No­var­tis of­floads a late-stage PI3Kẟ rare dis­ease pro­gram in a dis­count deal

Look­ing to build on their fran­chise HAE drug Ru­con­est, Dutch biotech Pharm­ing has snapped up a late-stage rare dis­ease drug from No­var­tis for a mod­est up­front of on­ly $20 mil­lion.

The phar­ma gi­ant is sell­ing glob­al rights to CDZ173, a small mol­e­cule phos­pho­inosi­tide 3-ki­nase delta (PI3Kẟ) in­hibitor in de­vel­op­ment for Ac­ti­vat­ed Phos­pho­inosi­tide 3-ki­nase Delta Syn­drome —or APDS. And No­var­tis will com­plete the Phase II/III reg­is­tra­tion study as well as an on­go­ing open-la­bel ex­pan­sion study for Pharm­ing.

In this dis­ease, a mu­ta­tion in the PIK3CD gene spurs over-ac­ti­va­tion of PI3Kẟ, lead­ing to a dys­func­tion­al im­mune sys­tem, le­sions as well as can­cer. GSK has one in Phase II for APDS, dubbed GSK2269557, though it re­cent­ly culled a COPD tri­al us­ing that drug as the com­pa­ny sliced back on the res­pi­ra­to­ry pipeline.

Pharm­ing sees the drug as a nice ad­di­tion to their HAE ther­a­py, ap­proved 5 years ago, as APDS is gen­er­al­ly han­dled by the same set of physi­cians, eas­ing com­mer­cial­iza­tion work.

It’s a rel­a­tive­ly small amount to pay for a late-stage drug, in­di­cat­ing that the com­mer­cial up­side here may not be huge. PI3Kẟ in­hi­bi­tion cer­tain­ly isn’t new, with Gilead out with Zy­delig and oth­ers like du­velis­ib join­ing the mix for on­col­o­gy, with lim­it­ed com­mer­cial suc­cess. No­var­tis was un­usu­al in ad­vanc­ing the drug for non-can­cer us­es.

So­cial im­age: No­var­tis, AP Im­ages

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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David Liu, Liu Group

David Liu un­veils newest ad­vance­ment in CRISPR tech: Prime edit­ing

The researcher behind base-editing is out with what some scientists are hailing as the biggest advancement in CRISPR technology since that 2016 breakthrough: “prime editing.” The new molecular gadget is capable of erasing any base pair and stenciling in another and cutting or adding long segments of DNA without breaking both strands of the helix.

David Liu, base editing pioneer and founder of Beam Therapeutics, published the findings in Nature alongside Andrew Anzalone. They estimated that the breakthrough “in principle” puts 89% of human diseases in purview — although experts cautioned that human therapies were a long way off.

Bhaskar Chaudhuri. Frazier Healthcare Partners

Fra­zier Health­care Part­ner­s' der­ma­tol­ogy up­start at­tracts a mar­quee syn­di­cate, $94M+ for 'in-be­tween' top­i­cal drug

For the past three years Frazier Healthcare Partners’ Bhaskar Chaudhuri has been carefully and quietly grooming Arcutis Therapeutics, a new dermatology play he co-founded to deliver topical formulations of well-known drugs. Now that the biotech is poised to enter Phase III, he’s being joined by a marquee syndicate for its $94.5 million Series C.

HBM Healthcare Investments, Vivo Capital, BlackRock, Omega Funds, Pivotal BioVentures, and Goldman Sachs jumped on board, joining Bain Capital Life Sciences, OrbiMed and RA Capital Management in backing Arcutis’ lead topical cream for plaque psoriasis.

A new com­pa­ny en­ters the Tec­fidera fight, of­fer­ing to kill two birds

The remedy for the most common side effect for one of the most common multiple sclerosis drugs is simple: aspirin.

Taking aspirin with Biogen’s Tecfidera will reduce the flush, a sometimes painful form of red skin irritation, many patients experiences. The problem is that the aspirin has to be taken at least 30 minutes before Tecfidera, turning a simple twice-a-day, one-dose oral drug into a staggered two-drug regimen.

Vas Narasimhan. Getty Images

Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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UP­DAT­ED: Bris­tol-My­ers makes Op­di­vo pitch for front­line lung can­cer with open la­bel PhI­II study

Despite a head start, when Bristol-Myers Squibb and its pioneering checkpoint inhibitor Opdivo suffered a key lung cancer setback in 2016, they found themselves relegated to the backseat as Merck’s Keytruda seized the wheel on the road to immunotherapy stardom. Bristol-Myers has since suffered blow after blow in its quest to take a big slice of the lucrative market, peppered with some small successes. On Tuesday, the New Jersey drugmaker touted positive data from a Phase III open-label study in a bid to carve itself a piece of the frontline lung cancer market.

UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

The FDA has approved Vertex’s new triplet for cystic fibrosis at a record-setting speed.

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Take­da tees up $420M deal for celi­ac an­ti­dote, con­tin­u­ing R&D re­fo­cus

Sometime in the 1st century AD, a patient presented to Arataeus looking like a varicose ghost. He was “emaciated and atrophied, pale, feeble and incapable of performing any of his accustomed works,” the Greek physician wrote, with hollow temples and huge veins running all over his body.

A dysfunctional digestive system, Arataeus concluded – an imbalance he attributed to a “heat” deficiency in a system he and other Greeks regarded as functioning similarly to an oven – and coined a term: coeliac disease, after the Greek word for abdomen.

CD47 play­er Tril­li­um chops dis­cov­ery ef­forts and 40% of staff; Brii Bio inks deal to bring an­tibi­otics to Chi­na

→ One month into his tenure at Canadian microcap biotech Trillium, Jan Skvarka is bringing out the ax as he sorts out the development plans for its CD47 drugs. The restructuring will see the discovery research unit nixed and the headcount will be reduced by 40% (from 43 to 26), reducing the burn rate from CDN$10 million to CDN$4-7 million per quarter. Meanwhile, the company will seek to partner out its preclinical STING agonist program, which it likely doesn’t have enough resources to tend to.