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

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

The White House’s Office of Science and Technology Policy (OSTP) last year sought to find ways to better coordinate large-scale clinical trials in the US — as the UK lead by example during the pandemic — especially for these emergency clinical trials.

The lobbying group PhRMA Tuesday called for more clinical trial diversity in underserved areas, including by making participation less of a burden, and expanding eligibility criteria when appropriate.