Clear­side Bio­med­ical pre­dicts its uveitis drug will be re­ject­ed, af­ter FDA re­quests more da­ta

In its sec­ond-quar­ter up­date ear­li­er this month, eye drug de­vel­op­er Clear­side Bio­med­ical in­di­cat­ed it was look­ing to out-li­cense its lead drug, Xipere, which is cur­rent­ly un­der FDA re­view. But those plans may be put on hold af­ter the com­pa­ny on Thurs­day dis­closed the FDA has asked for more da­ta on the drug’s mar­ket­ing ap­pli­ca­tion.

As a re­sult, Clear­side ex­pects to re­ceive a Com­plete Re­sponse Let­ter from the FDA on or be­fore the de­ci­sion date of Oc­to­ber 19, it said. The drug, Xipere (tri­am­ci­nolone ace­tonide oph­thalmic sus­pen­sion), is be­ing eval­u­at­ed for the treat­ment of mac­u­lar ede­ma as­so­ci­at­ed with uveitis, which is char­ac­ter­ized by in­flam­ma­tion of the mid­dle lay­er of the eye.

Agency of­fi­cials have re­quest­ed Clear­side pro­vide sta­bil­i­ty da­ta on tri­am­ci­nolone ace­tonide (TA) sus­pen­sion pro­duced, em­ploy­ing an ‘en­hanced man­u­fac­tur­ing process’ used by the com­pa­ny. Al­though the for­mu­la­tion of the TA sus­pen­sion has not changed — the agency wants to ver­i­fy the com­pa­ra­bil­i­ty of the sta­bil­i­ty pro­files of the batch­es made with the en­hanced man­u­fac­tur­ing process ver­sus the batch­es orig­i­nal­ly sub­mit­ted as part of the mar­ket­ing ap­pli­ca­tion, Clear­side said.

George Lasezkay “We be­lieve this is pri­mar­i­ly a tim­ing is­sue since our sta­bil­i­ty da­ta from pre­vi­ous­ly man­u­fac­tured batch­es have been con­sis­tent and pre­dictable, and we have every rea­son to be­lieve this will con­tin­ue to be the case…Dis­cus­sions with po­ten­tial XIPERE out-li­cens­ing part­ners re­main on­go­ing,” said Clear­side chief George Lasezkay. “We con­tin­ue to ex­pect that we will have suf­fi­cient re­sources to fund op­er­a­tions in­to the third quar­ter of 2020, with­out re­ly­ing on any part­ner­ship-re­lat­ed pay­ments that we might gain through XIPERE part­ner­ing or R&D col­lab­o­ra­tion agree­ments.”

In­vestors weren’t quite as op­ti­mistic. Shares of the Al­pharet­ta, Geor­gia-based com­pa­ny $CLSD tum­bled more than 38% to 78 cents be­fore the bell on Fri­day.

In a note ear­li­er this month, Need­ham’s Serge Be­langer sug­gest­ed the Xipere was poised to win FDA ap­proval with a broad pan-uveitis la­bel that would dif­fer­en­ti­ate it from oth­er cor­ti­cos­teroids, such as Al­ler­gan’s $AGN Ozur­dex, Bausch Health’s Re­tis­ert and Eye­Point Phar­ma­ceu­ti­cals’ Yu­tiq.

“We pre­vi­ous­ly pegged Xipere’s po­ten­tial peak sales in uveitis at $100MM-$150MM. The out-li­cens­ing of Xipere makes sense from a cor­po­rate strat­e­gy for CLSD since the com­pa­ny’s cur­rent fi­nan­cial re­sources (~$26MM as of 6/30/19) lim­it the abil­i­ty to prop­er­ly in­vest in a prod­uct launch. Xipere’s po­ten­tial is like­ly too small to mer­it its own com­mer­cial in­fra­struc­ture and makes more sense in the hands of a com­pa­ny with an ex­ist­ing sales force,” he wrote.

“We view the change in strat­e­gy as the ide­al sce­nario since it po­ten­tial­ly en­hances the com­mer­cial val­ue of Xipere, which CLSD would have been un­able to achieve with its lim­it­ed fi­nan­cial re­sources (~$26MM as of 6/30/19).”

On Thurs­day, the com­pa­ny sug­gest­ed the FDA’s re­quest had no readthrough to the rest of the com­pa­ny’s plat­form, which is in the pre­clin­i­cal stages and is fo­cused on gene ther­a­py and small mol­e­cules.

So­cial im­age: Shut­ter­stock

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.

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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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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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.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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The flu virus (CDC)

Roche tacks on an­oth­er Xofluza in­di­ca­tion as flu sea­son meets pan­dem­ic

Xofluza was heralded as the first new flu drug in 20 years when it got the FDA OK back in 2018. But even so, Roche saw tough competition from cheaper Tamiflu generics that appeared to be nearly as — if not just as — effective.

Now, the pharma says the drug also can be used to prevent influenza after exposure, snagging a new approval and adding to Xofluza’s appeal as flu season meets the pandemic.

Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.