BioCryst gets a big boost from its lat­est HAE da­ta, but it still faces a dan­ger­ous high wire act and gi­ant ri­vals

Jon Stone­house

A lit­tle more than a year ago, shares of Re­search Tri­an­gle Park, NC-based BioCryst $BCRX were crushed by a failed Phase II tri­al for their lead drug for hered­i­tary an­gioede­ma, or HAE. Set­backs like that are not new for BioCryst. But the biotech turned to an­oth­er HAE drug com­ing up the pipeline. And on Thurs­day, re­searchers de­tailed their sec­ond in­ter­im da­ta re­view of the drug, and won a 31% spike for their stock with an over­all pos­i­tive take on the mid-stage da­ta.

It’s just a snap­shot — in­ter­im re­views of what is, af­ter all, an in­ter­im study can be mis­lead­ing, if not pri­mar­i­ly aimed at boost­ing a stock — but the biotech did build some bad­ly need­ed con­fi­dence for this lat­est at­tempt to de­vel­op an im­por­tant new ther­a­py.

The num­bers did not re­flect a dose de­pen­dent re­duc­tion in HAE at­tacks, which the biotech – some­what con­tro­ver­sial­ly – has been ex­plain­ing as a mis­read by tri­al par­tic­i­pants. Re­searchers are in­sist­ing that the pa­tients are record­ing “tran­sient ab­dom­i­nal ad­verse events” as symp­toms of an HAE at­tack in the two high­est dos­es.

The de­tails on now 44 pa­tients from the APeX-1 tri­al:

In the in­tent-to-treat (ITT) pop­u­la­tion, cor­re­spond­ing re­duc­tions by treat­ment group were: 125 mg QD, 73% (p=0.004); 250 mg QD, 44% (p=0.090) and 350 mg QD, 45% (p=0.014) com­pared to place­bo.

Ob­vi­ous­ly, BioCryst has a long way to go be­fore it can pitch a drug like this to reg­u­la­tors. But its back­ers have been en­cour­aged by the pos­i­tive sets of da­ta that they have been see­ing. A fi­nal read­out of the Phase II is ex­pect­ed in the third quar­ter.

Leerink’s Ja­son Ger­ber­ry has been track­ing the drug, not­ing ear­li­er in the year that BioCryst not on­ly has to suc­ceed in tri­als, it al­so needs to beat the com­pe­ti­tion, no­tably Shire’s Cin­ryze as well as its ex­per­i­men­tal late-stage drug SHP643. As an oral drug, BioCryst would have a leg up over in­jecta­bles. But fi­nal safe­ty and ef­fi­ca­cy da­ta will still need to be strong, rel­a­tive to the com­pe­ti­tion.

Af­ter the first cut of the HAE da­ta, Ger­ber­ry not­ed that BioCryst’s drug “com­pares to stat sig mean re­duc­tions (at 12-weeks) in ITT groups for the fol­low­ing com­pet­i­tive agents: 50% for Shire’s Cin­ryze, 88-100% for Shire’s SHP643 and 67-88% for CSL’s Hae­gar­da.”

Shire’s piv­otal da­ta on SHP643 came through just days ago, and re­mains on the high side.

A 300 mg dose of the drug twice a month de­liv­ered an 87% re­duc­tion in mean HAE at­tack fre­quen­cy, com­pared to 0% in the place­bo group. It hit the pri­ma­ry end­point with “high­ly” sta­tis­ti­cal­ly sig­nif­i­cant re­sults as well as all the sec­on­daries.

Now Shire will dis­patch an NDA to the FDA lat­er this year, leav­ing lit­tle BioCryst to come up from be­hind with a lot to prove. An­oth­er fail­ure for BioCryst would be dealt with harsh­ly.

“These da­ta sup­port our hy­poth­e­sis re­gard­ing the ini­tial find­ings seen from the first in­ter­im analy­sis,” said Jon Stone­house, chief ex­ec­u­tive of­fi­cer and pres­i­dent of BioCryst. “We are de­light­ed to see that a dai­ly dose of 125 mg of BCX7353 re­sults in a high lev­el of ef­fi­ca­cy with an im­proved tol­er­a­bil­i­ty pro­file com­pared to the 350 mg dose ob­served in the first in­ter­im analy­sis. We look for­ward to com­plet­ing Part 3 of the tri­al to se­lect ap­pro­pri­ate dos­es for our piv­otal pro­gram.”

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Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

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Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

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Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

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To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

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Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

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Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

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