Sonny Hsiao, Acepodia CEO

Ace­po­dia un­corks a very ear­ly win for its an­ti­body-nat­ur­al killer cell con­ju­gates in HER2 tu­mors

Can the an­ti­body-drug con­ju­gate mod­el be ap­plied to an off-the-shelf NK cell ther­a­py? Ace­po­dia un­corked pre­lim­i­nary Phase I da­ta at #ES­MO21 that sug­gest the an­swer may be yes.

Pri­vate biotech Ace­po­dia’s lead “an­ti­body cell con­ju­ga­tion” (ACC) can­di­date ACE1702 was well-tol­er­at­ed in sev­en pa­tients with ad­vanced HER2 tu­mors who re­ceived low­er dos­es of the drug, CEO Son­ny Hsiao an­nounced ear­ly Thurs­day. The pa­tients who re­ceived the first four dose lev­els showed no signs of cy­tokine re­lease syn­drome, neu­ro­tox­i­c­i­ty or graft ver­sus host dis­ease — and one of them achieved a con­firmed par­tial re­sponse; not earth-shat­ter­ing, but a pos­i­tive sign.

“We didn’t ex­pect to see the re­sult on the ef­fi­ca­cy,” Hsiao told End­points News ahead of ES­MO. More than a decade af­ter the dis­cov­ery of the com­pa­ny’s ACC tech­nol­o­gy, Hsiao said the read­out “re­flects the hard work of our in­ter­na­tion­al teams as we con­tin­ue to gain mo­men­tum.”

The da­ta could al­so spell good news for JW Ther­a­peu­tics, which plunked down an undis­closed amount last sum­mer to de­vel­op and com­mer­cial­ize the can­di­date in main­land Chi­na, Hong Kong and Macau.

Ace­po­dia’s core tech­nol­o­gy traces back to Hsiao’s re­search at Berke­ley, where he dis­cov­ered a way to con­ju­gate an­ti­bod­ies with NK cells in a sim­i­lar fash­ion to AD­Cs. While most NK cell ther­a­pies are al­ready ad­min­is­tered in con­junc­tion with an­ti­bod­ies, they’re usu­al­ly giv­en sep­a­rate­ly, mak­ing for less po­ten­cy, Ace­po­dia be­lieves. By con­ju­gat­ing the two, the can­cer-tar­get­ing an­ti­bod­ies are less like­ly to dif­fuse through­out the body, Hsiao told End­points ear­li­er this year.

The ACCs al­so help ac­ti­vate the en­tire im­mune sys­tem to fight the tu­mor, Hsiao added, as con­ju­gat­ing the NK cells di­rect­ly pro­vides for an en­hanced im­mune re­sponse com­pared to cur­rent NK cell ther­a­pies or the typ­i­cal ADC.

“It’s a mild chem­i­cal con­ju­ga­tion re­ac­tion to link the an­ti­body, which means we don’t need ge­net­ic en­gi­neer­ing,” the CEO said on Wednes­day. “Our ACC will ac­tu­al­ly be more stream­lined, quick­er and eas­i­er to man­u­fac­ture, and that will al­so re­flect the low pro­duc­tion cost,” com­pared to oth­er cell ther­a­pies, he added.

The Phase I tri­al is ex­pect­ed to in­clude about 16 pa­tients to­tal, count­ing pa­tients who were giv­en high­er dos­es. Hsiao says da­ta on those pa­tients won’t be ready un­til Q2 of next year. Af­ter find­ing the max­i­mum tol­er­at­ed dose, the CEO ex­pects to ex­pand the study by ap­prox­i­mate­ly nine to 12 pa­tients be­fore leap­ing in­to a Phase II test.

The Alame­da, CA-based biotech hooked a $47 mil­lion Se­ries B round back in March, bring­ing its to­tal raise up to $57 mil­lion. The two pro­grams com­ing up be­hind ACE1702 will tar­get CD20 and PD-L1, re­spec­tive­ly, with plans to launch the CD20 pro­gram in­to the clin­ic next year. An IND for the PD-L1 pro­gram will like­ly fol­low in the sec­ond half of next year, Hsiao said.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Nkarta CEO Paul Hastings at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Nkar­ta un­der­scores safe­ty of CAR-NK, boasts ear­ly re­spons­es

The first generation of personalized CAR-T therapies made big waves in the treatment of lymphoma for their stunning efficacy. Nkarta is hoping its off-the-shelf natural killer cell approach will stand out on safety — while keeping some of those impressive numbers on responses.

In a new update from its Phase I dose escalation study, the South San Francisco-based biotech reported that seven out of 10 patients treated with the highest doses of its NK cell therapy, NKX019, achieved a complete response, translating to a complete response rate of 70%.

Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.

Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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