Roche/PTC edge clos­er to dis­rupt­ing mar­ket for SMA ri­vals Bio­gen and No­var­tis with more pos­i­tive da­ta

Roche and part­ner PTC Ther­a­peu­tics have bro­ken out de­tailed pos­i­tive da­ta back­ing their spinal mus­cu­lar at­ro­phy ther­a­py, which is ex­pect­ed to have a good shot at eclips­ing Spin­raza’s mar­ket lead­er­ship.

The FDA is set to make its de­ci­sion on the ther­a­py, ris­diplam, by May 24. If ap­proved — the drug is ex­pect­ed to gen­er­ate be­tween $2 bil­lion and $3 bil­lion in peak sales — and com­pete with Bio­gen’s first-to-mar­ket Spin­raza and No­var­tis’ most-ex­pen­sive-drug-in-the-world Zol­gens­ma.

On Thurs­day, the com­pa­nies re­port­ed de­tailed da­ta from the SUN­FISH tri­al, which test­ed ris­diplam against a place­bo in 180 pa­tients aged 2-25 years with type 2 and 3 SMA, a rare ge­net­ic mus­cle-wast­ing dis­ease. Most pa­tients in the tri­al were old­er, and a rel­a­tive­ly more se­vere form of the dis­ease. The main goal of the study — which was met — was the change in mo­tor func­tion af­ter one year. Roche ini­tial­ly an­nounced the pri­ma­ry end­point had been met last No­vem­ber.

The to­tal mean change in pa­tients re­ceiv­ing ris­diplam was sig­nif­i­cant­ly high­er than place­bo (1.55 point mean dif­fer­ence; p=0.0156). As ex­pect­ed, the strongest re­spons­es ver­sus place­bo were ob­served in the youngest pa­tients aged be­tween 2 and 5 years (78.1% ver­sus 52.9% achiev­ing ≥3 point in­crease).

It is hard to com­pute the clin­i­cal mean­ing­ful­ness of ris­diplam based on ab­solute 1.55 mean point re­duc­tion, even though sta­tis­ti­cal sig­nif­i­cance was hit, Sun­Trust Robin­son Humphrey an­a­lyst Robyn Kar­nauskas wrote in a note.

PTC man­age­ment has pre­vi­ous­ly sug­gest­ed that a 2-point dif­fer­ence would be clin­i­cal­ly mean­ing­ful us­ing the Mo­tor Func­tion Mea­sure scale (MFM-32) scale, but since the mean point dif­fer­ence was 1.55, clin­i­cal mean­ing­ful­ness may not be so ev­i­dent at first blush, she said.

These re­sults sup­ple­ment pos­i­tive da­ta from 41 pa­tient-FIRE­FISH study, which test­ed ris­diplam in pa­tients aged 1-7 months with type 1 SMA.

Ris­diplam is strate­gi­cal­ly po­si­tioned to have strong up­take in the type 2/3 SMA pop­u­la­tion, Cred­it Su­isse’s Mar­tin Auster wrote in a re­cent note. “While we ex­pect Zol­gens­ma to cap­ture the vast ma­jor­i­ty of the in­ci­dence Type 1/2 SMA pop­u­la­tion, we an­tic­i­pate ris­diplam will emerge as the dom­i­nant ther­a­py for old­er SMA pa­tients.”

Bio­gen se­cured the first-ever SMA ap­proval and has since been dosed in over 9,300 pa­tients. How­ev­er, com­par­ing Bio­gen’s da­ta to the Roche/PTC re­sults in SMA type 2/3 pa­tients is dif­fi­cult giv­en the pri­ma­ry end­points are dif­fer­ent in the two tri­als, in ad­di­tion to dif­fer­ent en­roll­ment cri­te­ria.

“While the im­pact of No­var­tis’ Zol­gens­ma has been rel­a­tive­ly mut­ed, we think that the like­ly ap­proval and launch of ris­diplam could im­pact Bio­gen’s Spin­raza fran­chise,” Cred­it Su­isse’s Evan Seiger­man wrote in a re­cent note. “We al­so think that Roche could price ris­diplam be­low Spin­raza to help en­cour­age use – es­pe­cial­ly in T2 or T3 pa­tients.”

Spin­raza, an an­ti­sense oligonu­cleotide, is in­ject­ed in the spine every four months fol­low­ing ini­tial load­ing dos­es. Zol­gens­ma, a gene-ther­a­py, is de­signed to be a one-shot cure, while ris­diplam is a dai­ly oral treat­ment, en­gi­neered to work by tweak­ing how the SMN2 gene is spliced, which rais­es func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues.

SMA is rare, af­fect­ing 1 per 8,000 to 10,000 peo­ple glob­al­ly, but rep­re­sents a lu­cra­tive bat­tle­ground for these drug­mak­ers. Spin­raza, launched in late 2016, car­ries a list price of $750,000 for the first year and $375,000 an­nu­al­ly there­after. Zol­gens­ma — on­ly ap­proved for pa­tients un­der the age of 2 — caused stick­er shock with its $2.1 mil­lion price tag and the in­evitable push­back from pay­ers, al­though No­var­tis has em­pha­sized that its five-year in­stall­ment plan and cu­ra­tive po­ten­tial makes it worth it.

“While there are al­ready 2 ef­fi­ca­cious prod­ucts ap­proved for SMA in the US, (Roche) man­age­ment…see a very large op­por­tu­ni­ty giv­en the con­ve­nient oral for­mu­la­tion of ris­diplam, num­ber of pa­tients not el­i­gi­ble for gene ther­a­py, and small up­front price com­pared to gene ther­a­py and oligonu­cleotide com­peti­tors,” SVB Leerink an­a­lysts wrote in a re­cent note.

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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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 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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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.

GSK touts topline win for PD-1 in head-to-head with Keytru­da — while steer­ing next big check­point drug in­to PhI­II

GSK is claiming a win for what it calls the largest head-to-head trial pitting a PD-1 against Merck’s best-selling Keytruda in a type of lung cancer, as its Jemperli met the primary endpoint of objective response rate.

In a separate positive move, GSK says it’s moving both arms of the COSTAR Lung trial into Phase III to test Jemperli as well as the TIM-3 inhibitor cobolimab.

Hesham Abdullah, GSK’s global head of oncology development, said in a statement that the two trials “support the ambition for dostarlimab to become the backbone of our ongoing immuno-oncology-based research and development programme when used alone and in combination with standard of care and future novel cancer therapies, particularly in patients with currently limited treatment options.”

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