FDA gives Mer­ck ‘re­al-time’ OK for Keytru­da, of­fer­ing a peek at the lat­est ex­ten­sion of the agen­cy's da­ta ex­press high­way

The FDA doesn’t al­ways wait for a mar­ket­ing ap­pli­ca­tion to come in be­fore it be­gins its re­view these days. And that suits Mer­ck just fine.

The fed­er­al agency has stamped a full ap­proval — fol­low­ing last year’s con­di­tion­al OK — on Mer­ck’s mega-block­buster Keytru­da for front­line use in a chemo com­bo for fight­ing non-squa­mous non-small cell lung can­cer. Reg­u­la­tors based their de­ci­sion on the da­ta set from KEYNOTE-189, put on dis­play last April, one in a se­ries of late-stage tri­als that Mer­ck has been rolling out to grab the lead on the PD-1/L1 mar­ket from a group of tough com­peti­tors at Bris­tol-My­ers Squibb.

The green light is any­thing but un­ex­pect­ed. But it is note­wor­thy for rea­sons that the en­tire in­dus­try should be pay­ing at­ten­tion to.

Roger Perl­mut­ter

What dis­tin­guish­es this par­tic­u­lar OK is the tim­ing. FDA com­mis­sion­er Scott Got­tlieb has been push­ing a new pi­lot pro­gram called re­al-time on­col­o­gy re­views, where reg­u­la­tors start their work ahead of a for­mal ap­pli­ca­tion. The first re­al-time OK went to No­var­tis a few weeks ago for two com­bi­na­tions us­ing Kisqali against breast can­cer. And top-tier drug de­vel­op­ers are find­ing that in­stead of just be­ing greet­ed with an open door, some­times the door is be­ing tak­en off the hinges en­tire­ly.

In this case they were deal­ing with a haz­ard ra­tio of 0.49, mark­ing a 51% drop in the risk of death for pa­tients tak­ing the com­bo. There’s still no me­di­an sur­vival da­ta for the com­bo avail­able, but the matchup of the Keytru­da/chemo ap­proach ver­sus chemo alone pro­vid­ed a clear set of ad­van­tages for Mer­ck.

  • The me­di­an pro­gres­sion-free sur­vival rate was 8.8 months for the com­bo, 4.9 months for chemo alone.
  • The over­all re­sponse rate was 48% com­pared to 19% in the con­trol.
  • Me­di­an re­sponse du­ra­tion hit 11.2 months for the Keytru­da arm and 7.8 months for con­trol.

These re­al-time re­views now on of­fer at the FDA look to ex­tend the da­ta ex­press high­way that’s been built for the agency’s break­through drug des­ig­na­tion and oth­er hur­ry-up cam­paigns that have trans­formed the speed and de­sign of on­col­o­gy stud­ies over the past few years. In this case the FDA wants to re­ly en­tire­ly on US stud­ies, ex­clud­ing any­thing out­side the bor­ders, while pre­fer­ring drugs that are de­liv­er­ing clear ben­e­fits and are be­ing test­ed on some ob­vi­ous end­points. And they don’t want any for­mu­la­tion changes to pon­der when mak­ing a snap call.

The FDA is tak­ing no chances with Keytru­da, a land­mark drug by any de­f­i­n­i­tion that is be­ing pushed through hun­dreds of com­bi­na­tion stud­ies and new ap­proach­es. Got­tlieb is like­ly to earn even more de­vo­tion from the in­dus­try’s lead­ers for fol­low­ing through on im­prov­ing the FDA’s re­spon­sive­ness. And Mer­ck is hap­py to have the fresh set of brag­ging rights to­day.

“Keytru­da is rapid­ly be­com­ing a foun­da­tion for the treat­ment of ap­pro­pri­ate pa­tients with metasta­t­ic non-small cell lung can­cer,” said Mer­ck R&D chief Roger Perl­mut­ter. “To­day’s ap­proval of the ex­pand­ed la­bel for Keytru­da based on da­ta from the KEYNOTE-189 tri­al is an im­por­tant mile­stone.”


President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Fangliang Zhang, AP Images

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.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

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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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.

RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.