Weeks af­ter FDA de­lay, Roche, PTC un­veil an­oth­er batch of pos­i­tive ris­diplam da­ta in se­vere SMA pa­tients

While the FDA has de­cid­ed it needs an­oth­er quar­ter to re­view Roche and PTC Ther­a­peu­tics’ oral SMA ther­a­py, its mak­ers are re­port­ing a steady drum­beat of new pos­i­tive da­ta. On Tues­day, the com­pa­nies un­veiled one-year da­ta in pa­tients with the most se­vere form of the dis­ease from a key tri­al.

The FDA is set to make its de­ci­sion on the ther­a­py, ris­diplam, by Au­gust. It is ex­pect­ed to com­pete with Bio­gen’s Spin­raza and No­var­tis’ Zol­gens­ma.

The da­ta were from Part 2 of the FIRE­FISH tri­al eval­u­at­ing ris­diplam in 41 in­fants aged 1 – 7 months old with symp­to­matic type 1 SMA. The study met the main goal — with 29% of in­fants sit­ting with­out sup­port for five sec­onds by month 12. Typ­i­cal­ly, no in­fants hit this mile­stone in the nat­ur­al his­to­ry of type 1 SMA.

Two piv­otal stud­ies con­sti­tute ris­diplam’s mar­ket­ing ap­pli­ca­tion. FIRE­FISH is an open-la­bel tri­al in in­fants with type 1 SMA, while SUN­FISH is place­bo-con­trolled and re­cruit­ed pa­tients aged 2 to 25 years with types 2 or 3 SMA. Both con­tain two parts: The first por­tion is used to de­ter­mine the op­ti­mal dose and as­sess the safe­ty, while the lat­ter is em­ployed to con­firm ef­fi­ca­cy.

Da­ta from FIRE­FISH Part 2 al­so showed 18 in­fants were able to hold their head up­right, 13 were able to roll to the side and 2 in­fants were able to stand with sup­port. In an ex­plorato­ry end­point, 95% of in­fants who were alive at 12 months (36/38) main­tained the abil­i­ty to swal­low and 89% (34/38) were able to feed oral­ly.

The drug’s safe­ty pro­file was con­sis­tent with pre­vi­ous stud­ies.

“We be­lieve that this ef­fi­ca­cy com­pares fa­vor­ably to the da­ta from Bio­gen’s EN­DEAR study of Spin­raza in Type 1 SMA, which showed that ~84% were alive af­ter 1 year of treat­ment and the event free sur­vival rate was ~55%,” Baird an­a­lyst Bri­an Sko­r­ney wrote in a note.

“While cross-tri­al com­par­isons are al­ways dif­fi­cult, we be­lieve that the strong da­ta Part 2 of the FIRE­FISH study of ris­diplam should lead to rapid up­take of this med­ica­tion once it is ap­proved, which would come at the detri­ment of Spin­raza as pa­tients are like­ly to switch to the more ef­fi­ca­cious med­ica­tion.”

Bio­gen’s Spin­raza, an an­ti­sense oligonu­cleotide, is in­ject­ed in the spine every four months fol­low­ing ini­tial load­ing dos­es. No­var­tis’ 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.

With Roche’s plan to make ris­diplam cheap­er than Spin­raza, the ap­peal of oral ad­min­is­tra­tion could make the drug an even big­ger threat to the Spin­raza fran­chise — which gen­er­at­ed near­ly $2.1 bil­lion last year — com­pared to the world’s most ex­pen­sive ther­a­py, Zol­gens­ma.

“Giv­en the dy­nam­ics sur­round­ing COVID-19, and the an­tic­i­pat­ed shift to­ward telemed­i­cine and min­i­miz­ing in-per­son in­ter­ac­tion with health­care providers, we be­lieve the com­pet­i­tive ad­van­tage of be­ing an oral ther­a­py that can be giv­en at home will be even more pro­nounced and could lead to the rapid up­take of ris­diplam should the med­ica­tion gain ap­proval by the Au­gust 24th PDU­FA,” Sko­r­ney added.

Ear­li­er this month, the FDA said it need­ed an­oth­er three months to re­view ad­di­tion­al da­ta on the drug sub­mit­ted by its mak­ers.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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