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.

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.

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.

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).

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.