear­ly Fri­day trad­ing.

→ Sep­a­rate­ly on Fri­day, the EMA al­so laid out guid­ance to help bio­phar­ma com­pa­nies pre­pare for Brex­it, so as to en­sure sup­ply is not dis­rupt­ed — on the as­sump­tion the UK will be­come a third coun­try as of 30 March 2019. For pre-sub­mis­sion meet­ings re­gard­ing hu­man and an­i­mal drugs re­quest­ed from last Oc­to­ber, the EMA will en­gage with man­u­fac­tur­ers via tele­con­fer­ence or vir­tu­al meet­ings. Be­tween 11 Feb­ru­ary and 15 March 2019, no such meet­ings for ini­tial mar­ket­ing au­tho­ri­sa­tion ap­pli­ca­tions will take place, while the EMA moves to its new home in Am­s­ter­dam, it said.

→ On Thurs­day at a par­lia­men­tary com­mit­tee meet­ing, UK health and so­cial care sec­re­tary Matt Han­cock said the British gov­ern­ment was pri­or­i­tiz­ing med­i­cines over food if a no-deal Brex­it would make it tricky to im­port prod­ucts in ei­ther cat­e­go­ry of goods. Last year, Han­cock  asked drug sup­pli­ers to keep six weeks worth of med­i­cines stock­piled in ad­di­tion to their buffer stocks and last month the UK’s Med­i­cines and Health­care prod­ucts Reg­u­la­to­ry Agency (MHRA) said that in the event of a no-deal, the UK would be no longer be part of the EMA um­brel­la, and that reg­u­la­to­ry sub­mis­sions for drugs would be made di­rect­ly to the MHRA.

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

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, and could help shore up public trust in the agency and any eventual vaccine.

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.

Vas Narasimhan (AP Images)

UP­DAT­ED: 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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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

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.