Kim Stratton, Orphazyme

Near­ly two years af­ter their nar­row PhI­II fail­ure, Or­p­hazyme gets speedy look at the FDA

Or­p­hazyme’s bet that, de­spite miss­ing both pri­ma­ry end­points on its piv­otal study, they would find wel­com­ing ears at the FDA has paid off, for now.

The Dan­ish biotech an­nounced to­day that the FDA has ac­cept­ed the ap­pli­ca­tion for their ex­per­i­men­tal drug ari­mo­clo­mol in the rare neu­rode­gen­er­a­tive con­di­tion Nie­mann-Pick dis­ease Type C and giv­en it pri­or­i­ty re­view. Al­though drugs that fail piv­otal stud­ies rarely re­ceive a pri­or­i­ty look at the agency, the dis­tinc­tion should come with lit­tle sur­prise. Or­p­hazyme has sig­naled re­peat­ed­ly since the 2019 re­sults came out that the FDA viewed an ap­pli­ca­tion fa­vor­ably and, the FDA gave it break­through des­ig­na­tion that No­vem­ber.

The agency set a PDU­FA date for March 17, 2021. Or­p­hazyme’s stock jumped 8.38% on the news, from 83.5 kro­nes to 90.5 kro­nes ($13.3 to $14.3).

“We look for­ward to col­lab­o­rat­ing with the FDA as they com­plete their re­view of this NDA to ad­dress the un­met med­ical need in NPC, and mean­while are work­ing to ex­pand our U.S. ac­tiv­i­ties in prepa­ra­tion for po­ten­tial com­mer­cial avail­abil­i­ty next year,” CEO Kim Strat­ton said in a state­ment.

Or­p­hazyme, a one-drug com­pa­ny tar­get­ing a string of neu­rode­gen­er­a­tive dis­or­ders, came out it with re­sults from a 50-per­son Phase II/III study on Nie­mann-Pick type C pa­tients, their lead in­di­ca­tion, in Jan­u­ary 2019. Ari­mo­clo­mol failed to hit both pri­ma­ry end­points, both a stan­dard scale for as­sess­ing symp­toms in pa­tients, called the NPC Clin­i­cal Sever­i­ty Scale, and the Clin­i­cal Glob­al Im­pres­sion of Im­prove­ment, a self-rat­ing tool that the FDA re­quest­ed. It al­so failed sev­er­al sec­ondary end­points.

Still, the drug on­ly nar­row­ly missed sig­nif­i­cance on the first pri­ma­ry end­point, where they saw both a 74% re­duc­tion in dis­ease sever­i­ty and a p-val­ue of 0.0506. And the com­pa­ny not­ed that two pre-spec­i­fied sub­group analy­ses reached sig­nif­i­cance, with dis­ease pro­gres­sion slow­ing in pa­tients over the age of 4 and in pa­tients who had been tak­ing an Acte­lion drug some­times pre­scribed for the dis­ease. All end­points were against place­bo.

In a de­gen­er­a­tive con­di­tion with no sig­nif­i­cant treat­ments, Or­p­hazyme was will­ing to bet that was enough to take to reg­u­la­tors. So far, they’ve been proven right.

Or­p­hazyme, eye­ing funds for com­mer­cial­iza­tion, filed for its first US of­fer­ing ear­li­er this month, pen­cil­ing in an ini­tial fig­ure of $115 mil­lion for the raise. In ad­di­tion to their lead in­di­ca­tion, they al­so have piv­otal stud­ies on­go­ing for ALS and spo­radic in­clu­sion body myosi­tis, a pro­gres­sive mus­cle dis­or­der.

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.

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.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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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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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, 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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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.

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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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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