Let's talk AI: Top R&D ex­ecs tack­le where we are and where we're head­ed with this cru­cial new tech­nol­o­gy

Bioreg­num Opin­ion Col­umn by John Car­roll at the US-Chi­na Bio­phar­ma In­no­va­tion and In­vest­ment Sum­mit in Shang­hai on Oc­to­ber 23, 2018; Cred­it: End­points News, Pharm­Cube

As a biotech ex­ec with a back­ground in com­pu­ta­tion­al chem­istry, Rosana Kapeller has been watch­ing the hub­bub over ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing in bio­phar­ma play out with a con­sid­er­able de­gree of skep­ti­cism.

“Peo­ple talk about ML and AI with­out know­ing what they are talk­ing about,” the out­spo­ken Nim­bus alum told me ear­li­er in the week. And there are some big pit­falls the unini­ti­at­ed are like­ly to drop in­to with­out warn­ing if they fol­low the crowd in­to AI with­out think­ing things through first. 

Now at Google’s GV as an en­tre­pre­neur-in-res­i­dence tak­ing a spe­cial­ist ap­proach to com­pu­ta­tion­al tech­nolo­gies, Kapeller has a lot of re­spect for Stan­ford AI star Daphne Koller, who’s dived in­to the com­plex task of draw­ing her own map to fol­low when it comes to us­ing AI in R&D. And she won­ders where oth­ers with­out Koller’s back­ground will wind up if they start us­ing AI and ML in par­tic­u­lar fields where the da­ta are lack­ing.

That’s the back­drop to a con­ver­sa­tion I’ve long looked for­ward to. 

Rosana Kapeller GV

Next Tues­day in Philadel­phia dur­ing the an­nu­al BIO con­fab, Kapeller and a num­ber of top ex­ecs from some of the biggest play­ers in the in­dus­try will join me to dis­cuss how they’re us­ing AI and ML right now. In every case, these are large­ly new ef­forts where bio­phar­mas are look­ing to this tech­nol­o­gy to im­prove their odds of suc­cess — which I think most every­one in R&D would agree are pret­ty poor. And con­trary to what you may as­sume, there’s a very prac­ti­cal at­ti­tude about what they can do and what they can’t, and how they be­lieve this field will evolve over the next 4-5 years.

Daphne Koller Twit­ter

The rest of the line­up for next week’s dis­cus­sion looks like this: GSK’s Tony Wood, Saurabh Sa­ha at Bris­tol-My­ers Squibb, Hugo Ceule­mans from J&J, Badhri Srini­vasan at No­var­tis and Shoibal Dat­ta, a Bio­gen vet now at PPD.

I hope you can join us for this event and con­tribute your own thoughts and ques­tions. You can get tick­ets here. 


Im­age: John Car­roll

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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News brief­ing: Ve­rastem CMO ex­its two weeks af­ter join­ing com­pa­ny; Ther­mo Fish­er inks $550M M&A deal

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

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A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

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Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Bris­tol My­ers Squibb gets re­view date for Op­di­vo com­bo in gas­tric can­cer, look­ing to over­turn Keytru­da's 3-year lead

The past two months have been tough for Bristol Myers Squibb and its checkpoint inhibitor Opdivo after setbacks in lung and brain cancers. But in the battle against Merck’s Keytruda, any success matters — and now Bristol could be looking at a quick approval for Opdivo in an unmatched indication.

The FDA will launch a speedy review of a combination of Bristol Myers Squibb’s Opdivo and chemotherapy to treat first-line patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer or esophageal adenocarcinoma, the drugmaker said Wednesday. The agency set an action date of May 25 for the application.

Covid-19 claims an­oth­er PDU­FA vic­tim as Glax­o­SmithK­line push­es back planned PD-1 roll­out

Bristol Myers Squibb isn’t the only pharma giant that’s been standing in the FDA’s waiting line for site inspections.

GlaxoSmithKline is telling us today that their H2 2020 PDUFA deadline for the PD-1 drug dostarlimab — picked up in its Tesaro buyout — was pushed back due to a delay in the manufacturing site inspection needed for a regulatory decision. And that is forcing the company to revise its timeline.

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