UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a block­buster rare dis­ease gi­ant in its sights in Alex­ion’s Soliris, small biotech Apel­lis has rea­son to think its com­peti­tor is wor­thy of the spot­light. Now, with the FDA on its side, Apel­lis will get its chance to be the David to Alex­ion’s Go­liath.

The FDA on Fri­day ap­proved Em­paveli (pegc­eta­coplan), a C3 com­ple­ment in­hibitor the biotech thinks can prove a wor­thy chal­lenger to Alex­ion’s C5 in­hibitors Soliris and fol­low-up drug Ul­tomiris in rare dis­ease parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH).

In piv­otal Phase III da­ta on which the agency based its re­view, Em­paveli best­ed Soliris in a head-to-head test in terms of he­mo­glo­bin pro­duc­tion with a rel­a­tive in­crease of 3.9 g/dL (p<0.0001), and some fa­vor­able trend lines in sec­ondary end­points such as trans­fu­sions and fa­tigue.

Em­paveli will come with the same black box warn­ing la­bel as Soliris, re­strict­ing use un­der a risk eval­u­a­tion and mit­i­ga­tion strat­e­gy for meningo­coc­cal in­fec­tions. The ther­a­py was ap­proved for pa­tients re­gard­less of pri­or ther­a­pies, mean­ing its use won’t be re­strict­ed to for­mer C5 pa­tients alone.

On a call with an­a­lysts Mon­day, Apel­lis said Em­paveli would come with an av­er­age an­nu­al whole­sale ac­qui­si­tion cost of $458,000. That’s “at par­i­ty” with Ul­tomiris and a dis­count on Soliris, the com­pa­ny said.

Apel­lis thinks that a big bump in he­mo­glo­bin lev­els could help it im­me­di­ate­ly cap­ture about a third of the ex­ist­ing PNH pa­tients who have a low and falling he­mo­glo­bin count that is cur­rent­ly un­der­served by the C5 drugs, CEO Cedric Fran­cois told End­points News pri­or to the ap­proval.

“If the Phase III study did one thing it is mak­ing it very clear that by con­trol­ling ex­travas­cu­lar he­mol­y­sis, you can el­e­vate the stan­dard of care in these pa­tients,” Fran­cois said.

The key to Apel­lis’ pitch is C3, which it be­lieves can of­fer a longer-last­ing so­lu­tion for he­mo­glo­bin pro­duc­tion as C5 of­ten doesn’t boost lev­els for more than a few days, the team said. With an ap­proval in hand, Apel­lis said it plans to tar­get those 500 or so US pa­tients with the low­est he­mo­glo­bin counts but thinks Em­paveli could have a mean­ing­ful up­take across the en­tire PNH spec­trum.

The com­pa­ny has al­ready built up a sales team that has been do­ing some of the ground­work on physi­cian out­reach in the lead-up to ap­proval. It’s a small group of PNH spe­cial­ists, and Apel­lis thinks it’s found a work­able, hy­brid dig­i­tal/in-per­son ed­u­ca­tion cam­paign to tell doc­tors about the drug’s ben­e­fits.

But beat­ing Alex­ion — and soon to be As­traZeneca — won’t be an easy task by any means. Alex­ion’s en­tire fran­chise was built around build­ing Soliris in­to a block­buster, and the com­pa­ny has been busy amid a gung-ho switch­ing cam­paign to fol­low-up drug Ul­tomiris.

As­traZeneca an­nounced its in­tent to buy Alex­ion late last year for a cool $39 bil­lion. The rare dis­ease spe­cial­ist will im­port its ex­pan­sive port­fo­lio of C5 drugs, which sport in­di­ca­tions be­yond PNH.

This sto­ry will be up­dat­ed.

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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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.

Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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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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Mark Foley, Revance CEO (Revance via Vimeo)

UP­DAT­ED: FDA re­jects Re­vance's pitch for Botox com­peti­tor as biotech blames man­u­fac­tur­ing is­sues

The FDA has told Revance Therapeutics that, no, it won’t be able to turn those frowns upside down.

Regulators issued a CRL to the biotech for its botox-based therapy for moderate to severe glabellar lines, also known as frown lines, Revance announced Friday. As is agency policy, the FDA does not comment on these types of communications, but Revance said the rejection came from deficiencies at a manufacturing site.

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