FDA rushed to ap­prove new drugs in 2019, some­times way ahead of sched­ule. Could that be dan­ger­ous?

While the ros­ter of 48 drugs (plus 4 no­table bi­o­log­ics) the FDA ap­proved in 2019 didn’t break any records with the sheer num­ber it was re­mark­able in one as­pect: The agency reached deep in­to a group of ther­a­pies they didn’t need to make a de­ci­sion on un­til lat­er this year, in some cas­es speed­ing drugs up by months ahead of even pri­or­i­ty re­view dead­lines.

On the sur­face, that’s great news for drug­mak­ers and the pa­tients they hope to help. But ac­cord­ing to a re­cent study on rushed ap­provals, it could al­so spell safe­ty trou­bles down the road.

Lau­ren Co­hen

Back in Ju­ly, a trio of econ­o­mists at NBER crunched the da­ta on drugs OK’d be­fore sup­posed dead­lines such as the end of months, years or be­fore hol­i­days and found that treat­ments ap­proved un­der these ap­par­ent “desk-clear­ing” ef­forts — in which reg­u­la­tors felt the pres­sure of in­for­mal per­for­mance bench­marks — are more like­ly to be as­so­ci­at­ed with post­mar­ket safe­ty is­sues.

“We see about twice as many ad­verse ef­fects,” Lau­ren Co­hen, a pro­fes­sor of fi­nance and en­tre­pre­neur­ial man­age­ment at Har­vard Busi­ness School and one of the au­thors, told the Wall Street Jour­nal.

Co­hen and his col­leagues, Umit Gu­run of the Uni­ver­si­ty of Texas at Dal­las and Danielle Li of MIT, con­sid­ered on­ly the surge of ap­provals in De­cem­ber, at the end of each month and be­fore hol­i­days such as Thanks­giv­ing.

In 2019 sev­en out of 48 nov­el chem­i­cal en­ti­ties were ap­proved in De­cem­ber, large­ly in line with the 15% pro­por­tion that the re­searchers not­ed in pre­vi­ous years. But no­tably, 21 OKs came through in Q4, ac­count­ing for 43% of the whole crop.

FDA spokesper­son Nathan Arnold ac­knowl­edged the De­cem­ber jump pat­tern but told the Jour­nal that its share has ac­tu­al­ly di­min­ished from the 1980s.

“While we can­not speak di­rect­ly to the re­sults on in­for­mal dead­lines high­light­ed in this study, FDA has—on mul­ti­ple oc­ca­sions—in­ves­ti­gat­ed a close­ly re­lat­ed is­sue, which is the re­la­tion­ship be­tween for­mal PDU­FA dead­lines and post­mar­ket safe­ty of drugs,” Arnold added. “We have not found ev­i­dence of such a re­la­tion­ship.”

Drugs that ap­peared to have been ap­proved with lit­tle heed of the PDU­FA dead­lines might be an­oth­er is­sue. Ver­tex’s Trikaf­ta (for cys­tic fi­bro­sis), BeiGene’s Brukin­sa (for man­tle cell lym­phoma), No­var­tis’ Adakveo and Glob­al Blood Ther­a­peu­tics’ Oxbry­ta (for sick­le cell dis­ease), as well as Al­ny­lam’s Givlaari (for acute he­pat­ic por­phyr­ia ) and As­traZeneca/Dai­ichi Sankyo’s En­her­tu (for HER+ breast can­cer) all be­longed to that group.

Co­hen, Gu­run and Li pre­scribed a so­lu­tion for the safe­ty con­cerns they spot­ted:

A po­ten­tial pol­i­cy re­sponse to the pat­terns we doc­u­ment is to more care­ful­ly scru­ti­nize drugs that are ap­proved dur­ing such out­put surges. Reg­u­la­tors could, for in­stance, re­view end-of-year de­ci­sions in the fol­low­ing year be­fore pro­vid­ing an ul­ti­mate ap­proval (or lack there­of).

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.