FDA ac­cepts Lex­i­con's heart fail­ure re­sub­mis­sion; Bris­tol My­ers buys out mava­camten roy­al­ties

Just two weeks af­ter the FDA again de­nied Lex­i­con Phar­ma­ceu­ti­cals’ at­tempt to over­turn a CRL in type 1 di­a­betes, the Texas biotech said the agency has ac­cept­ed the drug’s re­sub­mis­sion for heart fail­ure.

A de­ci­sion date is slat­ed for some­time in May 2023. The reg­u­la­tor will con­sid­er two Phase III tri­als, one in which wors­en­ing heart fail­ure sent type 2 di­a­betes pa­tients in­to the hos­pi­tal (SOLOIST-WHF) and an­oth­er that in­cludes pa­tients with type 2 di­a­betes, chron­ic kid­ney dis­ease and risks for car­dio­vas­cu­lar dis­ease (SCORED). In to­tal, so­tagliflozin has been test­ed in 14 Phase III tri­als in about 20,000 peo­ple.

The oral SGLT2 drug has been through the wringer in re­cent years, with the FDA clear­ly spelling out its re­jec­tion of the drug in type 1 di­a­betes treat­ment in March 2019, which at the time in­clud­ed part­ner Sanofi. The Big Phar­ma ex­it­ed the scene a few months lat­er, tak­ing a $260 mil­lion hit.

De­spite fal­ter­ing in the US, Lex­i­con snagged Eu­ro­pean Union ap­proval of the drug in adults with type 1 di­a­betes in April 2019 and is sold as Zyn­quista.

The ac­cept­ed fil­ing trig­gers Lex­i­con’s abil­i­ty to reel in an­oth­er $25 mil­lion through its loan fa­cil­i­ty with Ox­ford Fi­nance. The biotech got the first $25 mil­lion tranche in March, and can re­ceive an­oth­er $50 mil­lion next year if the drug is ap­proved for heart fail­ure. A fi­nal $50 mil­lion can be used there­after.

Bris­tol My­ers buys out fu­ture roy­al­ty oblig­a­tions on mava­camten

Three months af­ter land­ing a big vic­to­ry on mava­camten, Bris­tol My­ers Squibb has now bought out fu­ture roy­al­ty oblig­a­tions on the drug for $295 mil­lion, the Big Phar­ma re­vealed in an SEC fil­ing.

At the heart of the $13 bil­lion MyoKar­dia ac­qui­si­tion in 2020, mava­camten se­cured an FDA nod in April for treat­ing a con­di­tion in which blood is blocked from flow­ing out of the heart.

MyoKar­dia and Cy­to­ki­net­ics co-dis­cov­ered the drug, with the lat­ter now in late-stage tri­als for its own myosin in­hibitor, afi­camten. Roy­al­ty Phar­ma dished out $50 mil­lion up­front in Jan­u­ary and is will­ing to hand over an­oth­er $100 mil­lion in biobucks for roy­al­ty on the drug.

De­spite flunk­ing Phase III, In­flaRx takes Covid-19 drug to FDA

Ger­man biotech In­flaRx said it will sub­mit a re­quest to the FDA for emer­gency use au­tho­riza­tion of its Covid-19 mon­o­clon­al an­ti­body.

The mAb, named vilo­be­limab, failed a late-stage study a few months ago in crit­i­cal­ly ill pa­tients who were on me­chan­i­cal ven­ti­la­tion for Covid-19. De­spite the tri­al flop, the com­pa­ny met with the FDA, and In­flaRx said the Type B meet­ing led to “en­cour­ag­ing in­ter­ac­tions” with the agency.

“With emerg­ing COVID-19 vari­ants and cas­es and hos­pi­tal­iza­tions again on the rise, there re­mains an ur­gent need for new treat­ment op­tions, es­pe­cial­ly for the sick­est pa­tients who suf­fer from an in­flam­ma­to­ry re­sponse, lead­ing to or­gan fail­ure. Our con­struc­tive in­ter­ac­tions with the FDA and the help­ful guid­ance they pro­vid­ed have en­cour­aged us to move for­ward with ap­ply­ing for EUA for vilo­be­limab in crit­i­cal­ly ill COVID-19 pa­tients,” CEO Niels Riede­mann said in a state­ment.

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Ad­dress­ing the ‘Ca­pac­i­ty Crunch’ with a Scal­able Plat­form Process Ap­proach

The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.

A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

One of the cool things about adding EndpointsPharma to the daily roster is that my colleagues can now dedicate time to tracking quarterly updates and tuning into calls with Big Pharma companies. Check out their dispatch from the Q2 earnings below.

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Albert Bourla, Pfizer CEO (Laurent Gillieron/Keystone via AP)

Break­ing: Pfiz­er in hot pur­suit of a $5B buy­out of Glob­al Blood Ther­a­peu­tics — re­port

Pfizer CEO Albert Bourla has vowed to leave no stone unturned in the search for new biotech deals, and the BD team is not letting him down.

The Wall Street Journal reported today that Pfizer is in the final stages of acquiring Global Blood Therapeutics for $5 billion. According to the Journal report, though, Pfizer is not the only buyer at the deal table and while the pharma giant may be close to clinching it, there are no guarantees it will continue.

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Bob Bradway, Amgen CEO (Justin Kase Conder/AP Images for Amgen)

UP­DAT­ED: Am­gen chief Brad­way nabs a rare dis­ease play­er in $4B buy­out as the M&A tem­po ac­cel­er­ates

Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.

Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

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George Yancopoulos, Regeneron president and CSO (Brendan McDermid/Reuters/Alamy)

George Yan­copou­los says he's on the trail of the holy grail: ‘This could rep­re­sent the next break­through for im­munother­a­py’

Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.

While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.

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(AP Photo/Richard Vogel, File)

US de­clares mon­key­pox a na­tion­al health emer­gency, as new drug­mak­ers con­sid­er en­ter­ing vac­cine race

Rising monkeypox cases have put the US on high alert as it announces a national health emergency, which grants the government more power in its response.

The news comes as Bavarian Nordic continues to fill orders for its Jynneos vaccine and other companies – including Moderna – consider jumping into the vaccine race. Meanwhile, the New York Times reports that the US has allowed around 20 million doses of smallpox vaccine in its stockpile to expire.

Vlad Coric, Biohaven CEO

Bio­haven touts surge in Nurtec sales ahead of Pfiz­er takeover

Forget buyer’s remorse, Pfizer is likely feeling pretty good about its $11.6 billion Biohaven takeover deal following reports of a 57% sales boost for migraine med Nurtec.

Biohaven reported in Q2 results on Friday that it’s cleared the necessary antitrust hurdles to move forward with the sale of its calcitonin gene-related peptide (CGRP) assets to Pfizer. However, because the company is “focused on workstreams related to the closing” of the deal, it did not host a call with analysts and investors.

Pharma ads are showing up on cooler screens at retail pharmacies, including Walgreens and CVS, under a new OptimizeRx deal (OptimizeRx)

Phar­ma brands chill in the phar­ma­cy re­tail aisle with new style ads on re­frig­er­a­tion screens

Want a prescription drug with that soda? While not directly possible, ads for pharma brands now running on beverage and snack cooler screens at pharmacy retailers may at least inspire customers to think about it.

OptimizeRx is hooking up with Cooler Screens media company to bring prescription drug advertising to refrigerator front doors at pharmacies including Walgreens, CVS and Kroger.

The “point of dispense” ads show a full-door image on the cooler doors when a shopper is 12 feet away, but shrinks down to a smaller banner-sized ad so that the refrigerator contents can be seen when a person gets closer. The doors — which have to be specially installed by Cooler Screens — can detect when a person is nearby, how long a person “dwells” in front of the ad and if they do or don’t open the door.

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