Bio­Marin sub­mits NDA for con­tro­ver­sial achon­dropla­sia drug; Cell and gene ther­a­py boom sparks €49M buy­out

Bio­Marin is plow­ing ahead with its achon­dropla­sia can­di­date, sub­mit­ting an NDA to the FDA for vosori­tide Thurs­day af­ter­noon.

If ap­proved, the drug would be a once-dai­ly in­jec­tion for chil­dren with the con­di­tion, which is the most com­mon cause of dwarfism. The ap­pli­ca­tion fol­lows the re­lease of topline Phase III tri­al re­sults back in De­cem­ber show­ing vosori­tide im­proved an­nu­al growth by 1.6 cm against place­bo in chil­dren be­tween the ages of 5 and 14.

That fig­ure did not meet some an­a­lysts’ hopes of 2.0 cm growth, but for those with the con­di­tion, the drug’s po­ten­tial ef­fi­ca­cy is not at the fore­front of their con­cerns. Rather, many in­di­vid­u­als with achon­dropla­sia see Bio­Marin at­tempt­ing to solve a prob­lem that doesn’t ex­ist and prof­it off the stigma­ti­za­tion of their short statures.

The biotech coun­ters that achon­dropla­sia can lead to a host of oth­er med­ical prob­lems, such as skele­tal is­sues that re­quire cor­rec­tive surg­eries, sleep ap­nea, hear­ing loss and spinal prob­lems.

Thurs­day’s news comes on the heels of the FDA hand­ing Bio­Marin a sur­pris­ing set­back ear­li­er this week when the agency is­sued a CRL for the biotech’s he­mo­phil­ia A gene ther­a­py, val­rox. The pro­gram was wide­ly re­gard­ed as a shoe-in for ap­proval, but reg­u­la­tors end­ed up ask­ing for 2 years of fol­low-on da­ta from their Phase III tri­al, rather than the lat­est up­date from Bio­Marin’s in­ves­ti­ga­tors. — Max Gel­man

Cold chain lo­gis­tics sup­pli­er inks €49M buy­out deal

As the cell and gene ther­a­py space grows, as does a key ser­vice provider spe­cial­iz­ing in mov­ing the tem­per­a­ture-sen­si­tive ma­te­r­i­al around the world.

Brent­wood, TN-based Cry­oport is snap­ping up Paris’ CRY­OPDP in an all-cash deal val­ued at around €49 mil­lion ($57 mil­lion) — just €7 mil­lion more than the rev­enue CRY­OPDP in 2019.

“CRY­OPDP is es­pe­cial­ly strong in EMEA and APAC, where it en­joys a strong rep­u­ta­tion as a val­ued spe­cial­ist lo­gis­tics provider to CROs (clin­i­cal re­search or­ga­ni­za­tions), the phar­ma­ceu­ti­cal in­dus­try, and the life sci­ences clin­i­cal re­search com­mu­ni­ty,” Cry­oport CEO Jer­rell Shel­ton said in a state­ment. “Over the com­ing months we an­tic­i­pate fo­cus­ing more at­ten­tion on the fast-grow­ing cell and gene ther­a­py in­dus­try, where CRY­OPDP will play an in­creas­ing role.”

The French com­pa­ny brings over 220 em­ploy­ees across 22 fa­cil­i­ties in 12 coun­tries, who col­lec­tive­ly boast of com­plet­ing 300,000 tem­per­a­ture-con­trolled ship­ments an­nu­al­ly. — Am­ber Tong

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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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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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.

PhRMA sues Trump gov­ern­ment over drug im­por­ta­tion rule — days be­fore it's set to be ef­fec­tive

Ever since President Donald Trump floated the idea of using state-sponsored importation to lower drug prices, PhRMA has made its opposition abundant. Not only is the proposal dangerous and futile,  but the trade group has also argued that it may even be illegal.

Now that the FDA has issued its final rule permitting states to bring certain drugs from Canada, PhRMA is taking the government to court — just a few days before the rule is slated to take effect.