GSK/Tesaro PARP in­hibitor Ze­ju­la wins an­oth­er speedy re­view

Months af­ter part­ner Zai Lab $ZLAB scored an ac­cel­er­at­ed re­view for PARP ther­a­py Ze­ju­la in Chi­na, GSK has se­cured pri­or­i­ty US re­view for the drug, known chem­i­cal­ly as ni­ra­parib, to ex­pand the use of the drug in pa­tients with ad­vanced ovar­i­an can­cer.

GSK $GSK, which shelled out just over $5 bil­lion to swal­low Ze­ju­la-de­vel­op­er Tesaro, said the FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24.

The drug was sub­mit­ted for re­view in pa­tients with ad­vanced ovar­i­an, fal­lop­i­an tube, or pri­ma­ry peri­toneal can­cer pa­tients with or with­out BR­CA mu­ta­tions who have been treat­ed with three or more pri­or chemother­a­py reg­i­mens based on da­ta from the QUADRA tri­al, which showed 13 (28%) of 47 pa­tients in the pri­ma­ry ef­fi­ca­cy pop­u­la­tion achieved an over­all re­sponse — the best re­sponse record­ed from the start of the treat­ment un­til the dis­ease pro­gress­es — as per RE­CIST (Re­sponse Eval­u­a­tion Cri­te­ria in Sol­id Tu­mors) method­ol­o­gy.

Mary Lynne Hed­ley

“The re­sults of the QUADRA study demon­strate that ZE­JU­LA is ac­tive as a late-line treat­ment for pa­tients be­yond those with BR­CA mu­ta­tions,” said Tesaro’s COO Mary Lynne Hed­ley in a state­ment.

Ovar­i­an can­cer is the most com­mon cause of gy­nae­co­log­i­cal can­cer death in the Unit­ed States. Al­though most pa­tients with ad­vanced ovar­i­an can­cer re­spond to ini­tial ther­a­py, 70% will re­lapse and ul­ti­mate­ly suc­cumb to their dis­ease. Late-line treat­ment op­tions for pa­tients with ovar­i­an can­cer are few, with the pro­por­tion of pa­tients achiev­ing an over­all re­sponse typ­i­cal­ly less than 10%, and me­di­an over­all sur­vival af­ter third-line ther­a­py of 5–9 months, re­searchers not­ed in Lancet On­col­o­gy.

The drug, which com­petes against As­traZeneca’s $AZN Lyn­parza as well as Clo­vis’ $CLVS Rubra­ca, is al­ready ap­proved as a first­line main­te­nance treat­ment of women with re­cur­rent ovar­i­an, fal­lop­i­an tube, or pri­ma­ry peri­toneal can­cer when their can­cer comes back.

The once-dai­ly Ze­ju­la is cur­rent­ly be­ing eval­u­at­ed in three piv­otal tri­als, in­clud­ing a late-stage tri­al in pa­tients with first-line ovar­i­an can­cer (the PRI­MA tri­al), a Phase III tri­al for the treat­ment of pa­tients with germline BR­CA-mu­tat­ed, metasta­t­ic breast can­cer (the BRA­VO tri­al), and a reg­is­tra­tional Phase II treat­ment tri­al in pa­tients with ovar­i­an can­cer (the QUADRA tri­al). Part­ner J&J $JNJ is test­ing the use of the drug in prostate can­cer. Mean­while, a num­ber of com­bi­na­tion tri­als are al­so un­der­way, in­clud­ing test­ing the use of Ze­ju­la and Mer­ck’s $MRK key­stone check­point in­hibitor Keytru­da in breast and ovar­i­an can­cer.

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.

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 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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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.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: 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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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.

Covid-19 roundup: An mR­NA play­er gets a boost out of the lat­est round of an­i­mal da­ta; Phase­Bio pulls the plug on treat­ment tri­al

The big tell for CureVac $CVAC is coming up with a looming early-stage readout on their mRNA Covid-19 vaccine in the clinic. But for now they’ll make do with an upbeat assessment on the preclinical animal data they used to get into the clinic.

Researchers for the German biotech say they got the high antibody titers and T cell activation they were looking for, lining up a hamster challenge to demonstrate — in a simple model — that the vaccine could protect the furry creatures. Like the other mRNA vaccines, the drug sends instructions to spur cells to decorate themselves with the distinctive spike on the virus to elicit an immune response.