Pfiz­er and Sang­amo re-open PhI­II of he­mo­phil­ia A gene ther­a­py; AEON head­ed to PhI­II

A year af­ter vol­un­tar­i­ly paus­ing the Phase III study of their he­mo­phil­ia A gene ther­a­py pro­gram, and four months af­ter FDA lift­ed the clin­i­cal hold, Pfiz­er and Sang­amo Ther­a­peu­tics say they will re­sume dos­ing next month.

The move push­es back the orig­i­nal da­ta read­out by two years. At the time of the first pa­tient dos­ing in Oc­to­ber 2020, a Pfiz­er spokesper­son had told End­points News that a piv­otal read­out would come in ear­ly 2022. Fol­low­ing the hur­dles caused by the clin­i­cal hold, Pfiz­er said Fri­day morn­ing the new an­tic­i­pat­ed time­line is the first half of 2024.

With the AFFINE study now re­cruit­ing pa­tients again, Pfiz­er and Sang­amo plan to have all sites ac­tive be­fore year’s end. Pfiz­er had paused the study af­ter pa­tients ex­pe­ri­enced ab­nor­mal­ly high lev­els of an es­sen­tial blood clot­ting fac­tor known as fac­tor VI­II. The tri­al looks to en­roll more than 60 men aged 18-64 years with mod­er­ate to se­vere forms of the hered­i­tary bleed­ing dis­or­der.

Sang­amo and Pfiz­er are test­ing if a sin­gle in­jec­tion of the gene ther­a­py, dubbed giroc­toco­gene fitel­par­vovec, im­proves the an­nu­al­ized bleed­ing rate through 15 months of treat­ment, com­par­ing that met­ric to pri­or fac­tor VI­II pro­phy­lax­is re­place­ment ther­a­py.

The duo fol­lows be­hind Bio­Marin, which re­ceived the first gene ther­a­py con­di­tion­al nod for he­mo­phil­ia A last month in the EU. The com­pa­ny plans to re­sub­mit its ap­pli­ca­tion to the FDA by the end of this month.

While not a gene ther­a­py, Sanofi’s fac­tor VI­II re­place­ment drug, efane­soc­tocog al­fa, gained pri­or­i­ty re­view from the FDA last month.

AEON’s neck mus­cle drug head­ed to Phase III 

With sta­tis­ti­cal sig­nif­i­cance in a Phase II now in hand, AEON Bio­phar­ma’s ABP-450 in­jec­tion is like­ly to en­ter a Phase III next year for cer­vi­cal dys­to­nia in adults.

Marc Forth

The neu­ro­log­ic con­di­tion caus­es the head to in­vol­un­tar­i­ly twist and tilt, af­fect­ing neck mus­cle con­trac­tion. Two of the three dos­es, which in­cludes the bot­u­linum tox­in, were shown to have sta­tis­ti­cal sig­nif­i­cance at week four in re­duc­ing signs and symp­toms of CD based on a scale known as the Toron­to West­ern Spas­mod­ic Tor­ti­col­lis Rat­ing. The high­est dose “nar­row­ly missed sta­tis­ti­cal sig­nif­i­cance,” the com­pa­ny said Fri­day morn­ing. The p-val­ues came in at 0.007 for 150 units of the in­jec­tion, 0.0406 for 250 units and 0.0864 for 350 units.

The pri­ma­ry safe­ty goal was met in the place­bo-con­trolled 57-pa­tient study, the com­pa­ny said. Across all three dos­es, the ad­verse event rates were ei­ther sim­i­lar to or low­er than oth­er bot­u­linum tox­in prod­ucts used for CD. Ad­verse events re­lat­ed to the treat­ment were mild or mod­er­ate, ac­cord­ing to AEON.

The Irvine, CA biotech is al­so test­ing the in­jec­tion in pa­tients with mi­graines. Phase II da­ta for the mi­graine pre­ven­tion study will come out next year, pres­i­dent and CEO Marc Forth said in a state­ment.

The pri­vate com­pa­ny picked up an­oth­er $30 mil­lion from ex­ist­ing in­vestors last month, via a con­vert­ible note, to fu­el the late-stage pipeline.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.

Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024.

The decision to not re-commercialize Natpara will be a blow to not only the 2,400 people who were awaiting supplies of their reliable injection since 2019, but also the additional nearly 400 people who were accessing the drugs via the company’s Special Use Program as Takeda sought to resolve these manufacturing issues over the past five years.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Andrew Crockett, KalVista CEO

KalVista ends a PhII study ear­ly af­ter pa­tients suf­fer se­vere and life-threat­en­ing side ef­fects

KalVista took a beating Tuesday after announcing it would scrap a Phase II trial for one of its experimental drugs.

The biotech said in an early morning press release that it is terminating the study for KVD824 after multiple patients in every treatment group saw unsafe, elevated levels of certain liver enzymes. By ending the trial now, KalVista hopes to save some money and funnel it toward another study for its lead program, CEO Andrew Crockett said in a statement.

Pen­ny stock play­er to re­view all op­tions to try stay­ing afloat af­ter clin­i­cal tri­al fail

Adamis Pharmaceuticals is slowly tumbling down, and the biotech is looking at all its options.

After a Phase II/III trial failure last month that sent the penny stock player down an additional 50% to just 15 cents a share, the company said Monday that it is examining options to get the best value for its investors. A statement from Adamis indicates that alternatives include anything from a partnership to a sale of Adamis’ two commercial products, Zimhi and Symjepi.