De­fy­ing the odds, Pox­el plots a PhI­II course for its Type 2 di­a­betes med and shares surge on new da­ta

Thomas Kuhn, Pox­el

Pox­el is try­ing to mas­ter one of the tough­est acts in biotech: De­vel­op a new drug for Type 2 di­a­betes and win a reg­u­la­to­ry ap­proval — on its own.

It’s not easy, in the way that nav­i­gat­ing a mine field isn’t easy. But they are mov­ing ahead and say they have a very man­age­able game plan for get­ting through Phase III in Japan while look­ing for part­ners that can do much of the heavy lift­ing in Eu­rope and the US.

About 10 months ago, France’s Pox­el hit the brakes on a planned move to Nas­daq, un­able to gen­er­ate much en­thu­si­asm in a tough mar­ket. In­stead, ex­ecs raised some ad­di­tion­al cash from its pub­lic list­ing in Eu­rope to fund a Phase IIb tri­al of its lead ther­a­py for Type 2 di­a­betes that would hope­ful­ly pave the way to a launch in Japan.

To­day, the com­pa­ny says it scored the pri­ma­ry and at least one sec­ondary end­point for imeglim­in, track­ing he­mo­glo­bin A1c re­duc­tions of 0.52%, 0.94% and 1.00% for the 500 mg, 1000 mg and 1500 mg dose twice-dai­ly. Sev­en per­cent or less is the key num­ber for glycemic con­trol. The drug al­so trig­gered a sta­tis­ti­cal­ly sig­nif­i­cant drop in fast­ing plas­ma glu­cose. And now the com­pa­ny plans to set the stage for a piv­otal pro­gram in Japan that can launch lat­er this year — pro­vid­ed Japan­ese reg­u­la­tors sign off.

That was wel­come news to its in­vestors, who bid up shares {$POX­EL: PA} by more than 35%. But the biotech has a long way to go be­fore it can de­clare vic­to­ry.

Late-stage test­ing for a di­a­betes drug, which would be steer­ing in­to an in­tense­ly com­pet­i­tive mar­ket among a hand­ful of gi­ants in the field, is nei­ther easy nor cheap. Big Phase III bud­gets need­ed to suf­fi­cient­ly test a mass mar­ket drug have scut­tled ear­li­er con­tenders like Phe­nomix (RIP: 2010). MannKind strug­gled its way through the clin­ic to end up with an in­haled in­sulin that di­a­bet­ics — and af­ter Sanofi washed its hands of a deal, the rest of the play­ers — have shunned. And none of that has made di­a­betes par­tic­u­lar­ly pop­u­lar with VCs or gen­er­al­ist in­vestors.

But Pox­el ex­ecs say they have found a path that can work.

“For a biotech com­pa­ny like this ac­cess­ing this mar­ket (Japan) is much eas­i­er,” CEO Thomas Kuhn tells me. While most glob­al bio­phar­ma com­pa­nies pre­fer to hand off their drugs to Japan­ese part­ners bet­ter equipped to work with the coun­try’s reg­u­la­tors, Pox­el cre­at­ed their own lo­cal team in the Asian coun­try, and Kuhn says the coun­try low­ered reg­u­la­to­ry bar­ri­ers to out­side com­pa­nies a cou­ple of years ago, mak­ing it pos­si­ble for Pox­el to pi­o­neer this on their own.

But there are even more im­por­tant as­pects that make Japan one of the few places on the plan­et Pox­el could ex­pect to do this on their own. One is that reg­u­la­tors are open to new drugs in the field, Kuhn ar­gues, sen­si­tive to the is­sues Japan­ese pa­tients have with met­formin. Pox­el could get an ap­proval on a slate of three stud­ies in­volv­ing about 1,000 di­a­betes pa­tients in Japan, says the CEO, while it would ul­ti­mate­ly take some 7,000 pa­tients in the US. Japan­ese pa­tients are al­so much lean­er than the av­er­age US pa­tient, avoid­ing the ne­ces­si­ty of a car­dio out­comes study in ad­vance of an ap­proval. And fi­nal­ly, he notes, as a Mer­ck Serono spin­out, they have the kind of ex­per­tise nec­es­sary to make it all hap­pen.

Pox­el still says it would need a Japan­ese part­ner to com­mer­cial­ize the drug, but that’s some­thing that Kuhn feels is doable, giv­en the num­ber of glob­al and lo­cal com­pa­nies that work in the mar­ket — which he says is the sec­ond largest in the world.

The biotech has al­so com­plet­ed Phase II stud­ies in the EU and the US for the drug, which tar­gets mi­to­chon­dr­i­al dys­func­tion. So the com­pa­ny can con­tin­ue to par­lay with Japan­ese reg­u­la­tors as they work with con­tacts in the US and Eu­rope for those big­ger mar­kets. And a Nas­daq IPO re­mains an op­tion, when the time is right.

That may not be easy. But the way Kuhn says it, it doesn’t sound im­pos­si­ble ei­ther.

Fangliang Zhang, AP Images

Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom

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.

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.

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.

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.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.