Medicxi bankrolls the launch of a pair of biotech twins fo­cused on orex­ins for nar­colep­sy and more

The ven­ture in­vestors at Medicxi are fund­ing a pair of biotech spin­outs from UK-based So­sei Hep­tares, which has been fine tun­ing its ex­per­tise in G pro­tein-cou­pled re­cep­tors for a num­ber of years now.

Francesco De Ru­ber­tis

Medicxi is of­fer­ing €40 mil­lion to kick­start two new com­pa­nies — the twins Orex­ia and In­ex­ia, which will tar­get Orex­in OX1 and OX2 for neu­ro­log­i­cal dis­eases. The Hep­tares sub­sidiary will put its re­searchers to work on the two projects, iden­ti­fy­ing nar­colep­sy as one key dis­ease on the menu.

Orex­ins are well known tar­gets in the biotech world. These neu­ropep­tides are in­volved in the reg­u­la­tion of sleep, wake­ful­ness and ap­petite.

Mal­colm Weir

Medicxi part­ner Francesco De Ru­ber­tis will sit on the board to watch over the firm’s in­vest­ment. Both of these groups have deep roots in the UK, with So­sei look­ing to build up the Hep­tares op­er­a­tion un­der Mal­colm Weir.

The VC’s cash should be enough to get through proof-of-con­cept stud­ies, fit­ting neat­ly in Medicxi’s as­set-cen­tric fo­cus, which of­ten fo­cus­es the mon­ey on ear­ly-stage re­search pro­grams stripped of as much over­head as they can.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

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