TG Ther­a­peu­tics scores pri­or­i­ty re­view for PI3Kδ in­hibitor; Gala­pa­gos search­es for IBD tar­gets in new pact

TG Ther­a­peu­tics’ pitch for um­bral­is­ib as a safer PI3Kδ in­hibitor has con­vinced the FDA to put it on the fast lane.

Reg­u­la­tors have ac­cept­ed the NDA for the once-dai­ly drug, a dual in­hibitor of PI3K-delta and CK1-ep­silon first aimed at two in­di­ca­tions: mar­gin­al zone lym­phoma pa­tients who have re­ceived at least one an­ti-CD20-based reg­i­men, and fol­lic­u­lar lym­phoma pa­tients who have gone through at least two lines of sys­temic ther­a­pies.

As the MZL use was des­ig­nat­ed “break­through” ther­a­py, the FDA is al­so grant­i­ng pri­or­i­ty re­view and set­ting a PDU­FA date of Feb­ru­ary 15, 2021. The FL de­ci­sion has a dead­line in June.

“The FDA al­so no­ti­fied the Com­pa­ny that it is not cur­rent­ly plan­ning to hold an ad­vi­so­ry com­mit­tee meet­ing to dis­cuss this ap­pli­ca­tion,” TG Ther­a­peu­tics wrote, usu­al­ly a pos­i­tive sign.

The da­ta pack­age came most­ly from the UNI­TY-NHL Phase IIb tri­al, the com­pa­ny added, where both the MZL and FL co­horts met the 40%-50% tar­get on over­all re­sponse rate, the pri­ma­ry end­point. The sin­gle-agent study was specif­i­cal­ly de­signed to mod­el the stud­ies that brought ac­cel­er­at­ed FDA ap­proval for oth­er PI3Kδ drugs, CEO Michael Weiss pre­vi­ous­ly not­ed.

Gala­pa­gos inks tar­get dis­cov­ery deal fo­cused on in­flam­ma­to­ry bow­el dis­ease

Tak­ing the pre­ci­sion med­i­cine ap­proach on au­toim­mune dis­eases, Gala­pa­gos has en­list­ed Boston-based Sci­pher Med­i­cine to iden­ti­fy new tar­gets and path­ways that can lead to new treat­ments for in­flam­ma­to­ry bow­el dis­ease.

Gala­pa­gos — whose own drugs for in­flam­ma­tion and fi­bro­sis have in­spired multi­bil­lion-dol­lar deals from Gilead — has the op­tion to move up the tar­gets in­to drug dis­cov­ery and de­vel­op­ment. Sci­pher re­tains the rights to opt in, on top of re­ceiv­ing up­front and mile­stone pay­ments.

“Our break­through ap­proach com­bines ex­ten­sive pa­tient-lev­el mol­e­c­u­lar da­ta with AI meth­ods to dis­cov­er nov­el tar­gets with the po­ten­tial to treat spe­cif­ic groups of pa­tients, in­clud­ing those not re­spond­ing to ther­a­pies cur­rent­ly avail­able,” said Alif Saleh, CEO of Sci­pher.

Red­Hill finds a man­u­fac­tur­er for As­traZeneca drug — which is al­so fund­ing R&D work

Red­Hill Bio­phar­ma has tied a few more knots with its Dublin-based part­ners at Cos­mo, lin­ing up a man­u­fac­tur­ing deal as well as some cash for two ex­per­i­men­tal drugs.

Three drugs are cov­ered in the deal: Movan­tik, the treat­ment for opi­oid-in­duced con­sti­pa­tion Red­Hill re­cent­ly li­censed from As­traZeneca; RHB-204, an oral com­bi­na­tion of clar­ithromycin, ri­fabutin, and clo­faz­imine de­signed to tack­le pul­monary non­tu­ber­cu­lous my­cobac­te­ria (NTM) in­fec­tions; and a yet un­named drug for H. py­lori in­fec­tions.

Cos­mo has agreed to fund the piv­otal Phase III study for RHB-204, with $5 mil­lion due up­on sign­ing and $7 mil­lion di­vid­ed be­tween two mile­stone pay­ments, in ex­change for 15% roy­al­ty pay­ments. It’s al­so pay­ing $7 mil­lion up­front to li­cense Eu­ro­pean rights to the un­named ex­per­i­men­tal com­pound and com­mit­ting $2 mil­lion up­on ap­proval, while foot­ing 30% of the clin­i­cal tri­al costs in both the US and Eu­rope. Red­Hill will be en­ti­tled to 30% roy­al­ties.

As part of the agree­ment, Red­Hill is al­so hir­ing Cos­mo to be the sole man­u­fac­tur­er of both these drugs as well as Movan­tik. The Is­raeli biotech is pay­ing €5.5 mil­lion for the tech trans­fer, for­mu­la­tion and de­vel­op­ment work.

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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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.

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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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.

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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