UP­DAT­ED: Astel­las walks away from $450M+ Cy­to­ki­net­ics pact af­ter tak­ing a few ex­tra months to see progress

Months af­ter los­ing long­time part­ner Am­gen on its lead heart drug, Cy­to­ki­net­ics is wav­ing good­bye to an­oth­er al­ly — and with it, more than $450 mil­lion in po­ten­tial mile­stones.

Cy­to­ki­net­ics said in an SEC fil­ing last week that Astel­las is of­fi­cial­ly walk­ing away from a pact to re­search skele­tal sar­com­ere ac­ti­va­tors for dis­eases as­so­ci­at­ed with mus­cle weak­ness. The com­pa­nies had ex­tend­ed the re­search term of their part­ner­ship — which was ini­tial­ly set to ex­pire on Dec. 31 — through March of this year to fin­ish work they couldn’t get done dur­ing the pan­dem­ic, Cy­to­ki­net­ics CEO Robert Blum told End­points News. But on April 27, Astel­las gave writ­ten no­tice that it’s hit­ting the ex­it.

Ac­cord­ing to the fil­ing, Astel­las is giv­ing up “all li­cens­es and oth­er rights” grant­ed through the pact.

The re­search, de­vel­op­ment and com­mer­cial­iza­tion pact was ini­tial­ly struck back in 2013. But in April 2020, Astel­las re­turned the rights to relde­sem­tiv, Cy­to­ki­net­ics’ can­di­date to treat mus­cle wast­ing, and oth­er fast skele­tal reg­u­la­to­ry ac­ti­va­tor (FS­RA) com­pounds that were sub­ject to the orig­i­nal agree­ment. Astel­las agreed to pay one-third of the out-of-pock­et costs for Cy­to­ki­net­ics’ con­tin­ued Phase III tri­al for the drug in ALS, in re­turn for “low- to mid-sin­gle dig­it” roy­al­ties in the US, Cana­da, UK and EU for ei­ther 10 years af­ter the first sale, or un­til 2034, ac­cord­ing to an SEC fil­ing.

“The re­search pro­gram that was ex­tend­ed was done with joint agree­ment, just so we could wind down on a cou­ple of mat­ters that we agreed we want­ed to put to bed be­fore we stepped away,” Blum said“We joint­ly de­cid­ed that we were go­ing to fin­ish the re­search to­geth­er, and then go our sep­a­rate ways.”

End­points reached out to Astel­las, but did not re­ceive a re­sponse as of press time.

Cy­to­ki­net­ics is left with­out the op­por­tu­ni­ty to scoop up some ma­jor mile­stones laid out in the orig­i­nal deal. Ac­cord­ing to the deal, Astel­las could have nabbed ex­clu­sive rights to co-de­vel­op and sell the skele­tal sar­com­ere ac­ti­va­tors in all in­di­ca­tions, leav­ing Cy­to­ki­net­ics the right to co-pro­mote and do cer­tain sales work in the US, Cana­da and/or Eu­rope “un­der agreed sce­nar­ios.”

If Astel­las want­ed to take any of the can­di­dates for­ward on its own, it would have owed Cy­to­ki­net­ics $25 to $35 mil­lion per prod­uct, plus up to $250 mil­lion more for all prod­ucts and an­oth­er $200 mil­lion in com­mer­cial mile­stones. On top of all that, Cy­to­ki­net­ics would have been set to re­ceive roy­al­ties in the “mid-sin­gle-dig­it lev­el to low dou­ble-dig­its.”

The news comes about two years af­ter relde­sem­tiv flopped in a Phase II ALS tri­al. At the time, in­ves­ti­ga­tors said the study failed on change from base­line in slow vi­tal ca­pac­i­ty (SVC) af­ter 12 weeks of dos­ing (p=0.11). The analy­sis al­so showed poor p-val­ues for the ALS Func­tion­al Rat­ing Scale and slope of the Mus­cle Strength Mega-Score.

But de­spite fail­ing the pri­ma­ry end­point, the com­pa­ny ar­gued that the da­ta re­flect­ed “clin­i­cal­ly mean­ing­ful mag­ni­tudes of ef­fect” across all dose lev­els, and main­tained that the place­bo group de­clined at a slow­er rate than ex­pect­ed, which didn’t help.

“Re­sults from FOR­TI­TUDE-ALS are among the most im­pres­sive we have seen in a Phase II clin­i­cal tri­al in ALS,” lead in­ves­ti­ga­tor Je­re­my Shefn­er said back in 2019.

That wasn’t the first or last time Cy­to­ki­net­ics has tout­ed weak or failed da­ta. Back in No­vem­ber, the com­pa­ny cheered a Phase III read­out for its Am­gen-part­nered heart drug ome­cam­tiv mecar­bil. But while the can­di­date met the pri­ma­ry com­pos­ite end­point, it missed a sec­ondary end­point which an­a­lysts had been fol­low­ing as a key mark­er for suc­cess — re­duc­tion of car­dio­vas­cu­lar death.

Lat­er that month, Am­gen shrugged off its 14-year al­liance with Cy­to­ki­net­ics, leav­ing the com­pa­ny to sol­dier on alone.

“Car­dio­vas­cu­lar dis­ease is one of the most sig­nif­i­cant pub­lic health is­sues in the world which means pa­tients need more in­no­va­tion, not less,” Am­gen said in a state­ment. “… Un­for­tu­nate­ly, the re­sults of GALAC­TIC-HF did not meet the high bar we had set for the pro­gram.”

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.

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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.

Tim Miller, Forge Biologics CEO

CNS-fo­cused gene ther­a­py biotech forges man­u­fac­tur­ing deal for hear­ing loss drug

A growing gene therapy company is teaming up with one of the largest gene therapy manufacturers to get its drug into patients in clinical trials.

Myrtelle announced on Monday that it inked a deal with Forge Biologics to make Myr-201, a gene therapy indicated for monogenic hearing loss. The target patient population, Myrtelle said, is people with a type of hearing loss called DFNB8, or autosomal recessive deafness 8.

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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 has set Dec. 13 as the day that its Cardiovascular and Renal Drugs Advisory Committee will review Cytokinetics’ potential heart drug, setting up a key vote ahead of a Feb. 28, 2023 PDUFA date.

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.

Benjamine Liu, TrialSpark CEO

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A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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