With the ink on their $25M Astellas deal still drying, Aquinox shares get blasted on PhIII failure

June 27, 2018 07:28 AM EDT

John Carroll

With the ink on their $25M Astellas deal still drying, Aquinox shares get blasted on PhIII failure

Just weeks after Astellas agreed to pay Aquinox Pharmaceuticals $25 million upfront for Asian rights to their lead drug rosiptor, the little biotech says their Phase III trial was a complete disaster, leaving nothing to salvage.

David Main

CEO David Main didn’t sugarcoat it. No matter how hard they studied the data, the interstitial cystitis drug clearly couldn’t beat a placebo. He said:

We have conducted a number of sensitivity, subpopulation, and secondary endpoint analyses and none demonstrate a benefit of rosiptor over placebo.

Investors betting on a key success here clearly weren’t happy. Aquinox’s shares $AQXP were swiftly decimated, diving 85%.

The Vancouver-based biotech had hoped for better, recruiting 433 patients suffering from interstitial cystitis/bladder pain syndrome for the study, which had the same design as Phase II.

After burying the drug program, and watching its market cap shrivel, Main says it’s time to study what kind of strategic alternatives remains open. There is a mid-stage study underway for the same drug — a small molecule activator of SHIP1 — in chronic prostatitis/chronic pelvic pain syndrome. But virtually all of its eggs were in the one drug basket.

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Lessons for biotech and pharma from a doctor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

James Collins, Broad Institute via Youtube

February 21, 2020 11:48 AM ESTUpdated 12:53 PM

Natalie Grover

AI

R&D

UPDATED: A space odyssey for new antibiotics: MIT's machine learning approach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it harbors the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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February 21, 2020 10:51 AM EST

Jason Mast

R&D

Gilead loses two more patent challenges on HIV pill, setting up courtroom fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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ENDPOINTS CAREERS

Director/Senior Director, Business Development

Respivant Sciences

San Diego, CA

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Jim Scholefield via PR Newswire

February 20, 2020 08:57 AM ESTUpdated 09:47 AM

John Carroll

People

R&D

Merck loses its chief digital officer, spotlighting talent hunt for the hottest title in Big Pharma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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Kathy High (file photo)

February 21, 2020 06:54 AM EST

Amber Tong

Cell/Gene Tx

People

Gene therapy pioneer Kathy High has left Spark after completing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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February 21, 2020 10:30 AM EST

Amber Tong

Deals

Bankrupt antibiotics maker Aradigm turns to old partner/investor for final $3M fire sale

Grifols once paid Aradigm $26 million for a stake in its inhaled antibiotics. But with Aradigm now in bankruptcy, the Spanish drugmaker is dishing out a final $3.2 million to buy it all.

The fire sale — which comes one year after Aradigm filed for Chapter 11 following a regulatory trifecta for disaster — will see Grifols obtain assets and IP to Apulmiq (formerly Pulmaquin and Linhaliq in Europe), Lipoquin and free ciprofloxacin. In addition to waiving its claims in the bankruptcy case, Grifols also agreed to milestone payments up to $3 million more upon any regulatory approvals.

Tal Zaks (Moderna via YouTube)

February 19, 2020 10:01 AM ESTUpdated February 20, 05:17 AM

Jason Mast

R&D

For two decades, a new vaccine technology has been slowly approaching prime time. Now, can it stop a pandemic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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ENDPOINTS CAREERS

Clinical Trial Manager

Cold Genesys, Inc.

Irvine, CA

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February 21, 2020 10:09 AM EST

Jason Mast

R&D

DBV's peanut prevention patch approaches key stage of approval process

Almost a year and a half after DBV Technologies pulled its peanut allergy immunotherapy patch from FDA review, the biotech will get their day in court. The FDA has scheduled an advisory committee hearing for May 15.

In the two-horse race to develop the first immunotherapy for peanut allergy, DBV had the early lead, filing an NDA for their patch in 2018. But on December 20 of that year, the company withdrew their application after, they said, meeting with regulators and determining they had not submitted “sufficient detail regarding data on manufacturing procedures and quality controls.” Aimmune filed their BLA 3 days later and won approval as the first immunotherapy for peanuts this month.

February 21, 2020 09:15 AM ESTUpdated 11:09 AM

Kathy Wong

Peer Review

Antonio Gualberto starts post-Kura career at Eisai subsidiary H3; eFFECTOR co-founder Siegfried Reich jumps to Turning Point

→ Days after Kura Oncology announced the departure of co-founder Antonio Gualberto, we finally know where he wound up. Eisai subsidiary H3 Biomedicine has recruited him as CMO to finding the right patients to its four clinical-stage small molecule assets hitting genomic drivers of cancer.

“Challenges of these and many other precision medicine approaches are on one hand technical — a need for robust and precise diagnostics — and on the other hand derived by the challenge to alter standard clinical practice in settings where patient screening, e.g. by tumor DNA sequencing, is not standard practice,” he wrote to Endpoints News on his way back to Boston from Eisai’s Tokyo offices. “Only compelling clinical activity can drive clinicians and pathologists to modify standard clinical practice.”