Third Rock sinks $59M in­to Cas­ma's 're­cy­cling' tech, with neu­ro on the mind

It feels like launch sea­son, and Third Rock is on a roll. The ven­ture fund and start­up in­cu­ba­tor is un­veil­ing yet an­oth­er biotech — its third pub­lic launch in a num­ber of weeks. This one is step­ping out with a $58.5 mil­lion Se­ries A, and a locked and loaded staff of au­tophagy ex­perts.

Beth Levine

The com­pa­ny, called Cas­ma Ther­a­peu­tics, has qui­et­ly been work­ing on tech­nol­o­gy that it hopes will ar­rest — or even re­verse — the pro­gres­sion of both rare and broad ge­net­ic dis­eases, in­clud­ing the fail­ure-rid­den space of neu­rode­gen­er­a­tion.

To do this, Cam­bridge, MA-based Cas­ma is us­ing new strate­gies to boost the body’s abil­i­ty to break down ma­te­r­i­al it no longer needs, like mis­fold­ed pro­teins, in­vad­ing pathogens, or spare or­ganelles. Think of it as the cell’s re­cy­cling sys­tem. When this slaugh­ter and sal­vage process (called au­tophagy) isn’t work­ing — or when it’s over­whelmed by dis­ease — all sorts of things can go awry.

Cas­ma’s CEO Kei­th Dionne tells me the com­pa­ny is look­ing at us­ing small mol­e­cules to drug var­i­ous pro­teins in­volved in trig­ger­ing au­tophagy. The idea is to in­ter­vene at strate­gic points in the au­tophagy process to boost the re­cy­cling sys­tem. And they’ve got pre­clin­i­cal da­ta that sug­gest turn­ing the di­al up on au­tophagy could lead to treat­ments for a wide range of dis­ease, in­clud­ing lyso­so­mal stor­age dis­or­ders, liv­er and mus­cle dis­eases, in­flam­ma­to­ry dis­or­ders, and neu­rode­gen­er­a­tion.

An­drea Bal­labio

“In pre­clin­i­cal mod­els, we’re see­ing a ma­jor ef­fect in a num­ber of dis­eases,” Dionne said. “And it’s proven to be quite safe. If you ge­net­i­cal­ly in­duce au­tophagy in mice, they live health­i­er, longer lives — freer of dis­ease.”

Cas­ma al­ready has “4 to 5” pro­grams in the pipeline, Dionne said.

James Hur­ley

In­ter­est in this field has been warm­ing up since 2016, when the dis­cov­ery of key mech­a­nisms in au­tophagy earned the No­bel Prize for Phys­i­ol­o­gy or Med­i­cine. Since then, the sci­ence has moved rapid­ly. Many com­pa­nies are now pur­su­ing the idea of au­tophagy’s im­pact on dis­ease.

“There are a num­ber of drugs known to en­hance au­tophagy as a side ef­fect,” Dionne said. “The key thing we want to do is go af­ter the spe­cif­ic mech­a­nism, with­out a lot of oth­er side ef­fects.”

Her­bert “Skip” Vir­gin

Cas­ma’s sci­en­tif­ic founders cer­tain­ly have loads of com­bined ex­pe­ri­ence in au­tophagy. Among them is Beth Levine, di­rec­tor of the Cen­ter for Au­tophagy Re­search, who’s rec­og­nized as a world­wide ex­pert in au­tophagy for her dis­cov­ery of the mam­malian au­tophagy gene, BECN1, and oth­er com­po­nents of the au­tophagy path­way. Then there’s An­drea Bal­labio, a leader in the field of tran­scrip­tion­al reg­u­la­tion of lyso­so­mal bio­gen­e­sis and au­tophagy; James Hur­ley, who per­formed ground­break­ing stud­ies to de­ter­mine the struc­ture and ac­tiv­i­ty of au­tophagy core com­plex­es; and Her­bert “Skip” Vir­gin, who forged new ground in the un­der­stand­ing of au­tophagy’s role in in­flam­ma­tion and im­mu­ni­ty.

Dionne said the com­pa­ny’s Se­ries A should get them 2 to 3 years of run­way, giv­ing the com­pa­ny the chance to de­vel­op spe­cif­ic en­hancers of au­tophagy and val­i­date if they’re work­ing.

Im­age: Kei­th Dionne. Cas­ma

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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(Credit: Shutterstock)

Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Alan Wise (L) and Peter Trill (Duke Street Bio)

They sold their last biotech to Mer­ck. Now they're back with a PARP out­fit named af­ter a Lon­don street

In 2016, Peter Trill and Alan Wise sold IOmet Pharma (an I/O outfit as the name suggests) to Merck for $400 million.

Now, some six years later, the duo has returned with another cancer biotech, Duke Street Bio, that emerged from stealth Tuesday. Duke Street Bio, named for the street where it’s located in London, is making its public debut as a next-gen PARP player, hoping to break into a field that already has a number of Big Pharma competitors.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”