Ab­b­Vie scores speedy re­view for RA drug upadac­i­tinib; Mer­ck KGaA wins CRISPR patent

→ Ab­b­Vie $AB­BV has high hopes, pro­ject­ing peak po­ten­tial sales of $6.5 bil­lion, for its oral JAK1 in­hibitor upadac­i­tinib for rheuma­toid arthri­tis, which is up against well-en­trenched ri­vals. On Tues­day, the drug­mak­er said FDA had re­ward­ed the drug — which is al­so un­der EU re­view — pri­or­i­ty re­view, and that the US health reg­u­la­tor is ex­pect­ed to makes its de­ci­sion by the third quar­ter of 2019.

Pfiz­er has nabbed the Eu­ro­pean green light for its Avastin copy­cat — its sec­ond on­col­o­gy biosim­i­lar in the con­ti­nent— in a slate of can­cer in­di­ca­tions from car­ci­no­ma of the colon or rec­tum to non-small cell lung can­cer. The launch of Zirabev would fol­low a trail blazed by Am­gen and Al­ler­gan with Mvasi, which was ap­proved by the EU last year and by the FDA a few months pri­or.

→ While the CRISPR IP land­scape is as con­tentious as ever with the cus­tom­ary play­ers bat­tling it out, Ger­many’s Mer­ck has swooped in with a patent for proxy-CRISPR, an im­proved ver­sion of tra­di­tion­al CRISPR that makes the gene-edit­ing tech­nique “more ef­fi­cient, flex­i­ble and spe­cif­ic by open­ing the genome for mod­i­fi­ca­tion of DNA.”

In oth­er CRISPR news, Gami­da Cell $GM­DA has joined forces with Ed­i­tas Med­i­cine $ED­IT to ed­it NAM-NK cells — nat­ur­al killer cells that have been ex­pand­ed us­ing Gami­da Cell’s nicoti­namide-based, or NAM, tech­nol­o­gy — to im­prove the treat­ment of blood can­cers and sol­id tu­mors. The fi­nan­cial terms of the deal were not dis­closed. Gami­da Cell’s NAM-NK im­munother­a­py is cur­rent­ly un­der de­vel­op­ment in Phase I tri­als for use pa­tients with re­frac­to­ry non-Hodgkin lym­phoma and mul­ti­ple myelo­ma.

Si­mon Mo­roney

Ger­man can­cer drug­mak­er Mor­phoSys $MOR is los­ing one of its founders. CEO Si­mon Mo­roney, who has led the com­pa­ny for 27 years, has told the com­pa­ny’s board he in­tends to re­tire. Mo­roney will step down when his cur­rent con­tract ex­pires on June 30, 2020, or when a suc­ces­sor is ap­point­ed, whichev­er comes soon­er. In 2017, Trem­fya, sold by J&J $JNJ for use in plaque pso­ri­a­sis, be­came the first drug based on Mor­phoSys’s an­ti­body tech­nol­o­gy to re­ceive reg­u­la­to­ry ap­proval.

Is­raeli biotech Bioblast Phar­ma $ORPN has sold its Tre­halose clin­i­cal de­vel­op­ment pro­grams to See­los Ther­a­peu­tics $SEEL for $3.5 mil­lion in two in­stall­ments, and up to $17 mil­lion in mile­stone pay­ments.

Apart from Ar­a­digm $ARDM and Im­mune Phar­ma­ceu­ti­cals $IM­NPPernix Ther­a­peu­tics $PTX has al­so filed for Chap­ter 11 to un­der­take its re­struc­tur­ing and sale process, which it hopes to con­clude by May.

→ Gilead’s $GILD CAR-T ther­a­py Yescar­ta has notched an­oth­er ap­proval, this time in Cana­da for re­lapsed or re­frac­to­ry large B-cell lym­phoma in adult pa­tients who have un­der­gone at least two sys­temic ther­a­pies.

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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