Ne­glect­ed on Wall Street, lit­tle Stealth Bio finds an af­flu­ent chum with $30M in cash and high hopes for their PhI­II drug

Stealth Bio­Ther­a­peu­tics nev­er had much luck at gain­ing the at­ten­tion of Wall Street $MI­TO while strug­gling to ad­vance its late-stage drug for mi­to­chon­dr­i­al dis­ease. But it turns out Alex­ion was watch­ing close­ly.

Thurs­day morn­ing the big­ger rare dis­ease biotech agreed to front $30 mil­lion —half for stock — to pick out a ring­side seat as Stealth makes its way through the fi­nal stages of a Phase III with elamipre­tide. The drug’s been tout­ed as a po­ten­tial first-in-class ther­a­py, but in­vestors nev­er bought in, watch­ing Stealth’s shares drift steadi­ly low­er af­ter pric­ing the IPO at $12.

The deal gives Alex­ion an op­tion on rights to “co-de­vel­op sub­cu­ta­neous elamipre­tide in the US for PMM (ge­net­i­cal­ly de­fined pri­ma­ry mi­to­chon­dr­i­al my­opa­thy) and Barth syn­drome, as well as Leber’s hered­i­tary op­tic neu­ropa­thy, which is cur­rent­ly in ear­li­er stage clin­i­cal de­vel­op­ment.” Alex­ion would be free to take the drug glob­al on its own.

We’re not get­ting a read on the rest of the terms, which are ex­clud­ed in their state­ment as well as SEC fil­ings. But Stealth CEO Ree­nie Mc­Carthy says that the deal of­fers a shot at “syn­er­gies of ex­e­cu­tion that will both ex­pe­dite and in­crease pa­tient ac­cess fol­low­ing achieve­ment of key up­com­ing clin­i­cal and reg­u­la­to­ry mile­stones for our PMM and Barth syn­drome pro­grams.”

The drug is a small pep­tide that binds to car­di­olipin, de­scribed in the F-1 (Stealth op­er­ates in New­ton, MA but is based in the Cay­mans) as “an es­sen­tial struc­tur­al el­e­ment of mi­to­chon­dria.” And the mi­to­chon­dria are the pow­er­hous­es of hu­man cells, es­sen­tial to good health. The bind­ing process, they say, sta­bi­lizes the struc­ture of mi­to­chon­dria while ex­pe­ri­enc­ing ox­ida­tive stress.

Stealth shares jumped more than 15% on Thurs­day morn­ing, re­flect­ing some mod­est en­thu­si­asm for the deal.

“Mi­to­chon­dria play a crit­i­cal role in nor­mal or­gan func­tion and, when dys­func­tion­al, can have dev­as­tat­ing con­se­quences on mul­ti­ple or­gan sys­tems, lead­ing to many se­ri­ous dis­eases, such as pri­ma­ry mi­to­chon­dr­i­al my­opa­thy,” said John Orloff, the R&D chief at Alex­ion. “Giv­en our strong ex­ist­ing re­la­tion­ships with neu­ro­mus­cu­lar spe­cial­ists — who play a crit­i­cal role in treat­ing PMM — we be­lieve this is an ex­cit­ing po­ten­tial op­por­tu­ni­ty to fur­ther ex­pand our rare neu­rol­o­gy port­fo­lio and look for­ward to the pos­si­bil­i­ty of work­ing with Stealth to re­al­ize the promise of elamipre­tide for pa­tients.”

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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Vas Narasimhan. Getty Images

UP­DAT­ED: Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and briefly 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: 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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IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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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.

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.