Aeter­na Zen­taris shares evis­cer­at­ed by its lat­est late-stage drug dis­as­ter

David Dodd, Aeter­na Zen­taris

When Aeter­na Zen­taris $AEZS planned a move to Charleston, SC, a cou­ple of years ago, the biotech promised to cre­ate a batch of new jobs as it wres­tled to make a come­back fol­low­ing a sting­ing FDA re­jec­tion of its lead drug. This morn­ing, though, the com­pa­ny sim­ply added to its litany of set­backs.

Billed by at least one an­a­lyst as the fu­ture of the com­pa­ny, the biotech an­nounced that its drug Zop­trex failed a Phase III study for ad­vanced en­dome­tri­al can­cer. And now it’s sim­ply wash­ing its hands of the drug and go­ing back to see if they can wres­tle an ap­proval for an old drug that’s al­ready been re­ject­ed by the FDA.

Shares of Aeter­na Zen­taris were evis­cer­at­ed, drop­ping 57% in pre-mar­ket trad­ing.

In­ves­ti­ga­tors said the biotech’s drug looked re­mark­ably like the dox­oru­bicin com­par­i­son arm. Zop­trex scored a 10.9 month over­all sur­vival rate com­pared to 10.8 months for dox­oru­bicin. The sec­ondary end­points were al­so a match.

That re­sult fol­lows a Phase III fail­ure of their lead drug Macrilen in Jan­u­ary, which the FDA re­ject­ed three years ago. Aeter­na Zen­taris said it was con­sid­er­ing what next steps, if any, it would take on the drug af­ter the con­fir­ma­to­ry study end­ed in fail­ure. Then weeks lat­er the com­pa­ny added that the FDA had spelled out the ad­di­tion­al da­ta need­ed that could lead to an ap­proval. And this morn­ing, faced with a sep­a­rate dis­as­ter, they are now talk­ing not on­ly about pur­su­ing the new drug ap­pli­ca­tion, but mar­ket­ing plans as well.

It’s a tough sale, though, as the last bit of cred­i­bil­i­ty at the com­pa­ny takes a bruis­ing hit.

CEO David Dodd had this to say:

Our fo­cus has now shift­ed en­tire­ly to fil­ing our new drug ap­pli­ca­tion for Macrilen and, if the prod­uct is ap­proved, to its com­mer­cial launch as soon as pos­si­ble. We will al­so op­ti­mize our re­sources to be con­sis­tent with our fo­cus on Macrilen-re­lat­ed ef­forts. We con­tin­ue to be­lieve in the po­ten­tial that Macrilen pro­vides for us to be­come a fo­cused spe­cial­ty phar­ma­ceu­ti­cal com­pa­ny. Our in­ten­tion is to sub­mit the Macrilen NDA in the third quar­ter of 2017 and, if the prod­uct re­ceives FDA ap­proval, to com­mer­cial­ly launch the prod­uct in the first quar­ter of 2018.

Eli Lilly CEO David Ricks (Evan Vucci/AP Images)

A P val­ue of 0.38? NE­JM re­sults raise new ques­tions for Eli Lil­ly's vaunt­ed Covid an­ti­body

Generally, a P value of 0.38 means your drug failed and by a fair margin. Depending on the company, the compound and the trial, it might mean the end of the program. It could trigger layoffs.

For Eli Lilly, though, it was part of the key endpoint on a trial that landed them a $1.2 billion deal with the US government to supply up to nearly 1 million Covid-19 antibodies.

So what does one make of that? Was the endpoint not so important, as Lilly maintains? Or did the US government promise a princely sum for a pedestrian drug?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,800+ biopharma pros reading Endpoints daily — and it's free.

Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized Covid-19 patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

CMO Merdad Parsey (Gilead)

Gilead hits the brakes on a tri­fec­ta of mid- and late-stage stud­ies for their trou­bled fil­go­tinib pro­gram. It's up to the FDA now

Gilead $GILD execs haven’t decided exactly what to do with filgotinib in the wake of the slapdown at the FDA on their rheumatoid arthritis application, but they’re taking a time out for a slate of studies until they can gain some clarity from the agency. And without encouraging guidance, this drug could clearly be axed from the pipeline.

In their Q3 report out Wednesday afternoon, the company says researchers have “paused” a Phase III study for psoriatic arthritis along with a pair of Phase II trials for ankylosing spondylitis and uveitis. Late-stage studies for ulcerative colitis and Crohn’s are continuing, but you can see for yourself how big a hole this leaves in the inflammatory disease pipeline, with obvious implications if the company abandons filgo altogether.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,800+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron, GSK R&D chief

GSK's Hal Bar­ron ax­es a once-prized drug from J&J, con­tin­u­ing shift away from res­pi­ra­to­ry

Hal Barron’s revamp of the GlaxoSmithKline pipeline continued yesterday, as the British pharma announced they axed an asthma drug they once promised over $200 million to acquire.

Then led by CEO Andrew Witty and R&D chief Patrick Vallance, GSK picked up the drug, known elegantly as GSK3772847, from J&J in 2016, hoping to expand on the beachhead in asthma they had established the year prior with Breo Ellipta. They promised up to $227 million in upfront payments and milestones.

Ar­cus and As­traZeneca part­ner on a high stakes an­ti-TIG­IT/PD-L1 PhI­II can­cer study, look­ing to im­prove on a stan­dard of care

For AstraZeneca, the PACIFIC trial in Stage III non-small cell lung cancer remains one of the big triumphs for AstraZeneca’s oncology R&D group. It not only made their PD-L1 Imfinzi a franchise player with a solid advance in a large niche of the lung cancer market, the study — which continues to offer data on the long-range efficacy of their drug — also helped salve the vicious sting of the failure of the CTLA-4 combo in the MYSTIC study.

Re­gen­eron posts sec­ond look on Covid-19 an­ti­body cock­tail, boost­ing its case for EUA — but what about symp­tom al­le­vi­a­tion?

Regeneron has revealed a second cut of data on its Covid-19 antibody cocktail in the outpatient setting — data that it has sent straight to the FDA to boost its emergency use authorization request.

The new results reinforce what’s reported from the same trial last month, Regeneron said, incorporating a total of 799 non-hospitalized patients with mild-to-moderate disease. REGN-COV2 reduced viral load and patient medical visits (anything ranging from hospitalizations, emergency room, urgent care visits to physician office and telemedicine visits), meeting all the key endpoints.

Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,800+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis buys a new gene ther­a­py for vi­sion loss, and this is one pre­clin­i­cal ven­ture that did­n't come cheap

Cyrus Mozayeni got excited when he began to explore the academic work of Ehud Isacoff and John G. Flannery at UC Berkeley.

Together, they were engaged in finding a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness, potentially restoring the vision of a broad group of patients. And they did it by using a vector to deliver the genetic sequence for light sensing proteins.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,800+ biopharma pros reading Endpoints daily — and it's free.