Roche dumps its late-stage SMA drug af­ter gam­bling up to $545M-plus on the pro­gram

Af­ter run­ning in­to re­peat­ed set­backs with its ex­per­i­men­tal drug for spinal mus­cu­lar at­ro­phy, Roche has opt­ed to scrap the ther­a­py it grabbed three years ago with a $545 mil­lion buy­out deal.

Sangee­ta Jeth­wa

In a note to a pa­tients group — a fa­vorite av­enue for the Swiss phar­ma gi­ant — a Roche rep­re­sen­ta­tive ex­plained that reg­u­la­tors at the FDA and the EMA were de­mand­ing a new Phase III study of ole­soxime. That fol­lowed is­sues with man­u­fac­tur­ing and their analy­sis of the OLEOS study which “ac­tu­al­ly showed a wors­en­ing in mo­tor func­tion” among pa­tients on the drug.

Roche paid $140 mil­lion up­front for the com­pa­ny and the drug, with more than $400 mil­lion on the ta­ble in mile­stones.

The OLEOS study is now be­ing scrapped and Roche’s Sangee­ta Jeth­wa, head of their pa­tient part­ner­ship for rare dis­eases, had this to say to TreatS­MA:

Un­for­tu­nate­ly, de­spite all of our ef­forts and a strong de­sire to de­liv­er ole­soxime as a med­i­cine to peo­ple with SMA, we have con­clud­ed that this is not go­ing to be pos­si­ble.  Based on all of the avail­able ev­i­dence and the con­tin­ued dif­fi­cul­ties de­scribed above, we have de­cid­ed to stop fur­ther de­vel­op­ment of ole­soxime.

In a state­ment to End­points News, the com­pa­ny added:

Since ac­quir­ing Ole­soxime from Trophos in 2015, we have un­der­tak­en pre­clin­i­cal stud­ies, con­sult­ed with health au­thor­i­ties and ex­perts, re­for­mu­lat­ed the in­ves­ti­ga­tion­al mol­e­cule, per­formed bioavail­abil­i­ty stud­ies to op­ti­mize dose, and at­tempt­ed to op­ti­mize the study de­sign to en­able a phase III tri­al.

It is with re­gret that we now con­clude that we do not have the nec­es­sary body of clin­i­cal and sci­en­tif­ic ev­i­dence to sup­port fur­ther de­vel­op­ment in the new SMA land­scape.

We re­main com­mit­ted to SMA. Our in­ves­ti­ga­tion­al med­i­cine RG7916, an oral SMN2 splic­ing mod­i­fi­er that is sys­tem­i­cal­ly dis­trib­uted through­out the body, is cur­rent­ly be­ing eval­u­at­ed in three mul­ti-cen­tre Phase II /III tri­als, SUN­FISH, FIRE­FISH and JEW­ELFISH.

It couldn’t have come as a com­plete sur­prise. The drug — ac­quired in Roche’s ac­qui­si­tion of Trophos — had failed an ear­li­er mid-stage study, fo­cus­ing on mo­tor func­tion. And even be­fore the phar­ma gi­ant bought the biotech, the drug had failed a tri­al for ALS.

Roche isn’t get­ting to the fin­ish line on SMA, but it’s sound­ing a re­treat at a time of progress in the field. Bio­gen and Io­n­is scored an ap­proval for Spin­raza, and prompt­ly made it one of the most ex­pen­sive ther­a­pies in the world, with a start­ing price of $750,000 for the first year. AveX­is, mean­while, has be­gun clin­i­cal work on a gene ther­a­py for SMA, and oth­ers are al­so jump­ing in.

From left to right: Lilian Kim, Associate Director Business Development; John Moller, CEO; Yooni Kim, Executive Director, Asia Operations; Michelle Park, Director South Korea Operations.

Novotech CRO sees 26% growth in Asia tri­al ac­tiv­i­ty from biotechs, but still plen­ty of ca­pac­i­ty

As the Asia-Pacific clinical trials sector continues to grow rapidly, Novotech the Asia-Pacific-based CRO is seeing biotech clinical activity up by 26%. But says there is still plenty of capacity in the region that features advanced medical facilities, supportive regulatory environments, and more than 2.3 billion people, largely treatment naïve, living in urban areas.

China, South Korea and Australia have the most studies registered as recruiting or about to recruit according to ClinicalTrials.Gov.

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

UP­DAT­ED: Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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A preda­tor's world? Top an­a­lyst sees the 'haves' and the 'haven't­s' di­verge as biotech bub­bles form — and col­lapse

Josh Schimmer

We’ve all seen the deluge of cash that’s been pouring into biotech from every angle: VCs, IPOs and follow-ons have generated billions in capital for new and emerging drug developers with ready access to some powerful new tech. But Evercore ISI’s Josh Schimmer is asking where we’re headed from here.

His answer is neither apocalyptic nor universally blissful, but if he’s right — and this is a discussion we’re hearing much, much more about at a time of growing economic and industry uncertainty — we may well be at a crossroads that could affect valuations, M&A and the entire global industry that has formed over the past 5 years.

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US mulls tar­iffs on Swiss drug ex­ports, weigh­ing on No­var­tis and Roche –  re­port

The leading Swiss newspaper has reported that the US is considering placing tariffs on pharmaceuticals from Switzerland. Roche and Novartis stock each fell 1% after the news broke.

Neue Zürcher Zeitung reported that US Trade Representative Robert Lighthizer told pharmaceutical representatives the Trump administration was considering the move. Tariffs do not appear to be in the immediate offing, but they would potentially affect Swiss giants Novartis and Roche along with other companies that manufacture in Switzerland, including Merck KGaA and US biotech Biogen, which is currently constructing a new facility in the country.

Neil Kumar, Endpoints

Bridge­Bio drops bid to re­claim Ei­dos af­ter di­rec­tors spurn 3 of­fers

A couple of months ago a newly public BridgeBio turned some heads by disclosing that it had made a bid for subsidiary Eidos Therapeutics in hopes of gobbling up the 34% stake that it doesn’t already own. Two offers later, the parties are calling it off.

A special committee of independent directors at the smaller biotech led by RA Capital’s Rajeev Shah and ex-Portola CEO William Lis first rejected the parent company’s initial offer — which would swap 1.3 BridgeBio shares for each Eidos share — on September 12. In the latest announcement, BridgeBio revealed that it eventually raised the offer to 1.5 shares and made $110 million available for all-cash or mixed consideration options, but Eidos still wasn’t interested.

Mark Foley, Revance

HR vi­o­la­tion push­es Re­vance co-founder out, vault­ing for­mer Zel­tiq chief to the helm

Months after Revance amended the terms of its Botox biosimilar collaboration with Mylan, the Newark, California-based drug developer disclosed its co-founder Dan Browne is stepping down, in what appears to be mysterious circumstances.

The company — which is also developing a rival to Allergan’s formidable Botox franchise — on Monday said Browne is departing “due to misjudgment in handling an employee matter,” that has also culminated in his resignation from Revance’s board of directors.

In-house FDA re­view flags a sus­pi­cious im­bal­ance in deaths as Sh­iono­gi hunts an OK for an­tibi­ot­ic

Shionogi has some big questions to answer if they plan to win an FDA panel’s backing for their new antibiotic.

While investigators have provided positive efficacy data for their new product to treat cases of complex urinary tract infections, an FDA review has flagged an imbalance of deaths between the antibiotic and a control arm. And they want the agency’s outside advisers to take a good hard look at that when they meet on Wednesday.

Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

Eye­ing $86M, Galera leads a pack of three mod­est biotech IPOs push­ing past high pro­file stum­bles

Exactly one year after kicking off a pivotal Phase III study for its lead drug — a companion for cancer patients receiving radiotherapy — Galera is looking to the Nasdaq for some new cash to complete the clinical work and fuel its commercial drive.

CEO Mel Sorensen has penciled in an $86 million ask, which was filed on the same day as liver disease company 89bio and rare disease diagnostics shop Centogene. The trio marks the first batch of IPO filings in the wake of two highly anticipated but ultimately disappointing public debuts by BioNTech and Vir, signaling dwindling biotech fervor on Wall Street. 89bio and Centogene are seeking $70 million and $69 million, respectively.