Ear­ly-stage can­cer biotech nails $85M C round; Flem­ming Orn­skov's Gal­der­ma scores 'break­through' sta­tus

Zen­tal­is Phar­ma­ceu­ti­cals just nabbed an $85 mil­lion round from a syn­di­cate that in­cludes Ma­trix Cap­i­tal, Viking Glob­al In­vestors, Red­mile Group, Far­al­lon Cap­i­tal, Per­cep­tive Ad­vi­sors, Sur­vey­or Cap­i­tal and Even­tide As­set Man­age­ment. Their lead drug is ZN-c5, which is cur­rent­ly in Phase I/II tri­als. The biotech de­scribes that drug as a “po­ten­tial best-in-class oral Se­lec­tive Es­tro­gen Re­cep­tor De­grad­er for es­tro­gen re­cep­tor-pos­i­tive, HER2-neg­a­tive (ER+/ HER2-) breast can­cer.”

Zen­tal­is scored a part­ner­ship with Pfiz­er last year that match­es their CDK 4/6 Ibrance with their ear­ly-stage drug. The C round brings Zen­tal­is’s to­tal take to $147 mil­lion.

→ Der­ma­tol­ogy com­pa­ny Gal­der­ma, which months ago se­cured the ser­vices of for­mer Shire chief Flem­ming Ørn­skov as CEO, on Mon­day scored the FDA’s break­through sta­tus for nemolizum­ab, a ther­a­py un­der de­vel­op­ment to treat pru­ri­tus as­so­ci­at­ed with pruri­go nodu­laris, a rare chron­ic skin con­di­tion char­ac­ter­ized by thick skin nod­ules cov­er­ing large body ar­eas. 

→ Months af­ter scor­ing $56 mil­lion in fresh fund­ing, UK-based Healx is shar­ing its AI-based drug dis­cov­ery plat­form with Boehringer In­gel­heim, to help the Ger­man drug­mak­er ex­plore new in­di­ca­tions for the ex­ist­ing as­sets in its ar­se­nal.

→ Ger­many’s Al­giax Phar­ma­ceu­ti­cals has gar­nered a €5.5 mil­lion round. The biotech is fo­cused on the de­vel­op­ment of GA­BA(a) re­cep­tor mod­u­la­tors for neu­ro­path­ic pain. Oc­ci­dent and an undis­closed Ger­man fam­i­ly of­fice led this round, which will be used to fu­el a Phase IIa study named “CURE“ for its lead com­pound AP-325.

→ Life sci­ences ven­ture firm Ep­i­darex Cap­i­tal is launch­ing a new biotech fund called Ep­i­darex Ex­eed. WSJ Pro re­port­ed in March that Ep­i­darex Cap­i­tal had raised over $100 mil­lion for a new fund fo­cused on ear­ly-stage as­sets. In their re­lease to­day Ep­i­darex called the new ven­ture “a ther­a­peu­tic dis­cov­ery en­gine” and said it would ap­ply seed fund­ing and man­age­ment to “trans­form break­through life sci­ence in­no­va­tions in­to in­vestable as­sets over a pe­ri­od of 12-18 months.”

On­cono­va Ther­a­peu­tics — which picked up a few mil­lion in cash to fund the Phase III pro­gram for its lead drug, rigosert­ib, back in Mayhas col­lab­o­rat­ed with In­cep­tua Med­i­cines Ac­cess, a sub­sidiary of Icep­tua Group, to make avail­able in­tra­venous rigosert­ib via a pre-ap­proval ac­cess pro­gram in se­lect­ed coun­tries across the world. A pre-ap­proval ac­cess pro­gram al­lows for ex­per­i­men­tal agents in de­vel­op­ment to be made avail­able up­on re­quest of a physi­cian or a pa­tient for ap­pro­pri­ate pa­tients for whom no al­ter­na­tive treat­ment op­tion ex­ists in their coun­try.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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