GSK’s flop Poti­ga is picked up and head­ed for a quick makeover for pe­di­atric epilep­sy. Any bets on the prospec­tive price?

Glax­o­SmithK­line took a con­sid­er­able amount of heat last year when the phar­ma gi­ant de­cid­ed to yank a drug called Poti­ga, which had been ap­proved as an an­ti-con­vul­sant for adults but of­ten sold off la­bel to treat an ex­treme­ly rare form of epilep­sy caused by an er­rant KC­NQ2 gene. The drug was con­sid­ered a life­saver by the small group of kids and their fam­i­lies that ben­e­fit­ed from it, but GSK didn’t see enough of a com­mer­cial re­turn to make it worth­while. So they dis­card­ed it, along with oth­er parts of the com­pa­ny that have been stripped away in pur­suit of mak­ing the com­pa­ny more prof­itable in the long run.

“If there was a non­prof­it phar­ma­ceu­ti­cal man­u­fac­tur­er that want­ed to jump in, this is the sort of place that could be a good pos­si­bil­i­ty,” Aaron Kessel­heim, an as­so­ciate pro­fes­sor of med­i­cine at Har­vard Med­ical School, told the Chica­go Tri­bune.

Now, though, a Cana­di­an biotech called Xenon Phar­ma­ceu­ti­cals $XENE wants to see if this old drug has some com­mer­cial po­ten­tial that GSK was blind to. And there’s no sign that they see this as a char­i­ta­ble ven­ture.

The drug was on­ly ap­proved for adults, nev­er among chil­dren, though there was a big de­mand for it from the small num­ber of chil­dren who re­lied on it. Ac­cord­ing to the Chica­go Tri­bune piece, re­searchers nev­er ex­plored the pe­di­atric use be­cause Poti­ga earned a black box warn­ing for some dan­ger­ous side ef­fects, such as blue tinged skin and vi­sion prob­lems, which the fam­i­lies and kids who re­lied on it ev­i­dent­ly learned to live with.

Si­mon Pim­stone

Xenon, though, says it has a green light from the FDA to run the ac­tive in­gre­di­ent in Poti­ga — ezo­gabine, now dubbed XEN496 — through a tiny, piv­otal 20-pa­tient study to prove what GSK proved in adults and what the fam­i­lies have been say­ing for years: The drug con­trols seizures among chil­dren with the ge­net­ic trig­ger. And they are work­ing on a spe­cif­ic pe­di­atric for­mu­la­tion which they say “may” ad­dress the safe­ty is­sues.

“We have done an im­mense amount of dili­gence lead­ing up to the ad­di­tion of XEN496 to our nov­el and ro­bust pipeline of ion-chan­nel, an­ti-epilep­tic drugs,” says Si­mon Pim­stone, the CEO at Xenon. “Based on feed­back from key opin­ion lead­ers, ad­vo­ca­cy groups, pre-ex­ist­ing lit­er­a­ture, and promis­ing da­ta gen­er­at­ed to date, we be­lieve there is tremen­dous sup­port for us to vig­or­ous­ly pur­sue the de­vel­op­ment and com­mer­cial­iza­tion of XEN496 in or­der to reach the pe­di­atric KC­NQ2-EE pa­tient pop­u­la­tion as rapid­ly as pos­si­ble.”

GSK, for its part, said they lost ex­clu­siv­i­ty on the drug in the US mar­ket in 2016. And they’re giv­ing Xenon ac­cess to its drug da­ta — to be used in their ap­pli­ca­tion — for free. The drug can be or­dered from a Cana­di­an phar­ma­cy at a cost of $189 for a bot­tle of 84 50 mg pills; that’s $2.25 per pill.

Typ­i­cal­ly, if an ap­proval is in the works for the US this would be fol­lowed by a high, 6-fig­ure com­mer­cial price fol­low­ing a small, once-off study. I’ve asked the com­pa­ny for some guid­ance on their ex­pec­ta­tions, but haven’t heard back.

Re­pur­pos­ing old drugs and ap­ply­ing sky-high prices on them has be­come a reg­u­lar, though of­ten con­tro­ver­sial, strat­e­gy in biotech. Marathon stud­ied an old steroid drug nev­er ap­proved in the US specif­i­cal­ly for Duchenne mus­cu­lar dy­s­tro­phy, and then stirred up a hor­nets nest of trou­ble as law­mak­ers caught wind of their price. That drug wound up at PTC. And of course Mar­tin Shkre­li made a sim­i­lar ap­proach glob­al­ly un­pop­u­lar as he took con­trol of an old gener­ic and sim­ply hiked the price 5000%.

One of the ironies here is that the drug may not be as ef­fec­tive as a ke­to­genic di­et, some­thing that Johns Hop­kins has been study­ing for decades as a first ap­proach to child­hood epilep­sy. There have al­so been stud­ies that in­di­cate that a ke­to di­et — low carbs, mod­er­ate pro­tein and high fat — al­so works in this par­tic­u­lar ge­net­ic sub­set, among oth­ers.

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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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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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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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.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

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.