Jon P. Stonehouse, President, Chief Executive Officer of BioCryst

BioCryst bags $250M in a debt and roy­al­ty deal as the ex­ec­u­tive team preps a new drug launch for HAE

Three days af­ter land­ing an FDA OK for their HAE drug bero­tral­stat, BioCryst $BCRX now has some cash to see how far they can go in com­mer­cial­iz­ing the ther­a­py as Or­ladeyo.

The biotech re­port­ed Mon­day morn­ing that they’ve inked a deal with Roy­al­ty Phar­ma that trades a chunk of their rev­enue from the drug for cash in hand. Roy­al­ty is pay­ing out $125 mil­lion up­front, with a $200 mil­lion cred­it fa­cil­i­ty from Athyri­um Cap­i­tal Man­age­ment that is be­ing tapped for $125 mil­lion right off the bat. The rest will be held in re­serve.

8.75% on di­rect an­nu­al net sales of OR­LADEYO up to $350 mil­lion, 2.75% on sales be­tween $350 mil­lion and $550 mil­lion, no roy­al­ty on sales over $550 mil­lion

Roy­al­ty Phar­ma chief Pablo Legor­re­ta, who took the com­pa­ny pub­lic this year, gets  8.75% on di­rect an­nu­al net sales of Or­ladeyo up to $350 mil­lion, 2.75% on sales be­tween $350 mil­lion and $550 mil­lion, with no roy­al­ties on sales over $550 mil­lion. There’s al­so a 1% roy­al­ty take for one of the biotech’s ex­per­i­men­tal drugs, if it makes it to the mar­ket.

Legor­re­ta of­fered an en­dorse­ment with his mon­ey, call­ing their new­ly ap­proved ther­a­py “trans­for­ma­tive.”

But no mat­ter how much the ex­ec­u­tive crew at BioCryst have in the bank, mar­ket­ing this drug won’t be easy.

BioCryst has an ad­van­tage mar­ket­ing a new oral ther­a­py for HAE, but it comes with marked­ly low­er ef­fi­ca­cy rates, which an­a­lysts be­lieve could well keep pa­tients go­ing back to the cur­rent main­stays in the mar­ket.

An­oth­er lim­it­ing fac­tor is price. The biotech priced their drug at $485,000, which is in line with the mar­ket lead­ers, and way ahead what some an­a­lysts had pro­ject­ed.

BioCryst al­so plans to de­vote a por­tion of the new mon­ey to “ad­vance the de­vel­op­ment of BCX9930 in­to clin­i­cal tri­als in mul­ti­ple com­ple­ment-me­di­at­ed dis­eases.”

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,500+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,500+ biopharma pros reading Endpoints daily — and it's free.

Kaile Zagger, Infinant Health CEO

UC Davis mi­cro­bio­me spin­out re­brands in­fant sup­ple­ment busi­ness with na­ture fo­cus

When Kaile Zagger took the helm of UC Davis spinout Evolve Biosystems several months ago, the company billed itself as a probiotic maker.

However, she believes the company’s Evivo supplement designed to help infants develop a healthy gut microbiome is “so much more” — and that, she said, calls for a rebrand.

Evolve has, well, evolved into Infinant Health, the company announced on Monday. The new name is a mash-up of the words “infant” and “infinite,” representing the company’s goal of expanding beyond infant care. While its sole product, Evivo, is intended for newborns, Infinant is “quickly developing” an option for kids through the age of two.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,500+ biopharma pros reading Endpoints daily — and it's free.

Bob Azelby, Eliem Therapeutics CEO

Eliem says ear­li­er drug ex­po­sure is­sues have been re­solved, drops one epilep­sy in­di­ca­tion

After being forced to delay two Phase IIa trials and blaming CMC issues on a Phase Ib miss, Eliem Therapeutics believes it’s now in the clear.

The Seattle and UK-based biotech put out word Wednesday morning about how it conducted new early-stage studies to confirm why low exposure issues arose during the Phase Ib. After researchers compared the results of the studies, Eliem found “no meaningful difference” between them and ruled out CMC as the reason for the foiled Phase Ib study, the company said in a press release.