L to R: Jeroen Bekaert, Ivan Dimov and Nate Fernhoff, Orca Bio (Orca)

Months af­ter achiev­ing uni­corn sta­tus, Or­ca steers in­to the fast lane with an RMAT des­ig­na­tion for cell ther­a­py can­di­date

A few months af­ter its last big catch — a $192 mil­lion Se­ries D and rare uni­corn sta­tus — Or­ca Bio has reeled in the FDA’s re­gen­er­a­tive med­i­cine ad­vanced ther­a­py (RMAT) des­ig­na­tion for its ex­per­i­men­tal cell ther­a­py to treat blood stem cell trans­plant pa­tients.

Or­ca, one of this year’s End­points 11 star­tups, was found­ed by three en­tre­pre­neurs who were in­trigued by Irv Weiss­man’s cell pu­rifi­ca­tion work while study­ing at Stan­ford. No­var­tis — then San­doz — had pur­chased Weiss­man’s spin­out SyS­temix in the ’90s, then scrapped it due to prac­ti­cal chal­lenges. But Jeroen Bekaert, Ivan Di­mov and Nate Fern­hoff saw po­ten­tial there.

Now, the biotech has RMAT and or­phan drug des­ig­na­tion to steer its cell ther­a­py can­di­date Or­ca-T through the clin­ic. RMAT des­ig­na­tion comes with the ben­e­fits of both fast track and break­through ther­a­py des­ig­na­tions. Or­ca-T en­tered a Phase I/II tri­al in pa­tients un­der­go­ing stem cell trans­plants for var­i­ous blood can­cers last No­vem­ber, ac­cord­ing to an NIH fil­ing. The biotech ex­pects to read out da­ta from that tri­al this De­cem­ber at the an­nu­al Amer­i­can So­ci­ety of Hema­tol­ogy meet­ing.

“The RMAT and ODD for Or­ca-T is up­lift­ing news for pa­tients with var­i­ous blood can­cers in­clud­ing acute myeloid leukemia, acute lym­phoid leukemia, myelodys­plas­tic syn­drome and myelofi­bro­sis,” Di­mov said in a state­ment.

While a hematopoi­et­ic stem cell trans­plant is the best op­tion for some pa­tients with blood can­cer, the pro­ce­dure can lead to dan­ger­ous com­pli­ca­tions, such as graft-ver­sus-host dis­ease, in which im­mune cells from the donor at­tack the pa­tient’s healthy cells. Or­ca-T is de­signed to re­pro­gram the dis­eased blood sys­tem, by match­ing “a spe­cif­ic com­po­si­tion of stem and im­mune cells to the im­muno­log­i­cal needs of a pa­tient.”

Or­ca’s re­cent Se­ries D brought its to­tal raise to about $300 mil­lion, and came in as the com­pa­ny’s val­u­a­tion sur­passed $1 bil­lion. Plus, the biotech touts Weiss­man, for­mer No­var­tis CEO Joe Jimenez, and Lyell head and for­mer NCI chief Rick Klaus­ner as its ad­vi­sors.

Di­mov spoke with End­points News last month about the in­spi­ra­tion be­hind the biotech’s 2016 launch:

It al­most seems un­eth­i­cal from a hu­man per­spec­tive be­cause some of the ini­tial re­sults sug­gest high­ly cu­ra­tive ef­fects and the whole thing was shut down for over a decade be­cause of some of these chal­lenges of prac­ti­cal­ly im­ple­ment­ing these things and de­ploy­ing them. So that was sort of the key dri­ver for us to get start­ed. And we al­ways knew we want­ed to ful­fill that dream all the way through and not re­al­ly stop at a cer­tain stage and leave it for some­one else.

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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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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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.

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

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

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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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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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: FDA anoints Gilead­'s remde­sivir as the Covid-19 treat­ment win­ner, hand­ing down full ap­proval — de­spite some deep skep­ti­cism

Seven months into the Covid-19 pandemic, the race to develop a treatment for the disease that’s proved to be the biggest health crisis in a century has an officially designated winner: Gilead. And they’re picking up the prize — worth billions in peak sales — despite a major study that concluded the drug was no help in reducing the number of people who die from the virus.

The FDA handed down a thumbs-up for remdesivir, the company announced Thursday afternoon, as the drug becomes the first fully approved treatment for Covid-19 in the US. Remdesivir, to be marketed as Veklury, will come with a label for treatment in adults and children older than 12 in Covid-19 cases that require hospitalization.

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Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

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.

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.