PJ Anand, Alcyone Therapeutics CEO

A gene ther­a­py biotech is burst­ing on­to the scene with 12 pro­grams in tow. An ul­tra-rare neu­ro dis­ease is up first

In down­town Low­ell, MA, tucked in­to one of the coun­try’s old­est sur­viv­ing tex­tile mill com­plex­es is a new biotech toil­ing away on next-gen CNS ther­a­pies for se­vere neu­ro­log­i­cal dis­or­ders. And it’s start­ing with Rett syn­drome — a rare neu­rode­vel­op­men­tal con­di­tion that al­most ex­clu­sive­ly af­fects girls.

Kathrin Mey­er

Al­cy­one Ther­a­peu­tics emerged from stealth mode Wednes­day with $23 mil­lion from RTW In­vest­ments and 12 new gene ther­a­py pro­grams in the works. The com­pa­ny was cre­at­ed last sum­mer, short­ly af­ter founder and CEO PJ Anand met Kathrin Mey­er, a prin­ci­pal in­ves­ti­ga­tor in the gene ther­a­py unit at the Abi­gail Wexn­er Re­search In­sti­tute at Na­tion­wide Chil­dren’s Hos­pi­tal.

“We de­cid­ed that there was so much mu­tu­al syn­er­gy,” Anand told End­points News, lead­ing them to ex­e­cute a col­lab­o­ra­tion last year.

The com­pa­ny is in pre-IND en­abling stud­ies for one of its lead can­di­dates, AC­TX-101 — a Rett syn­drome treat­ment us­ing X-re­ac­ti­va­tion tech­nol­o­gy. Its oth­er lead can­di­date, AC­TX-401, is a gene ther­a­py for spinal mus­cu­lar at­ro­phy with res­pi­ra­to­ry dis­tress type 1 (SMARD1), for which it will file an IND this year.

Re­searchers at the Bay­lor Col­lege of Med­i­cine iden­ti­fied mu­ta­tions in a gene called MECP2 as the cause of Rett syn­drome back in 1999. The dis­or­der caus­es se­vere im­pair­ments that af­fect a child’s abil­i­ty to speak, walk, eat and breathe eas­i­ly, a com­mon sign be­ing con­stant repet­i­tive hand move­ments. The chal­lenge with con­ven­tion­al trans­gene re­place­ment, Anand ex­plained, is that over­pro­duc­tion of MECP2 is just as bad as un­der­pro­duc­tion.

“It is one of those Goldilocks in­di­ca­tions where you want to have the right amount of pro­tein,” he said. “The po­ten­tial of this plat­form is to in­crease the ef­fi­cien­cy of gene ther­a­py not by 1 time or 2 times, but by mul­ti­ple times.”

Al­cy­one is cou­pling its pre­cise CNS de­liv­ery plat­form with X-chro­mo­some re­ac­ti­va­tion tech­nol­o­gy, so in­stead of just re­plac­ing the gene in the ac­tive X-chro­mo­some (and risk­ing over­ex­pres­sion of MECP2), they’re se­lec­tive­ly re­ac­ti­vat­ing ar­eas in the in­ac­tive X-chro­mo­some to get MECP2 pro­duc­tion.

Anand has big plans to make this pro­gram a “pipeline in a prod­uct,” where the same gene prod­uct can be ap­plied to oth­er X-chro­mo­some-re­lat­ed syn­dromes. Pri­or to this, he found­ed Anun­cia, a spin­off look­ing to treat hy­dro­cephalus and cere­brospinal flu­id dys­func­tions, and co-found­ed the med­ical de­vice com­pa­ny Arthrome­da.

Al­cy­one’s join­ing a host of oth­er com­pa­nies on the hunt for the first Rett syn­drome ther­a­py, in­clud­ing New York biotech Anavex, which got fast track sta­tus for its blar­came­sine back in 2020. Aca­dia Phar­ma­ceu­ti­cals, mak­er of the Parkin­son’s dis­ease drug Nu­plazid, en­tered its trofine­tide in a piv­otal Phase III study for Rett syn­drome in late 2019. Newron Phar­ma­ceu­ti­cals dropped out of the race last May, culling its Rett syn­drome pro­gram af­ter sari­zotan failed a Phase II/III tri­al.

The 35-per­son team at Al­cy­one is al­so de­vel­op­ing can­di­dates us­ing con­ven­tion­al trans­gene re­place­ment, vec­tor­ized ex­on skip­ping and pro­mo­tor mod­u­la­tion.

“Our next-gen­er­a­tion pre­ci­sion de­liv­ery plat­form shows strong po­ten­tial in over­com­ing the fun­da­men­tal chal­lenge in CNS ther­a­py de­vel­op­ment,” Anand said.

Cor­rec­tion: Al­cy­one plans on fil­ing an IND this year for AC­TX-401, not AC­TX-101. 

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

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.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.