Per­lara col­lab­o­rates with Har­vard/Un­di­ag­nosed Dis­eases Net­work; Verona shares soar on pos­i­tive CF da­ta

→ Per­lara, the rare dis­ease com­pa­ny found­ed by Twit­ter’s fa­vorite biotech rebel Ethan Perl­stein, an­nounced two sci­en­tif­ic dis­cov­ery col­lab­o­ra­tions Fri­day with Har­vard Med­ical School and the Un­di­ag­nosed Dis­eases Net­work. The re­search part­ner­ships are for two rare mono­genic neu­rode­vel­op­men­tal dis­or­ders. One is caused by a mu­ta­tion in GNA01 and the oth­er caused by a mu­ta­tion in RPS6KA3 (Cof­fin-Lowry Syn­drome). Per­lara plans to de­vel­op and val­i­date ne­ma­tode and fly “pa­tient avatars” of path­o­gen­ic GNA01 and RPS6KA3 mu­ta­tions for use in high-through­put phe­no­typ­ic drug screens. Perl­stein, CEO of the Per­lara, is best known in the bio­phar­ma world through his out­spo­ken Twit­ter pres­ence, where he’s laid bare the dif­fi­cul­ty of rais­ing cap­i­tal as a young com­pa­ny work­ing on “dis­eases thought too rare to at­tract the in­ter­est of phar­ma­ceu­ti­cal com­pa­nies.” He’s been called rad­i­cal for his trans­paren­cy, and a rebel for chal­leng­ing fundrais­ing norms on a pub­lic stage. In lieu of sig­nif­i­cant ven­ture cap­i­tal funds, Perl­stein has in­stead re­lied heav­i­ly on part­ner­ships with pa­tient groups since the com­pa­ny’s 2014 in­cep­tion. Last year, how­ev­er, the com­pa­ny did raise a $7.4M Se­ries A.

→  Shares of Verona Phar­ma shot up 25% $VR­NA on Fri­day af­ter the Lon­don-based biotech tout­ed Phase IIa da­ta for its cys­tic fi­bro­sis ther­a­py RPL554. Pa­tients in the small study achieved a 6% sus­tained im­prove­ment in FEV1, ac­cord­ing to re­searchers, af­ter a sin­gle dose. The com­pa­ny de­scribes the drug as a “dual in­hibitor of the en­zymes phos­pho­di­esterase 3 and 4 that acts as both a bron­chodila­tor and an an­ti-in­flam­ma­to­ry agent in a sin­gle com­pound.”

→ The FDA has ac­cept­ed for re­view Re­gen­eron $REGN and its part­ner Sanofi’s $SNY mar­ket­ing ap­pli­ca­tion for Dupix­ent (dupilum­ab). The duo is ask­ing for the OK to sell the drug as an add-on main­te­nance treat­ment in cer­tain adults and ado­les­cents at least 12 years old with mod­er­ate-to-se­vere asth­ma. The ap­pli­ca­tion is backed by clin­i­cal da­ta from 2,888 adults and ado­les­cents from three piv­otal tri­als, the com­pa­nies said in a state­ment. The ac­tion date is set for Oc­to­ber 20. Dupix­ent, a ful­ly hu­man mon­o­clon­al an­ti­body, is al­ready ap­proved in the US for atopic der­mati­tis.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.