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.

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.”

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.

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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.