FDA, EMA put a star CAR-T from blue­bird and Cel­gene on the VIP list

Dur­ing the last AS­CO blue­bird bio and its part­ners at Cel­gene an­gled in­to cen­ter stage with an up­date on their BC­MA-tar­get­ing CAR-T drug bb2121 for mul­ti­ple myelo­ma that wowed the can­cer crowd.

David David­son, blue­bird

The FDA as well as Eu­ro­pean reg­u­la­tors were pay­ing at­ten­tion. To­day the two com­pa­nies an­nounced that both groups had put the pro­gram on their VIP list, with a break­through ther­a­py des­ig­na­tion right along­side PRIME sta­tus.

Here’s what they were re­spond­ing to from AS­CO:

With 18 pa­tients evalu­able for ef­fi­ca­cy, there were 15 in what blue­bird termed ac­tive dos­ing reg­i­mens above the min­i­mum for bb2121, and all of them achieved an ob­jec­tive re­sponse. Twelve achieved a very good par­tial re­sponse to com­plete re­sponse, as­so­ci­at­ed with longer sur­vival times. And of the 4 pa­tients evalu­able for min­i­mal resid­ual dis­ease sta­tus, all were MRD neg­a­tive with on­ly a few or no myelo­ma cells in cir­cu­la­tion.

That is out­stand­ing, and these days it’s the kind of ther­a­py that gets pushed along by reg­u­la­tors anx­ious to see these new stan­dards of care ex­pand on the mar­ket as fast as pos­si­ble. It’s al­so a big help for these com­pa­nies as they look to shine a light on this ther­a­py at the up­com­ing ASH con­fer­ence, which you can read more about here.

David David­son, the CMO for blue­bird bio, had this to say:

Ear­ly da­ta sug­gest that treat­ment with bb2121 has the po­ten­tial to in­duce durable re­spons­es in this pa­tient pop­u­la­tion. It is en­cour­ag­ing for both the FDA and EMA to iden­ti­fy bb2121 as a can­di­date for ac­cel­er­at­ed de­vel­op­ment as we con­tin­ue our work with Cel­gene to bring this ther­a­py to pa­tients in need of new op­tions.

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Jeff Albers, Blueprint CEO

Di­ag­nos­tic champ Roche buys its way in­to the RET ti­tle fight with Eli Lil­ly, pay­ing $775M in cash to Blue­print

When Roche spelled out its original $1 billion deal — $45 million of that upfront — with Blueprint to discover targeted therapies against immunokinases, the biotech partner’s RET program was still preclinical. Four years later, pralsetinib is on the cusp of potential approval and the Swiss pharma giant is putting in much more to get in on the commercial game.

Roche gains rights to co-develop and co-commercialize the drug, with sole marketing responsibility for places outside the US and China (where CStone has staked its claim).

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President Trump (left) and NIAID chief Anthony Fauci in the White House press room, April 22, 2020 (Michael Reynolds/Sipa via AP Images)

White House tries to dis­cred­it An­tho­ny Fau­ci — could he be on his way out?

For two months in late winter and early spring, Anthony Fauci and President Trump stood in uneasy co-existence at White House briefings — an unlikely truce between an infectious disease official who had helped combat AIDS and Ebola and a president who repeatedly denied the danger of a virus that would go on to kill 100,000 Americans, repeatedly rejected masks and certain social distancing efforts, and promoted a drug with little scientific basis.

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SPACs are rid­ing on the biotech IPO boom

Billionaire hedge fund manager Bill Ackman’s push to raise $4 billion for his blank check company, Pershing Square Tontine Holdings, is casting a spotlight on the SPACs. And amid a historic SPAC boom, biotechs are placing several milestones on what some observers say is shaping up to be a third major track — besides IPO and M&A — to go public.

“SPACs were approximately 3% of the IPO market back in 2014, now they are almost 35% of all new listings,” Jay Heller, the Nasdaq’s head of capital markets, told Endpoints News.

Stéphane Bancel, Moderna CEO (Andrew Harnik/AP Images)

A top an­a­lyst turns the spot­light on Mod­er­na, fu­el­ing a fast-and-fu­ri­ous Street race over the fu­ture of mR­NA

Bioregnum Opinion Column by John Carroll

Four months ago, one of the favorite talking points on the biopharma social media wave length was whether Moderna shares $MRNA were priced right or were wildly inflated.

After all, said the naysayers, the company had never actually pushed a treatment to an approval. Did messenger RNA really work, coding cells to make a drug or a vaccine? And how about all that chatter about how ‘secretive’ they are, or were?

Now, as CEO Stéphane Bancel and the top execs push the company to the forefront of a frantic race to develop the first vaccine to fight against the reignited wildfire spread of Covid-19, all those questions have been magnified — along with the stock price.

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En­gi­neer­ing an on/off switch for CAR-T out of yeast and Juras­sic Park

Almost as soon as CAR-T emerged in the mid-2010s as a near-cure for some cancers, so did a question: How do you give this without risking killing patients?

At the time, James Patterson was wrapping an MD-PhD at a yeast lab at London’s Francis Crick Institute. Yeast may seem an unlikely place to find a fix for cancer therapy, but reading through other researchers’ solutions to CAR-T’s toxicity, Patterson wondered if a method long used by yeast biologists called auxotrophy might be useful. You genetically modify cells to make them dependent on a particular nutrient. Then you can make them live or die — proliferate or deplete — by giving or taking away that nutrient.

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FDA re­jects Ver­ri­ca’s skin warts pitch, a month af­ter flag­ging ap­pli­ca­tion ‘de­fi­cien­cies’

Two years ago, Verrica Pharma landed a $75 million IPO on the promise they could develop the first ever-FDA approved drug for molluscum contagiosum, an infection that causes warts. Now that won’t happen, at least not this summer.

The FDA today rejected Verrica’s application to have their lead topical drug VP-102 approved for molluscum contagiosum. The news likely came as little surprise to Verrica or its investors; the biotech disclosed in late June that the FDA sent a letter saying “deficiencies” in their application precluded a discussion around labeling or post-marketing commitments.

Andrew Allen, Gritstone Oncology CEO

A neoanti­gen pi­o­neer says its tech is work­ing great. So what wrecked the share price?

Gritstone Oncology was one of the original neoantigen upstarts, raising cash and planning to disrupt the immuno-oncology field with a bold new approach to fighting cancer with a new brand of vaccines.

On Monday, the crew in charge ran out a full display of what they’ve been seeing in a Phase I study. And everything seems to be working perfectly with one big exception: It didn’t significantly shrink tumors, let alone eradicate them.

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Janet Woodcock, CDER chief (AP Images)

More Warp Speed con­tracts com­ing, vac­cine pro­duc­tion to be­gin in 4-6 weeks — of­fi­cials

Operation Warp Speed has already handed out 4 of what they once said would be 3-5 major contracts to develop Covid-19 vaccines, but administration officials indicated Monday that more would be on their way.

“The slate is not closed,”  a senior HHS official said on a call with reporters. “We’ve invested in four … but the slate is not closed.”

At the same time, the official indicated that Warp Speed would continue to focus on three technologies: mRNA, viral vectors and protein subunits. That leaves the door open for a wide range of platforms, notably including both of Merck’s vaccine candidates — one of which has already received BARDA funding — and one of Sanofi’s candidates. It appears to preclude, though, the potential for Inovio and Vaxart, among certain other small developers that have hyped their ties to the Trump administration, to be included.