Se­cre­tive? Mod­er­na de­tails its pipeline strat­e­gy at JPM but at least one crit­ic bites back

Stephane Ban­cel, Mod­er­na CEO

Mod­er­na out­lined its pipeline strat­e­gy at JP Mor­gan this week, fo­cus­ing specif­i­cal­ly on five clin­i­cal-stage pro­grams for new vac­cines as a rel­a­tive­ly low-risk ap­proach to demon­strat­ing that their mes­sen­ger RNA tech­nol­o­gy and the plat­form work. But the kick­back didn’t take long to strike, as a per­sis­tent crit­ic at Stat picked at one trou­bled pro­gram while rais­ing ques­tions about the over­all val­ue of an en­try-lev­el ap­proach fo­cused on a high vol­ume, low-mar­gin busi­ness like vac­cines.

Af­ter rais­ing $1.9 bil­lion from a host of deep-pock­et in­vestors and part­ners, the uni­corn biotech has be­come the sub­ject of in­tense in­ter­est, par­tic­u­lar­ly in Boston biotech cir­cles. And Mod­er­na’s re­luc­tance to de­tail its work in the past has left the biotech with a rep among some re­porters as a se­cre­tive, pun­ish­ing place that has seen off a string of staffers who couldn’t main­tain the fren­zied pace.

Mod­er­na CEO Stephane Ban­cel, though, says that at­tack is un­war­rant­ed. His pre­sen­ta­tion at JP Mor­gan is in­tend­ed to shine a light on what the com­pa­ny’s top prospects are as it preps for its first clin­i­cal da­ta.

Ban­cel’s strat­e­gy is sim­ple: In­stead of fo­cus­ing on a sin­gle lead ef­fort, the pri­vate biotech chose to ramp up a full pipeline of clin­i­cal pro­grams. If one fal­ters, oth­ers can step up to make the case that mR­NA is on its way to de­liv­er­ing a new ap­proach to ther­a­peu­tic de­vel­op­ment with a vast ar­ray of po­ten­tial tar­gets.

“Peo­ple are go­ing to re­al­ize one day that mR­NA is a very good way to make med­i­cines,” Ban­cel says. “We are play­ing a very long game, a 10, 20-year game. We are a very in­tense com­pa­ny. We want to get those drugs for pa­tients.” And that means tack­ling dis­eases like Zi­ka with their tech­nol­o­gy.

Mod­er­na went af­ter two pan­dem­ic flu strains ear­ly, says Ban­cel, so in­ves­ti­ga­tors could ac­cu­rate­ly mea­sure an­ti­body re­spons­es among treat­ment naive pa­tients who had nev­er been ex­posed to the virus. In ad­di­tion to Zi­ka there’s an undis­closed pro­gram part­nered with Mer­ck that’s now in the clin­ic and sev­en pre­clin­i­cal pro­grams, in­clud­ing an­oth­er Mer­ck part­ner­ship on a per­son­al­ized can­cer vac­cine.

One of the com­pa­ny’s most promi­nent crit­ics, Stat’s Dami­an Garde, re­port­ed to­day that safe­ty is­sues hob­bled a ther­a­py for Crigler-Na­j­jar syn­drome — part­nered with Alex­ion — which Ban­cel promised a year ago would get in­to the clin­ic in 2016. That safe­ty is­sue, Garde added, raised fresh ques­tions about Mod­er­na’s high-stakes gam­ble on mR­NA, un­der­min­ing its $5 bil­lion val­u­a­tion it has en­joyed while rais­ing a moun­tain of cash.

Ban­cel tells me that a long-an­tic­i­pat­ed move to cash in on their val­u­a­tion through an IPO, though, won’t be hap­pen­ing this year.

“We will not go pub­lic this year,” he tells me. “This has been dis­cussed by the board. 2017 is not the right time. 2016 and ’17 is a crit­i­cal time for the com­pa­ny to move from dis­cov­ery to a de­vel­op­ment com­pa­ny with scale.”

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.