Al Gianchetti, XyloCor CEO

Xy­lo­Cor wraps up PhII for heart dis­ease gene ther­a­py, plans for piv­otal tri­al

Xy­lo­Cor Ther­a­peu­tics says pa­tients with heart dis­ease who got its gene ther­a­py could ex­er­cise for longer and had few­er chest pain at­tacks. The biotech an­nounced it com­plet­ed a Phase I/II tri­al of the gene ther­a­py Thurs­day morn­ing, and plans to move for­ward with a piv­otal tri­al.

In the Phase II por­tion of the tri­al, 28 pa­tients with angi­na (or chest pain) caused by coro­nary artery dis­ease and who had no oth­er treat­ment op­tions were en­rolled and were giv­en the high­est test­ed dose from the first part of the tri­al. Pa­tients were fol­lowed for six months.

In terms of sec­ondary ef­fi­ca­cy end­points, pa­tients saw im­prove­ments on an ex­er­cise test, which chal­lenged how long they could walk on a tread­mill. They al­so had few­er chest pain at­tacks com­pared to base­line, and Xy­lo­Cor said that near­ly half of the 28 pa­tients could do or­di­nary phys­i­cal ac­tiv­i­ty with­out chest pain at six months.

For the key sec­ondary end­points, changes were sta­tis­ti­cal­ly sig­nif­i­cant, CEO Al Gi­anchet­ti told End­points News. 

There were no se­ri­ous ad­verse events re­lat­ed to the ade­n­ovirus vec­tor or drug, though there were some ad­verse events re­lat­ed to the ad­min­is­tra­tion pro­ce­dure, “but none were un­ex­pect­ed,” Gi­anchet­ti said. The gene ther­a­py was in­ject­ed di­rect­ly to the heart by sur­geons.

Gi­anchet­ti not­ed that by tar­get­ing di­rect­ly to the heart, they could give “rel­a­tive­ly low dos­es” of the vi­ral-based ther­a­py. Gi­anchet­ti de­clined to get in­to the specifics on both the ef­fi­ca­cy and safe­ty da­ta, not­ing that Xy­lo­Cor plans to present the re­sults at a sci­en­tif­ic meet­ing.

Ronald Crys­tal

Xy­lo­Cor was found­ed by Weill Cor­nell gene ther­a­py pro­fes­sor Ronald Crys­tal and Bay­lor Col­lege of Med­i­cine heart surgery pro­fes­sor Todd Rosen­gart, who once was a pro­fes­sor at Weill. The ther­a­py, li­censed from Weill, de­liv­ers the gene for VEGF, an es­sen­tial growth fac­tor for blood ves­sel cells.

The goal is that by de­liv­er­ing the gene to the heart, the treat­ment can pro­mote new blood ves­sel growth, where blood can flow through the heart in­stead of the dis­eased ves­sels.

Oth­ers are al­so try­ing to use ge­net­ic med­i­cine to tack­le heart dis­ease, which is the lead­ing cause of death in the US. Tenaya Ther­a­peu­tics is work­ing on a num­ber of pro­grams for ge­net­ic heart con­di­tions as well as heart fail­ure, though it has yet to put these can­di­dates in the clin­ic.

And Verve Ther­a­peu­tics is us­ing base edit­ing to per­ma­nent­ly turn off a gene in the liv­er that en­codes for bad cho­les­terol. While it’s test­ing the ther­a­py first in pa­tients who have fa­mil­ial hy­per­c­ho­les­terolemia, a rare ge­net­ic dis­ease, it hopes to one day use it for pa­tients with ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease.

Gi­anchet­ti joined as Xy­lo­Cor’s first CEO in 2016. Based just out­side Philadel­phia in Wayne, PA, Xy­lo­Cor raised a Se­ries A ex­ten­sion of $22.6 mil­lion in 2021. And mov­ing for­ward with the new da­ta in hand, Gi­anchet­ti said Xy­lo­Cor needs to raise more cap­i­tal as well, though he did not spec­i­fy how.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.