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. 

Adaptive Design Methods Offer Rapid, Seamless Transition Between Study Phases in Rare Cancer Trials

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UPDATED: Roivant bumps stake in Immunovant with a $200M deal. But with M&A off the table, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Sanofi preps a multibillion-dollar buyout of an mRNA pioneer after falling behind in the race for a Covid-19 jab — report

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Anthony Sun, Zentalis and Zentera CEO (Zentalis)

With clinical trials lined up for Zentalis drugs, China's Zentera sets its sights on more dealmaking and an IPO

As Zentalis geared up for an AACR presentation of early data on its WEE1 inhibitor earlier this year, its Chinese joint venture Zentera wasn’t idle, either.

Zentera, which has headquarters in Shanghai, had already nabbed clearance to start clinical trials in China for three of the parent company’s drugs. In May — just a month after Zentalis touted three “exceptional responses” out of 55 patients for their shared lead drug, ZN-c3 — it got a fourth CTA approval.

Thomas Soloway, T-knife CEO

What happens when you give a mouse a human self-antigen? Investors bet $110M to find out

T-knife Therapeutics launched last August on a mission to isolate T cell receptors not from human donors, but from mice. Now, with a new CEO and a candidate bound for the clinic, the Versant-backed company is reloading with a fresh $110 million.

“What we are trying to do for the field of TCR therapy and solid tumor therapy is very analogous to what the murine platforms have done in antibody development,” CEO Thomas Soloway told Endpoints News. 

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UPDATED: Watch out GlaxoSmithKline: AstraZeneca's once-failed lupus drug is now approved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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Not all mRNA vaccines are created equal. Does it matter?; Neuro is back; Private M&A affair; and more

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.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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Bristol Myers pulls lymphoma indication for Istodax after confirmatory trial falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's oncology head Rick Pazdur defends the accelerated approval pathway, claiming it is 'under attack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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