FDA mo­tions Al­ny­lam's lumasir­an in­to a PhI­II short track, set­ting up a shot at an OK in '20

Al­ny­lam $AL­NY may be go­ing so­lo on the de­vel­op­ment of rare dis­ease drug lumasir­an af­ter Sanofi took a pass on its op­tion, but the FDA has come through with a ma­jor as­sist. The com­pa­ny an­nounced to­day that reg­u­la­tors have agreed on a Phase III study de­sign that would ac­cel­er­ate — should all go well — its jour­ney to ap­proval.

For the tri­al de­signed to test lumasir­an in the ul­tra-rare pri­ma­ry hy­per­ox­aluria type 1 (PH1), the new­ly reached align­ment al­lows Al­ny­lam to adopt a pri­ma­ry end­point based on a bio­mark­er: the re­duc­tion of uri­nary ox­alate at six months. The FDA has al­so sanc­tioned a study size of around 25 pa­tients with PH1.

Pritesh Gand­hi

The use of a bio­mark­er spot­lights the FDA’s will­ing­ness to let drug­mak­ers shoot for faster OKs, rather than forc­ing them to wait for da­ta on dis­ease pathol­o­gy (Al­ny­lam notes the bio­mark­er is “di­rect­ly linked to the patho­phys­i­ol­o­gy of PH1 and known to be well cor­re­lat­ed with dis­ease pro­gres­sion”).

The tri­al de­sign pact with the agency al­so marks an ad­vance for the late-stage pipeline at Al­ny­lam as it waits for an ex­pect­ed ap­proval of patisir­an, which will launch their first com­mer­cial op­er­a­tions in RNAi. This biotech has been through all the ups and downs of­ten seen with any new ap­proach to drug de­vel­op­ment. And it’s poised for some ma­jor changes.

With the study de­sign ham­mered out, Al­ny­lam ex­pects to start the PhI­II study in mid-2018, re­port topline re­sults in 2019 and, if pos­i­tive, sub­mit an NDA in ear­ly 2020.

“We are very pleased with the FDA’s shared sense of ur­gency to eval­u­ate the ef­fi­ca­cy and safe­ty of lumasir­an as a po­ten­tial ther­a­peu­tic op­tion for pa­tients as rapid­ly as pos­si­ble,” said Pritesh Gand­hi, gen­er­al man­ag­er of the lumasir­an pro­gram, in a state­ment.

Al­ny­lam is cur­rent­ly con­duct­ing a Phase I/II Part B study in 20 pa­tients. Pre­lim­i­nary re­sults, an­nounced last year, sug­gest that lumasir­an led to a mean max­i­mal re­duc­tion in uri­nary ox­alate of 66% in an un­blind­ed group of four pa­tients, and all of them achieved uri­nary ox­alate lev­els at or near the nor­mal range. Lumasir­an, an RNAi ther­a­peu­tic, is de­signed to “re­duce the he­pat­ic lev­els of the [gly­co­late ox­i­dase] en­zyme, there­by de­plet­ing the sub­strate nec­es­sary for ox­alate pro­duc­tion.” It was grant­ed a break­through ther­a­py des­ig­na­tion in March.

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

De­spite safe­ty, ef­fi­ca­cy con­cerns, FDA Ad­Com nar­row­ly rec­om­mends Mallinck­rodt drug

A day after the FDA detailed significant concerns about just how effective or safe an experimental Mallinckrodt liver drug was, an advisory committee decided by a single vote to recommend it for approval.

“I voted yes to approve, but I also wrote in, ‘but barely,’” Paul Ridker, a cardiologist at Brigham and Women’s Hospital and one of the panelists, said at the meeting, according to FierceBiotech. The vote was 8-7.

John Furey, Imvax CEO

A neu­ro­sur­geon spent the past 30 years de­vel­op­ing a neoanti­gen tu­mor vac­cine. Now he has $112M to put it to piv­otal test

As a neurosurgeon, David Andrews knew there wasn’t much he could do for his glioma patients after resecting — rarely fully — their tumor. Even with the best treatment and care available, median overall survival is just somewhere between 14 and 16 months.

Then in the 1990s, his mentor at Thomas Jefferson University introduced him to Renato Baserga, a pathologist who had been studying the effect of using antisense oligonucleotide to knock out the insulin-like growth factor type 1 receptor in cancers. As IGF-R1 drives tumor growth and metastasis, the preclinical reasoning went, implanting a molecule targeting the receptor together with the tumor material near lymph nodes can slow down the spread of the cancer.

Sanjiv Patel, Relay CEO

Re­lay reaps $400M IPO wind­fall af­ter draw­ing the cur­tain on mo­tion-based drug de­sign pipeline

A red-hot venture market back in 2018 propelled Relay Therapeutics to a whopping $400 million crossover round. And now, the biotech is riding on a red-hot IPO market to a public debut that brought in the same exact amount.

The $400 million raise breaks down to 20 million shares sold at $20 each — above even the amended proposed range of $16 to $18.

It’s also double what Relay had penciled in with the initial S-1 filing, in yet another illustration that those figures are often nothing more than a placeholder these days.

Mene Pangalos, AstraZeneca R&D chief (AstraZeneca via YouTube)

A day af­ter Mod­er­na vac­cine re­sults, ru­mors swirl of pend­ing As­traZeneca da­ta

A day after Moderna and the NIH published much-anticipated data from their Phase I Covid-19 vaccine trial, attention is turning to AstraZeneca which, according to a UK report, is expected to publish its own early data tomorrow.

ITV’s Robert Peston reported that AstraZeneca will publish the Phase I data in The Lancet. 

AstraZeneca and Moderna represent the two most ambitious Covid-19 vaccine efforts, having set the quickest timelines for approval (though they were recently joined in that regard by the Pfizer-BioNTech partnership) and some of the loftiest goals in total doses. Yet there is even less known about AstraZeneca’s vaccine’s effect on humans than there was about Moderna’s before yesterday. Although, in a controversial move, Moderna released some statistics from its Phase I in May, AstraZeneca has yet to say anything about what it saw in its Phase I trial — a move consistent with the scientific convention to withhold data until it can be published in a peer-reviewed journal.

BioAt­la rakes in $72.5M Se­ries D, ad­vanc­ing re­search for pH-de­tect­ing can­cer treat­ments

A little over a year after agreeing to a worldwide collaboration with hefty Chinese biotech BeiGene worth up to $270 million, BioAtla is ready for another haul.

The San Diego-based biotech pulled in a $72.5 million haul for their Series D financing, which the company announced Wednesday. Funds will go toward their four main clinical programs developing cancer treatments, two of which are currently in Phase II, BioAtla president and former Celgene COO Scott Smith said.

Ludwig Hantson, Alexion CEO

Why pay $4B for a steady di­et of dis­ap­point­ment? Porges turns thumbs down on Alex­ion’s M&A strat­e­gy, of­fers some point­ers

When Alexion announced recently that it was paying $1.4 billion to bag Portola and its underperforming Factor Xa inhibitor reversal agent, you could hear the head-scratching going on around virtual Wall Street.

Why was Alexion going down the discount lane for new products? And why something like this? Analysts have been urging Alexion to get serious about M&A for years if it was serious about diversifying the company beyond Soliris and its successor drug. But this wasn’t the kind of heavy-impact deal they were looking for.

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