David Baltimore (Vincent Yu/AP Images)

No­bel lau­re­ate David Bal­ti­more throws his weight be­hind I/O start­up look­ing to craft off-the-shelf CAR ther­a­pies

In the in­su­lar world of biotech, names mat­ter — and no names shine brighter than those lu­mi­nar­ies with a No­bel Prize on their record. Now, a Los An­ge­les start­up work­ing at the fur­thest fron­tiers of cell ther­a­peu­tics is stand­ing on the shoul­ders of a lau­re­ate of its own and look­ing to rewrite the fight against sol­id tu­mors.

On Tues­day, Ap­pia Bio launched with $52 mil­lion in Se­ries A fi­nanc­ing led by 8VC and a plat­form fo­cused on what are called in­vari­ant nat­ur­al killer T cells (iNKT), an ex­treme­ly rare im­mune cell the biotech hopes to reengi­neer to bust ma­lig­nant tu­mors through a “best of both worlds” at­tack plan, the com­pa­ny said.

Ap­pia’s work is found­ed on sci­ence from UCLA pro­fes­sor Lili Yang along­side a man­age­ment team of Kite Phar­ma vet­er­ans and a first-time CEO all grav­i­tat­ing around one man — David Bal­ti­more, the 83-year-old No­bel lau­re­ate and pres­i­dent emer­i­tus at Cal­Tech.

Bal­ti­more’s work on hu­man virus­es earned him the prize for phys­i­ol­o­gy and med­i­cine back in 1975, and since then his labs have most­ly been in­volved in non-trans­la­tion­al bench sci­ence. But in re­cent years, his Cal­Tech re­search team has propped up a trans­la­tion­al wing fo­cus­ing on the use of AAV-based gene trans­fer for use in stem cells.

Lili Yang

Yang, a 13-year vet­er­an of Bal­ti­more’s lab who moved up from grad­u­ate re­search as­sis­tant to post­doc to lead sci­en­tist on en­gi­neered im­mu­ni­ty, moved to UCLA in 2013 as an as­sis­tant de­part­ment head in mi­cro­bi­ol­o­gy, im­munol­o­gy and mol­e­c­u­lar ge­net­ics, where she start­ed work­ing on the po­ten­tial for ther­a­peu­tic iNK­Ts in on­col­o­gy, this time us­ing lentivi­ral gene trans­fer in­stead of AAV. In Sep­tem­ber 2019, her team pub­lished in Cell Stem Cell proof of con­cept for an “off-the-shelf,” en­gi­neered iNKT pro­lif­er­at­ed in vi­vo through stem cells.

Af­ter show­ing some suc­cess in mice with­out any se­ri­ous safe­ty flags, Yang and col­lab­o­ra­tors Bal­ti­more and Pin Wang, a pro­fes­sor of chem­i­cal en­gi­neer­ing and ma­te­ri­als sci­ence and bio­med­ical en­gi­neer­ing at USC, saw a path to a po­ten­tial prod­uct and kick­start­ed Ap­pia last year.

The promise of an al­lo­gene­ic cell ther­a­py has been a hot area of in­vest­ment in re­cent years, but re­searchers have had a hard time show­ing that reengi­neered T cells that don’t come from pa­tients’ own bod­ies can pass the im­mune sys­tem with­out heinous side ef­fects. But with iNK­Ts, which co-ex­press NK and T cell mark­ers, the Ap­pia team thinks it can evade the graft vs. host dis­ease en­dem­ic to al­lo­gene­ic cell ther­a­pies and en­gi­neer mul­ti­ple routes of at­tack on tu­mors us­ing the in­nate and ac­tive im­mune sys­tem.

JJ Kang

Join­ing the sci­en­tif­ic brain­trust is JJ Kang, a for­mer part­ner at The Col­umn Group who re­ceived her doc­tor­ate in chem­i­cal bi­ol­o­gy at Cal­Tech be­fore a stint as pres­i­dent at Tenaya Ther­a­peu­tics from 2016 to 2018 — what she told End­points News was like “run­ning a com­pa­ny with train­ing wheels.” Bal­ti­more was in­stru­men­tal in in­tro­duc­ing Kang to the Col­umn team, and when she got the chance to head Ap­pia, she jumped.

“It’s so much about peo­ple and the way that things mesh to­geth­er,” she said. “In the aca­d­e­m­ic col­lab­o­ra­tion that led up to it, you al­ready had the founders that worked to­geth­er, and adding on the man­age­ment founders, we all kind of meshed well and it made sense to build a com­pa­ny.”

For Bal­ti­more, who helped Kang arrange the CEO role, he told End­points that de­spite her lack of ex­pe­ri­ence in im­munol­o­gy, Kang has tak­en to the plat­form and was ready to lead a biotech with big am­bi­tions.

“I think she’s in the per­fect po­si­tion to now move in­to a re­spon­si­ble man­age­r­i­al role,” Bal­ti­more said of Kang’s prepa­ra­tion for the job. “She has every oth­er form of ex­pe­ri­ence, she knows the peo­ple and the moves to get a com­pa­ny go­ing, and she’s re­al­ly de­vot­ed to the tech­nol­o­gy.”

The rest of the found­ing team is com­prised of Jeff Wiezorek, the for­mer head of cell ther­a­py de­vel­op­ment at Kite Phar­ma who will take on the CMO role, and Ed­mund Kim, Ap­pia’s chief op­er­at­ing of­fi­cer and for­mer VP of cor­po­rate de­vel­op­ment at Kite. Wiezorek ac­tu­al­ly shared a bench with Yang while at Cal­Tech — start­ing to see the bonds formed here? — while Kim is the odd man out look­ing to bring his ex­pe­ri­ence at a mar­ket­ed cell ther­a­py to bear at the fledg­ling firm.

Hav­ing that de­vel­op­ment ex­pe­ri­ence will bode well as the Ap­pia team hopes to trans­late its win­ning pre­clin­i­cal da­ta in­to a suc­cess­ful hu­man tri­al, which Kang said the com­pa­ny hopes to en­ter by late 2023. Bal­ti­more said the plat­form holds promise, but it’s still too ear­ly to say whether it will even­tu­al­ly prove ef­fec­tive.

“There’s a strong like­li­hood it will work, but you don’t know un­til you test it,” he said. “There will be sur­pris­es, there will be an­noy­ances, but I feel con­fi­dent that this is the right mo­ment to try.”

The Se­ries A was joined by Two Sig­ma Ven­tures among oth­ers, with par­tic­i­pa­tion from seed in­vestors Sher­pa Health­care Part­ners and Freeflow Ven­tures.

Am­gen lays off about 300 work­ers, cit­ing 'in­dus­try head­wind­s'

Amgen has laid off about 300 employees, a company spokesperson confirmed to Endpoints News via email Sunday night.

Employees posted to LinkedIn in recent days about layoffs hitting Amgen last week. The Thousand Oaks, CA-based biopharma, which employs about 24,000 people, said the reduction “mainly” impacted US-based workers on its commercial team.

Drug developers of all sizes, including small upstarts and pharma giants, have let employees go in recent months as the biopharma market drags through a quarters-long winter doldrum.

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Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Boehringer In­gel­heim touts pre­ven­tion re­sults in rarest form of pso­ri­a­sis

Boehringer Ingelheim uncorked some positive results suggesting that Spevigo can help prevent flare-ups in patients with a severe form of psoriasis, months after the drug was approved to treat existing flares.

Spevigo, an IL-36R antibody also known as spesolimab, met its primary and a key secondary endpoint in the Phase IIb EFFISAYIL 2 trial in patients with generalized pustular psoriasis (GPP), Boehringer announced on Monday. While the company is keeping the hard numbers under wraps until later this year, it said in a news release that it anticipates sharing the results with regulators.

As­traZeneca, No­vo Nordisk and Sanofi score 340B-re­lat­ed ap­peals court win over HHS

AstraZeneca, Novo Nordisk, and Sanofi won an appeals court win on Monday, as the US Court of Appeals for the Third Circuit found that the companies cannot be forced to provide 340B-discounted drugs purchased by hospitals from an unlimited number of community and specialty pharmacies.

“Legal duties do not spring from silence,” the decision says as the court makes clear that the federal government’s interpretation of the “supposed requirement” that the 340B program compels drugmakers to supply their discounted drugs to an unlimited number of contract pharmacies is not correct, noting:

Ap­peals court toss­es J&J's con­tro­ver­sial 'Texas two-step' bank­rupt­cy case

A US appeals court has ruled against Johnson & Johnson’s use of bankruptcy to deal with mounting talc lawsuits, deciding that doing so would “create a legal blind spot.”

The Third Circuit Court of Appeals reversed a previous bankruptcy court decision on Monday, calling for the dismissal of a Chapter 11 filing by J&J’s subsidiary LTL Management.

Faced with more than 38,000 lawsuits alleging its talc-based products caused cancer, J&J spun its talc liabilities into a separate company called LTL Management back in October 2021 and filed for bankruptcy, a controversial move colloquially referred to as a “Texas two-step” bankruptcy. Claimants argued that the strategy is a misuse of the US bankruptcy code — and on Monday, a panel of judges agreed.

Chad Mirkin, Flashpoint co-founder

‘The field is at a flash­point’: New Chad Mirkin-found­ed biotech hopes to make more ef­fec­tive can­cer vac­cines

Following the success of the mRNA Covid vaccines, cancer vaccines are seeing renewed interest after years of middling results. But a group of researchers suggests that more attention needs to be paid not to what goes into those vaccines, but how the parts are put together.

In a recent paper published in Nature Biomedical Engineering, researchers led by Northwestern University’s Chad Mirkin describe how the placement of different antigens in a cancer vaccine impacts its efficacy. The paper builds on past work done by Mirkin’s lab that suggests the structure, or how the parts of a vaccine are arranged, impact a vaccine’s efficacy, not just its components.

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#JPM23: Reg­u­la­to­ry un­cer­tain­ty? What about M&A? Da­ta rule? Alessan­dro Masel­li and John Car­roll take out their crys­tal balls

Endpoints editor and founder John Carroll sat down the Catalent CEO Alessandro Maselli to talk about what’s ahead in 2023. Right or wrong, this covers all the big issues faced by biopharma. This transcript has been edited for brevity and clarity.

John Carroll:

I think 2022 had to be one of the worst years ever for crystal balls. You went into 2022 thinking all sorts of nice things about what was ahead, not thinking about a European land war, maybe not thinking that the Federal Reserve was going to be jacking up interest rates as fast as they could to get ahead of inflation. Just a tremendous number of macroeconomic issues that were out there. The sudden and complete collapse of support on the markets in Nasdaq for biotech. A lot of darlings in the industry that had been out there for a while suddenly found themselves moving from a really hot market to a really cold market all of a sudden and had to make a lot of different changes in terms of strategizing.

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

Al­to Neu­ro­science bags $25M for four Phase II drugs

Another $25 million is flowing the way of a California biotech attempting to fix the “trial and error” system in neuroscience drug R&D.

Alto Neuroscience picked up the capital from Alpha Wave Ventures via an extension to its Series B, bringing total equity raised to $100 million since the startup’s 2019 founding. The biotech also recently signed up for a $35 million credit facility.

All that capital will help the biotech investigate four drugs through four Phase II readouts, Alto said Monday morning. That means enough money to keep the lights on into 2025, a year longer than projected under the original Series B close.

Tyler Golato, VitaDAO co-initiator

Pfiz­er-backed de­cen­tral­ized co­op­er­a­tive rais­es $4.1M to fund longevi­ty re­search

Months after Pfizer Ventures put a decentralized cooperative on the map by announcing a $500,000 infusion into its mission of funding human longevity research, the group — VitaDAO — is tying the bow around a $4.1 million fundraising round.

As its name implies, VitaDAO calls itself a DAO, or decentralized autonomous organization. Like many other such groups, it aims to run on a relatively flat, global structure using contracts on blockchains and crypto-tied tokens in hopes of proving a new way to fund big ideas and bridge the valley of death for very early-stage research on anti-aging.