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.

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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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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David Mazzo, Caladrius Biosciences CEO

Blam­ing Covid dis­rup­tion, sup­ply short­ages, mi­cro­cap biotech hits the brakes on cell ther­a­py tri­al

More than two years into the Covid-19 pandemic, we’re not done seeing its disruption of biotech.

That’s according to Caladrius execs, who cited the pandemic as one reason for suspending its Phase IIb trial of a cell therapy as a treatment for coronary microvascular dysfunction (CMD). The company plans to go ahead on an interim analysis before deciding what to do with the program, which was built on a licensing deal with Shire in 2018.

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.

Bris­tol My­ers Squibb sues No­var­tis for roy­al­ties sur­round­ing the use of trans­genic mice

Two Big Pharma companies are going to the mat over genetically modified mice in a licensing dispute.

Bristol Myers Squibb is suing Novartis in New York over a dispute concerning an evaluation, research and commercialization agreement stretching back to the late ’90s initially inked between Novartis and BMS’ predecessor Medarex. The deal in question allowed Novartis to use Medarex’s patented transgenic mice to develop therapeutic drugs. Novartis agreed to pay Medarex – and subsequently BMS – a royalty on sales of drugs it developed using the mice.