Work­ing to get its pain R&D strat­e­gy back on track, Acel­Rx steps clos­er to an FDA pitch

Acel­Rx has de­liv­ered an­oth­er pos­i­tive piece of the Phase III puz­zle for ARX-04, its un­der-the-tongue ap­pli­ca­tion of a 30 mcg dose of the pow­er­ful opi­oid sufen­tanil de­signed to de­liv­er a jolt of fast pain re­lief.

Ex­pand­ing on an im­prove­ment in pain scores al­ready record­ed for the first co­hort of 40 in­jured pa­tients who turned up at the ER and re­ceived a sin­gle dose of ARX-04, the Red­wood City, CA-based biotech says that it record­ed a slight­ly bet­ter out­come for a small group of 36 more pa­tients who were green-light­ed for mul­ti­ple ap­pli­ca­tions.

The lat­est batch of da­ta leaves this treat­ment on track to a near-term NDA, com­pa­ny ex­ecs tell me, as Acel­Rx works to stay on track with this pro­gram af­ter be­ing de­railed on its lead ef­fort last year.

The biotech $ACRX re­port­ed last Feb­ru­ary that ARX-04 cut base­line pain scores by an av­er­age of 2.7 on a 10-point scale. In the sec­ond co­hort, the drop was 3.1 points from a base­line score of 8.1, hit­ting the pri­ma­ry end­point. Most of the pa­tients, 75%, got the pain re­lief they were look­ing for from a sin­gle dose un­der the tongue, while 7 re­quired two dos­es and two got a third. Three were al­so dosed with mor­phine. Ap­pro­pri­ate­ly, the re­sults were pre­sent­ed at the 7th World Con­gress of Moun­tain & Wilder­ness Med­i­cine.

Pam Palmer, CMO and Co-Founder of Acel­Rx

Those are small num­bers, but com­pa­ny co-founder and Chief Med­ical Of­fi­cer Pam Palmer says that the com­pa­ny has run 11 stud­ies look­ing at dif­fer­ent dos­es of sufen­tanil, in­clud­ing the 15 mcg dose. And all of that will be used to sup­port its pitch to the agency.

“We want­ed to go af­ter a broad­er pa­tient pop­u­la­tion than the post-op­er­a­tive set­ting,” she notes, not­ing that that is where you’ll find the bulk of the re­lat­ed painkillers used. Ac­cord­ing to the CDC, there are some 136 mil­lion vis­its to the emer­gency de­part­ment each year, ac­count­ing for 44 vis­its for every 100 Amer­i­cans. Acel­Rx says that close to 50 mil­lion of those vis­its re­quire at least a cou­ple of dos­es of opi­oids to con­trol pain. And that’s the mar­ket the com­pa­ny hopes to tap in on, once it gets past the FDA.

It’s al­so no easy task to re­cruit emer­gency room pa­tients for a clin­i­cal tri­al, she adds in ex­pla­na­tion of the num­bers used in the late-stage study, not­ing that most peo­ple rush­ing to the ER for help aren’t in the mood to be re­cruit­ed for a tri­al.

The U.S. Army Med­ical Re­search and Ma­teriel Com­mand has helped fund the work on ARX-04, look­ing for a treat­ment that can be used in bat­tle­field sit­u­a­tions. And the treat­ment has al­so demon­strat­ed its use­ful­ness with­out se­vere­ly af­flict­ing cog­ni­tion, notes the com­pa­ny – a use­ful fea­ture for a drug that could be used in com­bat.

Acel­Rx’s stock, though, still hasn’t re­cov­ered from a nasty shock back in the spring of 2015, when the biotech had to sud­den­ly and un­ex­pect­ed­ly re­nege on its promise to in­vestors than no new Phase III study would be re­quired for its top opi­oid drug Za­lvi­so.

Like ARX-04, Za­lvi­so of­fers a twist on dos­ing the pow­er­ful opi­oid sufen­tanil. It is sup­posed to al­low hos­pi­tal pa­tients to self-ad­min­is­ter mi­cro tablets of the drug un­der the tongue, re­plac­ing an IV, for light­ning fast pain re­lief. But reg­u­la­tors, who have been on high alert wher­ev­er opi­oids are in­volved, want to see da­ta from an­oth­er study — the fourth from Acel­Rx -to bet­ter eval­u­ate the risks of dis­pens­ing er­rors.

The set­back forced the com­pa­ny to re­struc­ture and re­con­fig­ure its com­mer­cial­iza­tion plans. Its CEO at the time left soon af­ter.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

Mod­er­na says Covid-19 vac­cine boost­er in­creased im­mune re­sponse against vari­ants of con­cern

About a month after announcing their variant-specific Covid-19 vaccine boosters showed promising results in mice, Moderna says it now has some human data to back it up.

Volunteers given a booster shot about six to eight months after receiving their second dose saw increased antibody levels against SARS-CoV-2 and two variants of concern: B.1.351, which was first identified in South Africa, and P.1, first identified in Brazil, the company said on Wednesday.

FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.

Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.