A2 Bio­ther­a­peu­tics nabs $71M-plus Se­ries B for se­lec­tive cell ther­a­py pipeline

More than 15 years af­ter beat­ing can­cer him­self, A2 Bio­ther­a­peu­tics CEO Scott Forak­er has pulled in a $71.5 mil­lion Se­ries B round to send a mys­tery can­di­date for sol­id tu­mors in­to the clin­ic.

“It’s quite per­son­al for me and I re­al­ly am dri­ven to make a dif­fer­ence,” said Forak­er, a non-Hodgkin’s lym­phoma sur­vivor. “I know what it’s like to sit in an in­fu­sion chair for hours and get dos­es of chemother­a­py.”

The Agoura Hills, CA-based biotech is work­ing on an en­gi­neered T-cell ther­a­py, which us­es an ac­ti­vat­ing and block­ing mech­a­nism to se­lec­tive­ly tar­get can­cer cells. The ac­ti­va­tor at­tacks tu­mor cells, while the block­er rec­og­nizes nor­mal cells and pro­tects them.

Scott Forak­er

“The de­struc­tion of nor­mal cells as you’re try­ing to kill the tu­mor cells is quite a prob­lem,” Forak­er said. “Most can­cer ther­a­peu­tics re­al­ly try to care­ful­ly bal­ance pro­vid­ing enough tox­i­c­i­ty to kill the cells, but not so much that it harms the nor­mal cells too much.”

Forak­er — along with rough­ly half of A2’s staff of more than 40 — came by way of Am­gen. For the last 8 years, he served as VP and gen­er­al man­ag­er of the com­pa­ny’s biosim­i­lars di­vi­sion. But when fel­low Am­gen col­league Sasha Kamb left to start A2, “every­one was quite in­trigued,” Forak­er said. The op­por­tu­ni­ty arose to join Kamb at A2 last May, and he jumped.

A2 has al­so tapped William Go, for­mer VP of clin­i­cal de­vel­op­ment at Kite Phar­ma, and Michelle Kreke, who led Kite’s process de­vel­op­ment, to its team. “We think we’ve com­bined the best of biotech… R&D drug de­vel­op­ment,” Forak­er said.

The biotech emerged from stealth mode last year with a $57 mil­lion Se­ries A and its sights set on the clin­ic. While Forak­er didn’t di­vulge much about the com­pa­ny’s pipeline, he said the Se­ries B will fund work on three can­di­dates, the first of which is ex­pect­ed to hit the clin­ic in 2022. The two oth­ers, he said, are not far be­hind.

“This is a re­al­ly fas­ci­nat­ing new tech­nol­o­gy that has the po­ten­tial to rev­o­lu­tion­ize the way… sol­id tu­mor can­cers are treat­ed,” he said. The com­pa­ny will work close­ly with the FDA over the next year on clin­i­cal de­vel­op­ment plans, he added.

Se­ries B fi­nanc­ing will al­so sup­port the ex­pan­sion of A2’s pipeline be­yond its cur­rent three can­di­dates. A2 re­searchers be­lieve dif­fer­ent com­bi­na­tions of ac­ti­va­tors and block­ers can be used to treat dif­fer­ent types of can­cers.

“The po­ten­tial of this ther­a­py is quite vast which is part of the rea­son we’re so ex­cit­ed about it, (and) anx­ious to get it in­to hu­man clin­i­cal test­ing,” Forak­er said.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

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He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

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All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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