Cancer Therapies Draw Venture Cash

"Venture capitalists are pouring billions of dollars into an effort to treat a wider range of cancer patients with a new and powerful type of immunotherapy.

Immune-cell therapies first approved in the U.S. in 2017, known as CAR-T, revolutionized the treatment of certain blood cancers, causing long-term remissions in many patients. The therapies involve genetically engineering patients' own immune cells to find and wipe out the cancer.

But these treatments come with hefty price tags, partly because of the steps involved, and, for now, only apply to the minority of cancer patients with blood cancers. Today's cellular therapies are "missing a huge amount of market based on the cost and complexity," said Dr. Daina Graybosch, a senior analyst covering immuno-oncology for investment bank SVB Leerink.

Spurred by the promise seen in blood-cancer treatment, investors are backing biotech companies aiming to lower the cost of cell therapy and extend it to solid tumors, which form in organs around the body and account for about 90% of cancers in adults.

Venture firms invested $3.83 billion in cancer cell-therapy startups last year, more than double the $1.82 billion invested in 2019, according to market tracker CB Insights. Through April 2, cell-therapy startups raised $1.89 billion.

These companies face several obstacles, including the difficulty of cutting through the defenses solid tumors mount against immune cells. Investors are betting that new tools like gene-editing will help startups clear those hurdles and expand cell therapy.

"It's just the beginning of a wide-open field," said Dr. Kenneth Meehan, director of the transplant and cellular therapy program at Dartmouth and Dartmouth-Hitchcock's Norris Cotton Cancer Center in Lebanon, N.H.

CAR-T therapy involves collecting T cells, a type of white blood cell, from patients and shipping them to a drug-company lab, where scientists equip them with molecular hooks, called chimeric antigen receptors (the CAR in CAR-T), that latch onto a marker found on cancers. Then they are injected back into the patient.

The newest CAR-T drug, Abecma, was developed by Bristol-Myers Squibb Co. and bluebird bio Inc. for multiple myeloma, a type of blood cancer. In a study, 72% of patients partially or completely responded to the therapy and 28% had complete responses -- the disappearance of any sign of the disease. U.S. regulators approved Abecma in March.

"I'm just amazed that I continue to improve," said Susan Voigt, a 69-year-old mother of seven who participated in clinical trials of Abecma. She has been in remission since receiving the treatment in 2018 at the Dana-Farber Cancer Institute in Boston.

Abecma, given as a one-time infusion, has a wholesale list price of $419,500. Bristol-Myers and bluebird expect it will be covered by Medicare and commercial insurers, but that can vary by plan, a Bristol-Myers representative said.

CAR-T therapies carry warnings about cytokine-release syndrome, the release of inflammatory cytokine molecules that can be severe or fatal. Its symptoms can include fever, low blood pressure and difficulty breathing, and have occasionally besieged Covid-19 patients. Cytokine release-syndrome cases were mostly low-grade in the Abecma trial, Bristol-Myers and bluebird said.

Venture investors' bets on new cell therapies reflect a broad scientific push to find out if living cells and genes can address a wider group of illnesses.

"This is really a modality that promises to cure some of our chronic diseases," said Dr. Jurgen Eckhardt, head of Leverkusen, Germany-based Leaps by Bayer, the venture-capital arm of Bayer AG and an investor in cell-therapy companies such as Century Therapeutics Inc.

Several companies aim to lower costs through off-the-shelf therapies that don't use patients' own cells, including startups such as Century, Acepodia Inc., Appia Bio Inc., Caribou Biosciences Inc. and Celularity Inc., as well as publicly traded biotechs including ImmunityBio Inc.

Startups like Century and Cytovia Therapeutics Inc. create immune cells using induced pluripotent stem cells, in which mature cells are reprogrammed back to an earlier state. That lets scientists develop them into specific types of immune cells." [1]

1. Cancer Therapies Draw Venture Cash
Gormley, Brian. Wall Street Journal, Eastern edition; New York, N.Y. [New York, N.Y]14 May 2021: B.11.