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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Jeffrey Nau, Oyster Point Pharma CEO

FDA OKs an in­haled ver­sion of smok­ing ces­sa­tion drug Chan­tix — for a com­mon eye dis­ease

Oyster Point Pharma now has its first FDA-approved product — Tyrvaya. And the biotech has taken a unique route to get there by using an old drug with a storied past.

The New Jersey biotech announced this morning that the FDA has approved their nasal spray product for dry eye disease on Friday — the first nasal spray to be approved for the disease. The product’s active ingredient is 0.03 mg of varenicline, also known as smoking cessation aid Chantix.

Omeros plunges deep­er af­ter FDA re­jects rare dis­ease drug, ask­ing for more in­for­ma­tion

Omeros practically warned investors that a complete response letter was coming when it disclosed that the FDA found deficiencies in its BLA for narsoplimab. But the agency did not elaborate on the specifics of those deficiencies for the drug, which was being positioned as a treatment for the rare but serious blood clotting disease known as hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

UP­DAT­ED: Third time's the charm: Adamis nabs ap­proval of high-dose nalox­one in­jec­tion af­ter two CRLs

If at first you don’t succeed, and at second you don’t succeed, try, try again.

That’s been the unofficial mantra for Adamis Pharmaceuticals’ high dose naloxone injection, which received two CRLs in the span of a year between the Novembers of 2019 and 2020. But on Monday, word came through that the FDA approved the drug on Adamis’ third attempt, giving doctors another tool to treat individuals who overdose on opioids.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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