J&J jumps in­to gam­ma delta field with La­va col­lab­o­ra­tion; Myokar­dia guns for $605M raise

→ Jump­ing in­to the erupt­ing field of gam­ma delta T cells, J&J has signed a col­lab­o­ra­tion with La­va Ther­a­peu­tics. Like a se­ries of oth­er biotechs that have emerged in re­cent years, Nether­lands-based La­va de­vel­ops bis­pe­cif­ic an­ti­bod­ies that grab gam­ma delta T cells, which are in­volved in rec­og­niz­ing can­cers and oth­er types of anti­gens, to at­tack eva­sive tu­mors. Fi­nan­cial terms were not dis­closed.

→ Fol­low­ing pos­i­tive re­sults from a Phase III study on its lead drug, Myokar­dia is try­ing to raise $605 mil­lion in a stock of­fer­ing. The of­fer­ing comes af­ter the tri­al re­sults al­most dou­bled the biotech’s stock price from $61.09 to $120.16 as of this morn­ing, and with Myokar­dia prepar­ing to ask the FDA for ap­proval ear­ly next year. The drug treats hy­per­trophic car­diomy­opa­thy, a dis­or­der that in­ter­feres with the abil­i­ty of the heart to pump blood through the body. Peak sale es­ti­mates are for over $2 bil­lion.

→ The FDA has ap­proved Po­m­a­lyst – the thalido­mide-de­riv­a­tive de­vel­oped by Cel­gene for mul­ti­ple myelo­ma and now owned by Bris­tol My­ers Squibb – for AIDS-re­lat­ed and HIV-neg­a­tive Ka­posi’s Sar­co­ma. Bris­tol My­ers said this is the first new ap­proved treat­ment in over 20 years for Ka­posi’s Sar­co­ma and the first oral agent.

Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

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).

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 85,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 85,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

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

Covid-19 roundup: Philip Mor­ris-backed Med­ica­go be­gins dos­ing to­bac­co-based vac­cine; Af­ter re­ports of de­lays, gov­ern­ment dis­putes, Mod­er­na sets PhI­II launch

One of the dark horse candidates in the race to develop a new vaccine to fight Covid-19 has begun a Phase I trial of a new jab developed with tobacco leaves.

Philip Morris-backed Medicago, which has deals in place to use an adjuvant from GSK and another from Dynavax in their vaccine, dosed their first patients in Phase I on Monday. And a Phase II/III trial is slated to begin in October.

That’s around the same time that the leaders in this race plan to start seeking emergency use approvals for the first round of vaccines.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 85,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 85,200+ biopharma pros reading Endpoints daily — and it's free.