A 7-year-old al­liance be­tween Cel­gene and Ac­celeron is pay­ing off with more promis­ing PhI­II da­ta for lus­pa­ter­cept

Jay Back­strom

Just days af­ter hit­ting its first set of marks in a crit­i­cal­ly im­por­tant Phase III study, Ac­celeron $XL­RN and its part­ners at Cel­gene $CELG say that their prospec­tive block­buster lus­pa­ter­cept has rung all the right bells in a piv­otal study for be­ta tha­lassemia.

Their drug has suc­ceed­ed in the close­ly watched BE­LIEVE study, say in­ves­ti­ga­tors, with a “high­ly sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in the pri­ma­ry end­point of ery­throid re­sponse, which was de­fined as at least a 33% re­duc­tion from base­line in red blood cell (RBC) trans­fu­sion bur­den with a re­duc­tion of at least 2 units dur­ing the pro­to­col-de­fined pe­ri­od of 12 con­sec­u­tive weeks, from week 13 to week 24, com­pared to place­bo.”

The re­searchers added that the drug al­so hit on sta­tis­ti­cal sig­nif­i­cance for all the key sec­ondary end­point, fur­ther build­ing the case they plan to take reg­u­la­tors in the US and Eu­rope.

Ac­celeron’s stock price jumped 11% in af­ter-mar­ket trad­ing.

The drug is de­signed to spur the pro­duc­tion of red blood cells by tar­get­ing trans­form­ing growth fac­tor-be­ta pro­teins. In the first Phase III the biotechs says that the drug al­so sig­nif­i­cant­ly helped pa­tients with myelodys­plas­tic syn­dromes and ane­mia.

The al­liance be­tween Ac­celeron and Cel­gene on this ane­mia drug (ini­tial­ly dubbed ACE-536) dates back 7 years, when Cel­gene paid $25 mil­lion to opt in. Mile­stones amount­ed to a mod­est $217 mil­lion.

The pos­i­tive da­ta have been boost­ing Cel­gene at a time the com­pa­ny has strug­gled to over­come a se­ries of set­backs on their top pipeline prospects. Leerink’s Ge­of­frey Porges has been leery about the lack of de­tails, hold­ing back any glee un­til he sees the ac­tu­al da­ta — per­haps at ASH. Still, he was will­ing to give the part­ners a high per­cent­age chance of win­ning an ap­proval and set sales at 2023 at a like­ly $1.5 bil­lion on the MEDAL­IST suc­cess. The an­a­lyst put out a new note giv­ing the part­ners a thumbs up on the sec­ond Phase III win, not­ing some big gains for Cel­gene’s bat­tered stock price in the wake of their suc­cess here.

Af­ter the pos­i­tive re­sult for MEDAL­IST shares of Ac­celeron have gained ~40% and we be­lieve an­oth­er 5-10% boost could come now that the sec­ond phase III is clear­ly pos­i­tive. Cel­gene’s shares have gained ~9% since the MEDAL­IST dis­clo­sure and we be­lieve this sec­ond pos­i­tive set of news could fur­ther im­prove sen­ti­ment in this stock ahead of the Q2 earn­ings re­lease lat­er this month.

“For decades, the man­age­ment of be­ta-tha­lassemia in adults has been lim­it­ed to trans­fu­sions and iron chela­tion. Re­duc­tion of trans­fu­sion bur­den rep­re­sents an im­por­tant step for­ward for pa­tients with this rare and de­bil­i­tat­ing blood dis­ease,” said Jay Back­strom, chief med­ical of­fi­cer for Cel­gene.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug -- but holds da­ta in re­serve

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.

UP­DAT­ED: Ab­b­Vie do­nates $1M+ of the HIV drug that Chi­na is now rec­om­mend­ing for coro­n­avirus treat­ment

AbbVie is donating more than $1 million worth of an HIV drug to help combat the fast-spreading coronavirus outbreak in China, the company announced on Friday.

China’s National Health Commission has suggested Aluvia, a pill containing lopinavir and ritonavir, as one of two possible treatments for the symptoms of the virus currently known as 2019-nCoV in the absence of effective antiviral medications. The other part is nebulized alpha-interferon.