Sanofi un­der for­mal in­ves­ti­ga­tion for De­pakine al­le­ga­tions; Beam li­cens­es CAR-T tech from Ox­ford Bio­med­ica

Sanofi is fac­ing a for­mal in­ves­ti­ga­tion on manslaugh­ter charges, due to ac­cu­sa­tions that its epilep­sy drug De­pakine caused birth mal­func­tions and slow neu­ro­log­i­cal de­vel­op­ment when tak­en dur­ing preg­nan­cy.

The French phar­ma was for­mal­ly charged in Feb­ru­ary, years af­ter ev­i­dence sur­faced that the drug, sodi­um val­proate, posed neu­rode­vel­op­men­tal risks. Sodi­um val­proate first hit the mar­ket in 1967 for the treat­ment of epilep­sy and bipo­lar dis­or­der, and is cur­rent­ly pre­scribed in more than 100 coun­tries.

Be­tween 2006 and 2014, France’s so­cial af­fairs in­spec­tion agency IGAF be­lieves that 425 to 450 ba­bies ex­posed to the drug suf­fered con­gen­i­tal birth de­fects or were still­born, ac­cord­ing to a Reuters re­port. The EMA re-eval­u­at­ed the drug in 2014, and the fol­low­ing year rec­om­men­da­tions were en­forced that prac­ti­tion­ers not pre­scribe the treat­ment for women of child­bear­ing age.

Sanofi de­nied the al­le­ga­tions. “All these el­e­ments do not in any way pre­judge the re­spon­si­bil­i­ty of the com­pa­ny,” the com­pa­ny wrote in a state­ment to Reuters.

The As­so­ci­a­tion for the as­sis­tance of par­ents of chil­dren suf­fer­ing from an­ti-con­vul­sant syn­drome (APE­SAC) ini­ti­at­ed the crim­i­nal in­ves­ti­ga­tion. To be­gin a for­mal in­ves­ti­ga­tion, an in­ves­tiga­tive judge had to deem the case se­ri­ous enough to war­rant a fur­ther look, ac­cord­ing to Reuters. — Nicole De­Feud­is

Beam inks CAR-T deal with Ox­ford Bio­med­ica

The gene edit­ing ex­perts at Beam Ther­a­peu­tics have added Ox­ford Bio­med­ica’s LentiVec­tor plat­form to its CART- reper­toire. The li­cense gives Beam ac­cess to the same tech­nol­o­gy No­var­tis and Juno have lever­aged to man­u­fac­ture their cell ther­a­pies.

By car­ry­ing out tar­get­ed base changes, Beam promis­es to knock out the ex­pres­sion of mul­ti­ple cell sur­face tar­gets on T cells at once.

Un­der the new deal, Ox­ford Bio­med­ica was paid an undis­closed up­front sum to kick off de­vel­op­ment and man­u­fac­tur­ing for one pre­clin­i­cal pro­gram. More pay­ments and mile­stones are to come. — Am­ber Tong

Red­Hill grabs full com­mer­cial­iza­tion rights to ex-As­traZeneca drug

When Red­Hill Bio­phar­ma bought Movan­tik, a treat­ment for opi­oid in­duced con­sti­pa­tion, from As­traZeneca in April, it al­so in­her­it­ed a co-com­mer­cial­iza­tion al­liance with Dai­ichi Sankyo. It’s now re­vised the deal to grab ex­clu­sive re­spon­si­bil­i­ty to mar­ket the drug and pay roy­al­ties to the Japan­ese phar­ma in­stead.

“The clos­ing of this new agree­ment with Dai­ichi Sankyo will al­low us to have full con­trol over brand strat­e­gy and com­mer­cial­iza­tion ac­tiv­i­ties for Movan­tik in the U.S. while al­so in­creas­ing our mar­gins,” Rick Scrug­gs, Red­Hill’s chief com­mer­cial of­fi­cer and head of US op­er­a­tions, said in a state­ment.

In ad­di­tion to a mid-teen roy­al­ty rate on net sales, Dai­ichi is en­ti­tled to three lump sums pay­ments from 2021 to 2023. They al­so re­ceived 283,387 Amer­i­can De­posi­tary Shares of Red­Hill’s stock $RDHL as part of a sub­scrip­tion agree­ment.

Movan­tik, a pe­riph­er­al­ly act­ing μ-opi­oid re­cep­tor an­tag­o­nist, gen­er­at­ed $96 mil­lion in the US last year. Red­Hill ac­quired glob­al rights to the drug ex­clud­ing Eu­rope, Cana­da and Is­rael. — Am­ber Tong

PPD en­ters three-year agree­ment with Pfiz­er 

Wilm­ing­ton, NC-based con­tract re­search or­ga­ni­za­tion PPD has inked a three-year agree­ment to help grow Pfiz­er’s port­fo­lio in “mul­ti­ple ther­a­peu­tic ar­eas.” The deal builds on the com­pa­nies’ cur­rent re­la­tion­ship, in which PPD of­fers lab ser­vices and clin­i­cal de­vel­op­ment ex­per­tise to fa­cil­i­tate drug de­vel­op­ment. PPD and Pfiz­er are cur­rent­ly keep­ing the fi­nan­cial terms un­der wraps.

Pfiz­er has 23 drugs in Phase III de­vel­op­ment, rang­ing from vac­cines to on­col­o­gy. It’s cur­rent­ly work­ing with BioN­Tech on four Covid-19 vac­cine pro­grams, two of which are be­ing fast tracked by the FDA. Their can­di­date BNT162b2 just en­rolled in a Phase II/III study, set­ting it up for reg­u­la­to­ry re­view as soon as Oc­to­ber. — Nicole De­Feud­is

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

Embattled CDC director Robert Redfield (AP Images)

Covid-19 roundup: CDC ad­vi­so­ry com­mit­tee de­lays pri­or­i­ty dis­tri­b­u­tion vote; EU re­port­ed­ly in­dem­ni­fy­ing vac­cine mak­ers

A federal committee that advises the CDC was expected to hold a vote Tuesday on a plan regarding the distribution for initial doses of approved Covid-19 vaccines. But that vote has been scrapped.

The Advisory Committee on Immunization Practices, or ACIP, won’t be voting until the committee members learn more about which vaccines become available first, the Wall Street Journal reported. The vote could potentially wait until a specific vaccine is authorized before recommending how to dole out the first doses.

Anthony Coyle (Pfizer via Youtube)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-Suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.