BioCryst shares are blast­ed as a PhI­II HAE 'suc­cess' un­der­whelms an­a­lysts

It was de­signed to be the oral fix for hered­i­tary an­gioede­ma (HAE) — a rare, po­ten­tial­ly life-threat­en­ing ge­net­ic dis­or­der — but BioCryst Phar­ma­ceu­ti­cal’s ex­per­i­men­tal cap­sule has un­der­whelmed in a piv­otal late-stage study, de­spite meet­ing the pri­ma­ry end­point.

Two dos­es of the oral drug, BCX7353, (110 mg and 150 mg) were in­ves­ti­gat­ed in a 121-pa­tient, place­bo-con­trolled tri­al called APeX-2 for the pre­ven­tion of an­gioede­ma at­tacks — re­cur­rent episodes of se­vere swelling com­mon­ly in the limbs, face, in­testi­nal tract, and air­way.

Both dos­es met the main goal of re­duc­ing the rate of at­tacks. The high­er dose di­min­ished the at­tack rate by 44% ver­sus place­bo (p<0.001),  while the low­er dose cut the at­tack rate by 30% (p=0.024). That didn’t cut it on Wall Street, though. An­a­lysts have long sug­gest­ed a rate of at least 50% would en­sure BCX7353 would be com­pet­i­tive in a mar­ket that is cur­rent­ly mo­nop­o­lized by high­ly ef­fec­tive in­jecta­bles.

Shares of North Car­oli­na-based BioCryst $BCRX were halved in pre­mar­ket trad­ing.

In­jectable med­ica­tions, such as Shire’s 2018-ap­proved lanadelum­ab, can re­duce HAE at­tack fre­quen­cy by about 80%. “(W)e would’ve thought the drug’s ef­fi­ca­cy would’ve need­ed to at least near that of (Shire’s old­er HAE in­jectable) Cin­ryze in or­der for it to be dis­rup­tive to the HAE treat­ment land­scape,” Stifel an­a­lysts wrote in a note.

Need­ham an­a­lysts not­ed re­cent­ly:

Based on dis­cus­sions with KOLs, ef­fi­ca­cy rates in the 50%-range would be suf­fi­cient for an oral prod­uct to play a sig­nif­i­cant role in HAE pro­phy­lax­is mar­ket giv­en the high de­mand for oral al­ter­na­tives. Re­call, Cin­ryze was a game-chang­er for HAE pa­tients de­spite IV dos­ing of every 3-4 days and ef­fi­ca­cy rates of ~50%. The po­ten­tial of BCX7353 in HAE pro­phy­lax­is hinges on meet­ing stat sig in APeX-2, with an ef­fi­ca­cy rate of at least 50% and a clean safe­ty/tol­er­a­bil­i­ty pro­file.


Im­age Source: Shut­ter­stock

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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UP­DAT­ED: Pan­el of neu­ro­science ex­perts lays out the com­pli­ca­tions with us­ing Bio­gen's new Alzheimer's drug

Treatment of early Alzheimer’s patients with Biogen’s new drug Aduhelm should closely resemble how the drug was studied in its pivotal clinical trials, according to new recommendations from a panel of neuroscience experts led by UNLV’s Jeffrey Cummings.

“Those considering aducanumab therapy should understand that the expected benefit is slowing of cognitive and functional decline; improvement of the current clinical state is not anticipated,” they wrote Tuesday in The Journal of Prevention of Alzheimer’s Disease, noting that some of their recommendations are more specific or more restrictive than the information provided in the FDA’s prescribing information.

Eye­ing quick ap­proval, Ab­b­Vie of­fers a close-up on their pres­by­opia drug da­ta

AbbVie picked up some bonus points earlier this year as one of its pipeline adds from the $63 billion Allergan buyout hit its top-line marks. And now the researchers have produced the detailed data on the case they are making with regulators, with an eye on a major new market and a hoped-for approval before New Year’s.

AGN-190584 is aiming to be the first easy-on eyedrop for presbyopia, a common ailment for large numbers of people who find it harder and harder to read things like a watch or cell phone close up. Anyone who’s held a book out at arm’s length in order to read it will be very familiar with the condition, if not the exact diagnosis.

Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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J&J’s Rem­i­cade — the poster child for how to block biosim­i­lars — fi­nal­ly set­tles Pfiz­er suit

Biosimilars have proven time and again (although mostly in Europe) that competition works to bring down the cost of a once-pricey biologic, and can even expand its use.

J&J’s Remicade, however, has always proven to be an outlier.

Back in 2016, Pfizer won FDA approval for its infliximab biosimilar, known as Inflectra, but when the launch foundered, the company sued J&J, claiming that the company’s plan to block biosimilar competition worked incredibly well. Pfizer even went on to win FDA approval for a second infliximab biosimilar in 2017, known as Ixifi, but decided to never launch it.

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