Keith Dionne, Casma CEO (Casma)

Kei­th Dion­ne's Cas­ma lines up a $50M ven­ture round to con­tin­ue the pre­clin­i­cal work on a cel­lu­lar re­cy­cling pro­gram

Two years af­ter launch­ing out of Third Rock’s in­cu­ba­tor, Cas­ma Ther­a­peu­tics has land­ed a $50 mil­lion Se­ries B for pre­clin­i­cal de­vel­op­ment of its mus­cu­lar dy­s­tro­phy and Au­tophagy De­grad­er Plat­form (ADP) pro­grams.

Cas­ma kicked off in 2018 with a $58.5 mil­lion Se­ries A and a staff of ex­perts in au­tophagy, the cell’s “garbage dis­pos­al.” The biotech’s TRPML1 ag­o­nist pro­gram for mus­cu­lar dy­s­tro­phy is ex­pect­ed to hit the clin­ic in 2022, ac­cord­ing to CEO Kei­th Dionne, and the ADP pro­gram is about a year be­hind that.

“If you imag­ined your house, where you plug up the garbage dis­pos­al, plug up the toi­lets and stop tak­ing out the trash, you would find, prob­a­bly fair­ly quick­ly, the house would be­come un­liv­able. And that’s es­sen­tial­ly the same thing that hap­pens in­side of a cell,” Dionne said. “If we don’t have these mech­a­nisms of main­tain­ing home­osta­sis to clear out ma­te­ri­als that were maybe used once, or maybe mu­tat­ed to be­gin with but oth­er­wise built up in­side of the cell —  the cell be­comes dys­func­tion­al.”

The biotech seeks to boost the cell’s re­cy­cling sys­tem, which could treat a range of ill­ness­es, in­clud­ing lyso­so­mal stor­age and in­flam­ma­to­ry dis­or­ders, liv­er and mus­cle dis­eases, and neu­rode­gen­er­a­tion.

The dis­cov­ery of au­tophagy mech­a­nisms won the No­bel Prize in 2016. While there are cur­rent­ly no ap­proved drugs in the field, some have shown to en­hance au­tophagy as a side ef­fect.

“When we launched the com­pa­ny, it was re­al­ly with the re­al­iza­tion that we thought we could, and that we need­ed to, de­vel­op drugs that were spe­cif­ic to the in­duc­tion of au­tophagy, not hit­ting au­tophagy as one of the 5 or 10 oth­er path­ways that they (oth­er drugs) were touch­ing on,” Dionne said. “Look­ing at the pre­clin­i­cal lit­er­a­ture it be­came very ap­par­ent that the num­ber of dis­eases that are im­pact­ed by this is quite large. And again, that is part of the ex­cite­ment of get­ting go­ing.”

The com­pa­ny will use the Se­ries B fund­ing to ad­vance both its ADP and TRPML1 pro­grams. TRPML1 reg­u­lates the re­pair of the plas­ma mem­brane of mus­cle cells fol­low­ing dam­age, ad­dress­ing the “core pathol­o­gy in mul­ti­ple forms of mus­cu­lar dy­s­tro­phy,” ac­cord­ing to Cas­ma.

Skip Vir­gin

The biotech’s sci­en­tif­ic founders in­clude au­tophagy ex­pert Beth Levine, who died ear­li­er this year af­ter a fight with breast can­cer. The for­mer Cen­ter for Au­tophagy Re­search di­rec­tor was known for dis­cov­er­ing the mam­malian au­tophagy gene, BECN1. The team al­so in­cludes An­drea Bal­labio, a leader in tran­scrip­tion­al reg­u­la­tion of lyso­so­mal bio­gen­e­sis and au­tophagy; James Hur­ley, who’s stud­ied the struc­ture of au­tophagy com­plex­es; and Vir Biotech­nol­o­gy CSO and EVP of re­search Her­bert “Skip” Vir­gin, who’s re­searched the role of au­tophagy genes in in­flam­ma­tion and im­mu­ni­ty.

The Se­ries B was led by The Col­umn Group, and joined by Third Rock, Even­tide As­set Man­age­ment, Schroder Ad­veq, and oth­er undis­closed in­vestors.

“It’s ex­cit­ing to work in a new space of sci­ence, open­ing up a whole new field, and then to see that start to trans­late in­to ther­a­peu­tics. So that’s pulled us through this whole Covid pe­ri­od and … kept us go­ing through the en­tire process to make progress on them. So (there’s) just a pal­pa­ble sense of ex­cite­ment around the com­pa­ny,” Dionne said.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA have vowed not to let politics get in the way of science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped health agencies under his purview — including the FDA — of their rulemaking ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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Eli Lilly CSO Dan Skovronsky (file photo)

#ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

#ES­MO20: As­traZeneca aims to spur PRO­found shift in prostate can­cer treat­ment with Lyn­parza OS da­ta

AstraZeneca has unveiled the final, mature overall survival data that cemented Lynparza’s first approval in prostate cancer approval — touting its lead against rivals with the only PARP inhibitor to have demonstrated such benefit.

But getting the Merck-partnered drug to the right patients remains a challenge, something the companies are hoping to change with the new data cut.

The OS numbers on the subgroup with BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer are similar to the first look on offer when the FDA expanded the label in May: Lynparza reduced the risk of death by 31% versus Xtandi and Zytiga. Patients on Lynparza lived a median of 19.1 months, compared to 14.7 months for the anti-androgen therapies (p = 0.0175).

#ES­MO20: It’s not just Keytru­da any­more — Mer­ck spot­lights 3 top ear­ly-stage can­cer drugs

Any $12 billion megablockbuster in the portfolio tends to overshadow everything else in the pipeline. Which is something Merck can tell you a little bit about.

Keytruda not only dominates the PD-(L)1 field, it looms over everything Merck does, to the point some analysts wonder if Merck is a one-trick pony.

There’s no shortage of Keytruda data on display at ESMO this weekend, but now the focus is shifting to the future role of new drugs and combos in maintaining that lead position for years to come. And the pharma giant has a special focus for 3 early-stage efforts where Roger Perlmutter’s oncology team is placing some big bets.

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Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.