News brief­ing: Ot­su­ka’s $886M can­cer drug is now a 3-time los­er in PhI­II; Keytru­da ap­proved for clas­si­cal Hodgkin lym­phoma

Two years af­ter Ot­su­ka un­veiled a flop in their first Phase III tri­al of guadecitabine as a front­line treat­ment, they’re back with 2 more piv­otal set­backs. But re­searchers are leav­ing the door ajar for more stud­ies ahead.

The com­pa­ny’s Cal­i­for­nia sub­sidiary As­tex, ac­quired for $886 mil­lion back in 2013, says that AS­TRAL-2 and AS­TRAL-3 — which re­cruit­ed pa­tients who had been pre­vi­ous­ly treat­ed for AML and MDS/CMML — couldn’t beat the mark for sta­tis­ti­cal sig­nif­i­cance.

As­tex pres­i­dent and CMO Mo­ham­mad Az­ab not­ed:

The AS­TRAL stud­ies gen­er­at­ed for the med­ical com­mu­ni­ty one of the largest bod­ies of clin­i­cal and ge­net­ic da­ta from prospec­tive ran­dom­ized stud­ies us­ing hy­pomethy­lat­ing agent treat­ment. Guadecitabine was as­so­ci­at­ed with im­proved out­comes in cer­tain sub­groups, but that needs to be val­i­dat­ed by ad­di­tion­al stud­ies.

The ac­tu­al da­ta will be pre­sent­ed at a sci­en­tif­ic con­fer­ence to be se­lect­ed lat­er.

Ot­su­ka was pulled in by promis­ing mid-stage da­ta on the drug, con­fi­dent that it had the kind of proof-of-con­cept da­ta in hu­mans that jus­ti­fied an am­bi­tious Phase III pro­gram. The drug is billed as a next-gen­er­a­tion DNA hy­pomethy­lat­ing agent, but it ini­tial­ly failed against a group of pa­tients who re­ceived one of sev­er­al al­ter­na­tive ther­a­pies: azac­i­ti­dine, decitabine, or low dose cy­tara­bine. — John Car­roll

Keytru­da gets an­oth­er ap­proval, this time in clas­si­cal Hodgkin lym­phoma

The FDA has ap­proved an ex­pand­ed in­di­ca­tion for Mer­ck’s best-sell­er Keytru­da drug.

Adults with re­lapsed or re­frac­to­ry clas­si­cal Hodgkin lym­phoma are now able to be treat­ed with Keytru­da as a monother­a­py, Mer­ck said Thurs­day. The ap­proval is based on the re­sults from a Phase III study that showed Keytru­da re­duced the risk of dis­ease pro­gres­sion or death by 35% com­pared to Take­da’s Ad­cetris.

Ad­di­tion­al­ly, Keytru­da re­ceived an up­dat­ed pe­di­atric in­di­ca­tion for the treat­ment re­frac­to­ry cHL, or cHL that has re­lapsed af­ter two or more lines of ther­a­py.

Mer­ck’s Phase III study was a ran­dom­ized, open-la­bel, ac­tive-con­trolled tri­al con­duct­ed in 304 pa­tients, who re­ceived ei­ther Keytru­da or Ad­cetris through an IV once every three weeks. Treat­ment was con­tin­ued un­til un­ac­cept­able tox­i­c­i­ty, dis­ease pro­gres­sion or un­til pa­tients reached 35 dos­ing cy­cles af­ter about two years.

Keytru­da is al­ready ap­proved to treat a va­ri­ety of can­cers, in­clud­ing melanoma, non-small cell lung can­cer, small cell lung can­cer, and head and neck squa­mous cell can­cer, among oth­ers. The drug net­ted Mer­ck more than $6.6 bil­lion in sales through the first half of 2020, and peak an­nu­al sales could hit $24.3 bil­lion by 2026, ac­cord­ing to Eval­u­atePhar­ma. — Max Gel­man

Pe­ter Thiel-backed skin mi­cro­bio­me biotech nets $17 mil­lion Se­ries B

Az­i­tra, a biotech once fund­ed by bil­lion­aire Pe­ter Thiel, has pulled in an­oth­er $17 mil­lion, in­clud­ing $8 mil­lion from its part­ners at Leaps by Bay­er.

The com­pa­nies part­nered up ear­li­er this year to de­vel­op Az­i­tra’s skin mi­cro­bio­me prod­ucts, main­ly the biotech’s pro­pri­etary pan­el of Staphy­lo­coc­cus epi­der­midis strains to iden­ti­fy po­ten­tial treat­ments for skin con­di­tions and dis­eases. Thurs­day’s cash will build on that col­lab­o­ra­tion deal, which brings Az­i­tra’s to­tal mon­ey raised to $34 mil­lion when in­clud­ing the rest of the round af­ter an ex­tend­ed Se­ries A closed in late 2019.

Az­i­tra is fo­cus­ing its re­search in three spe­cif­ic ar­eas: con­sumer skin­care prod­ucts, bio-en­gi­neered skin dis­ease treat­ments and ad­vanced mi­cro­bio­me prod­ucts for mil­i­tary and pub­lic health ap­pli­ca­tions. Its lead can­di­date is a cream for can­cer pa­tients who de­vel­op rash­es while tak­ing EGFR ther­a­py, cur­rent­ly in Phase I tri­als. — Max Gel­man

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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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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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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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.

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.

News brief­ing: Gilead com­pletes $21B buy­out of Im­munomedics; In­nate re­ceives $50M mile­stone pay­ment from As­traZeneca

Gilead’s $21 billion mega-acquisition of Immunomedics is now officially complete, the companies announced Friday morning.

The full merger process took a little over a month, with Gilead and Immunomedics signing an agreement on Sept. 13. Gilead acquired all outstanding stock of Immunomedics for $88 per share, a 108% premium on the previous day’s closing price.

Gilead’s big prize was Trodelvy, approved in July for the treatment of metastatic triple-negative breast cancer. The drug also impressed at last month’s ESMO conference, reducing the risk of death by 52% in a Phase III study.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.