At­lantic Health­care's drug fails late-stage test; Trans­late Bio breaks out ear­ly CF da­ta

→ Cam­bridge, UK-based At­lantic Health­care dis­closed that the late-stage pro­gram for its ex­per­i­men­tal drug, al­i­cafors­en en­e­ma, has failed in pa­tients with pou­ch­i­tis, a pro­gres­sive dis­ease char­ac­ter­ized by in­flam­ma­tion, ul­cer­a­tion and in­creas­ing­ly un­con­trolled, fre­quent and ur­gent emp­ty­ing of the bow­el (up to 10-20 times a day and night). “Al­though we are dis­ap­point­ed that the Phase 3 tri­al did not achieve sta­tis­ti­cal sig­nif­i­cance, we be­lieve the per­cent­age of pa­tients achiev­ing re­mis­sion in stool fre­quen­cy and the en­do­scop­ic re­sponse ob­served in a num­ber of sub­pop­u­la­tions of pa­tients could be note­wor­thy,” said chief To­by Wil­son Wa­ter­worth. “Hav­ing con­sult­ed with key opin­ion lead­ers and reg­u­la­to­ry ad­vi­sors, we now plan to meet with the U.S. Food and Drug Ad­min­is­tra­tion and Eu­ro­pean Med­i­cines Agency to dis­cuss a path­way to reg­u­la­to­ry ap­proval.”

→ mR­NA ther­a­py de­vel­op­er Trans­late Bio $TBIO un­veiled some ear­ly da­ta from a 12-pa­tient tri­al on its ex­per­i­men­tal in­haled ther­a­py, MRT5005, for pa­tients with cys­tic fi­bro­sis (CF) on Wednes­day. CF oc­curs when the cys­tic fi­bro­sis trans­mem­brane con­duc­tance reg­u­la­tor (CFTR) pro­tein is ei­ther not made cor­rect­ly, or not made at all, and is char­ac­ter­ized by a build-up of thick sticky mu­cus in the lungs, di­ges­tive sys­tem and oth­er or­gans. While there was vari­abil­i­ty seen per­cent pre­dict­ed forced ex­pi­ra­to­ry vol­ume in 1 sec­ond (ppFEV1) in pa­tients from day to day — some im­prove­ments sup­port a CFTR-re­lat­ed mech­a­nism and may sug­gest that MRT5005 can en­able the pro­duc­tion of func­tion­al pro­tein, said Steven Rowe, the prin­ci­pal in­ves­ti­ga­tor of the Phase I/II study. “If fu­ture da­ta con­firms this ear­ly pos­i­tive sig­nal, MRT5005 has the po­ten­tial to pro­vide a clin­i­cal­ly mean­ing­ful ben­e­fit for peo­ple with CF in­de­pen­dent of CFTR geno­type.”

→ In­vest­ment firm EQT is tak­ing a ma­jor­i­ty stake in North Dako­ta-based Alde­vron, a glob­al sup­pli­er of plas­mid DNA used in gene ther­a­pies, as well as pro­teins, an­ti­bod­ies and mR­NA. The com­pa­ny’s founders, man­age­ment and TA As­so­ci­ates will re­tain a mi­nor­i­ty stake.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Fireside chat between Hal Barron and John Carroll, UKBIO19

It’s time we talked about bio­phar­ma — live in Lon­don next week

Zoom can only go so far. And I think at this stage, we’ve all tested the limits of staying in touch — virtually. So I’m particularly happy now that we’ve revved up the travel machine to point myself to London for the first time in several years.

Whatever events we have lined up, we’ve always built in plenty of opportunities for all of us to get together and talk. For London, live, I plan to be right out front, meeting with and chatting with the small crowd of biopharma people we are hosting on October 12 at Silicon Valley Bank’s London headquarters. And there’s a lengthy mixer at the end I’m most looking forward to, with several networking openings between sessions.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

Pfizer and BioNTech's original Marvel comic book links evolving Covid vaccine science to Avengers' evolving villain-fighting tools.(Source: Pfizer LinkedIn post)

Pfiz­er, BioN­Tech part­ner with Mar­vel for Avengers and Covid-fight­ing com­ic book

Pfizer and BioNTech are collaborating with Marvel to celebrate “everyday” people getting Covid-19 vaccines in a custom comic book.

In the “Everyday Heroes” digital comic book, an evolving Ultron, one of the Avengers’ leading villains, is defeated by Captain America, Ironman and others. The plotline and history of Ultron is explained by a grandfather who is waiting with his family at a clinic for Covid-19 vaccinations.

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FDA+ roundup: Ad­comm date set for Cy­to­ki­net­ics heart drug; New gener­ic drug guid­ance to re­duce fa­cil­i­ty de­lays

The FDA on Wednesday set Dec. 13 as the day that its Cardiovascular and Renal Drugs Advisory Committee will review Cytokinetics’ potential heart drug, meaning regulators aren’t likely to meet the Nov. 30 PDUFA date that was previously set.

The drug, known as omecamtiv mecarbil, read out its first Phase III in November 2020, hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as the key to breaking into the market, failing to significantly differ in reducing cardiovascular death from placebo.

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Car­olyn Bertozzi (Illustration: Assistant editor Kathy Wong for Endpoints News)

Car­olyn Bertozzi, re­peat biotech founder and launch­er of a field, shares in chem­istry No­bel win

Carolyn Bertozzi, predicted by some to become a Nobel laureate, clinched one of the world’s top awards in the wee hours of Wednesday, winning the Nobel Prize in Chemistry alongside a repeat winner and a Copenhagen researcher.

The Stanford professor, Morten Meldal of University of Copenhagen and 2001-awardee K. Barry Sharpless of Scripps shared the prize equally. The Nobel is sometimes split in quarters and/or halves.

Eli Lil­ly and Te­va pre­pare for court bat­tle over mi­graine med ri­val­ry

It looks like Eli Lilly and Teva Pharmaceuticals are going to trial.

A federal appeals court on Monday refused to invalidate three of Teva’s patents for its migraine treatment Ajovy, while also declining to issue a summary judgment in favor of either company, which would effectively end the case without a full trial.

Teva filed suit against Lilly back in 2018, alleging that the company infringed upon nine patents with its rival migraine drug Emgality. The rival drugs were both approved in September 2018 for the preventative treatment of migraine, and are designed to block calcitonin gene-related peptide (CGRP), a protein associated with the onset of migraine pain.

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Rep. Vern Buchanan (R-FL) (Bill Clark/CQ Roll Call via AP Images)

Af­ter cov­er­age re­stric­tions for Alzheimer's drugs, bi­par­ti­san House bill would force CMS to re­view drugs in­di­vid­u­al­ly

When Biogen’s controversial Alzheimer’s drug Aduhelm was hit with a national decision from CMS that restricted coverage to only randomized trials, practically guaranteeing a commercial flop in the near term, questions surfaced over why CMS also included all amyloid-targeted monoclonal antibodies for Alzheimer’s disease.

With Eisai and Biogen’s second Alzheimer’s drug, lecanemab, now showing it can slow the rate of cognitive decline versus placebo, lining up for a likely full approval next spring, the question now turns to whether that data, which is being presented at the Clinical Trials on Alzheimer’s Congress in San Francisco in late November, will be enough for CMS when it asks, “Does the anti-amyloid mAb meaningfully improve health outcomes (i.e., slow the decline of cognition and function) for patients in broad community practice?”

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