According to STAT article “ Who will Vertex buy? How much will it spend? Deal questions dominate its earnings call”Who (or what) is Vertex NASDAQ: VRTX Pharmaceuticals going to buy? The question — asked many different ways by analysts — dominated the company’s year-end earnings conference call on Monday evening. Vertex CEO Reshma Kewalramani did not respond with a detailed shopping list. Sorry to disappoint. But Vertex ended 2020 with just under $7 billion in cash — “growing financial firepower,” she said — that will be used to acquire assets in the “mid and late stage” of clinical development and complement its cystic fibrosis drug franchise.
Vertex revenue grew an impressive 55% to $6.2 billion in 2020, but the company guided to just under 10% revenue growth for this year. The guidance is almost certainly conservative, but it still underscores Vertex’s challenge — finding a way to maintain the impressive growth that investors have come to expect when its current cystic fibrosis medicines can already treat 90% of patients. On its Monday night call, Kewalramani said the company remains confident in its existing pipeline of experimental medicines. But in her response to all the questions about potential M&A, there was a tacit acknowledgment that Vertex needs to also look outside for growth.
Later this year, Vertex and partner Crispr Therapeutics NASDAQ:CRSP expect to complete the enrollment of approximately 45 patients each into twin clinical trials investigating CTX-001, their CRISPR-based genome-editing treatment for sickle cell disease and beta thalassemia. In response to questions, Kewelramani sounded confident that the FDA would agree that these studies are sufficient for regulatory purposes, although she noted there is no formal agreement in place at this time. How long the patients will need to be followed is also still unclear, although the timelines suggest that Vertex and Crispr could potentially file CTX-001 in the middle or late in 2022. That’s significant because their closest competitor, Bluebird Bio, won’t be filing with the FDA until late 2022.
Regarding VX-864, its experimental treatment for the rare, inherited lung disease called alpha-1 antitrypsin deficiency, Vertex said a readout of a Phase 2 study is expected in the first half of the year. Last October, Vertex was forced to shelve its first drug for alpha-1 antitrypsin deficiency due to liver toxicity. Independent study monitors are taking regular looks at the safety data from the ongoing VX-864 study, and to date, patient enrollment continues, Vertex said Monday. This suggests that VX-864 isn’t running into the same liver toxicity issues that beset Vertex’s previous compound.
Avalon GloboCare Corp. (NASDAQ: AVCO) (Avalon or The Company), a clinical-stage global developer of cell-based technologies and therapeutics, today announces the expansion of its co-development research program with the Massachusetts Institute of Technology (MIT) to apply two leading technologies—CRISPR-based genome editing and QTY protein design—to potentially treat and prevent cancer metastasis.
Metastasis—the spreading of cancer cells from the initial tumor site to surrounding tissues and distant organs—is responsible for the vast majority of cancer deaths. In an ongoing co-development research program, Avalon and the laboratory of Dr. Shuguang Zhang at MIT’s Media Lab have developed a novel approach to potentially block the signals that cancer cells require to metastasize in order to prevent cancer spread and death.
In the first quarter of 2020, Avalon GloboCare (NASDAQ: AVCO) successfully completed a phase 1 first-in-human clinical study of AVA-001 in China for the treatment of relapsed refractory B cell acute lymphoblastic leukemia (R/R B-ALL). 90% of R/R B-ALL patients achieved complete remission with one dose and within one month of treatment, and then proceeded to a curative-intent allogeneic bone marrow transplant. Also there was minimal and well tolerated side effects with little neurotoxicity or cytokine release. This paradigm of bridging CAR-T cell therapy to bone marrow transplant creates a new horizon with potentially ground breaking commercial application for patients with relapsed/refractory B-ALL and other hematologic cancers.
So far in 2021in addition to the MIT deal, Avalon also announced a collaboration with the University of Pittsburgh Medical Center for development of their FLASH-CAR™ RNA-based cellular therapies includes utilization of Avalon’s new Point-of-Care Modular Autonomous Processing System (PMAPsys™). First FLASH-CAR™ candidate, AVA-011, on-track for clinical study in B-cell lymphoblastic leukemia and non-Hodgkin’s lymphoma patients. The FLASH-CAR™ technology modifies patients’ T-cells and natural killer (NK) cells using a ribonucleic acid (RNA)-based platform rather than a viral vector, and is being co-developed with the Company’s strategic partner, Arbele Limited. The adaptable FLASH-CAR™ platform can be used to create personalized cell therapies from a patient’s own cells, as well as “off-the-shelf” cell therapies from a universal donor.
By avoiding viral vectors and complicated bio-processing procedures, the FLASH-CAR™ technology significantly reduces manufacturing costs and development times, resulting in more affordable and potentially breakthrough therapies for cancer patients. Avalon’s innovative FLASH-CAR™ technology can be used to generate universal cell therapies that may allow for widespread patient accessibility enabling broader commercial adoption compared to currently available CAR-T cell therapies. Avalon’s first FLASH-CAR™ platform candidate, AVA-011, targets both CD19 and CD22 tumor antigens on cancer cells and is in development for patients with relapsed/refractory B-cell lymphoblastic leukemia and non-Hodgkin’s lymphoma.
“This is an important milestone in the clinical development and production of AVA-011 and other RNA-based FLASH-CAR™ candidates ahead of the start of our first-in-human clinical trials,” said David Jin, M.D., Ph.D., President and Chief Executive Officer of Avalon GloboCare. “We are excited about our partnership on the PMAPsys™ with UPMC, which we believe will help accelerate the path to bringing our own and third-party cell therapies to market. We are working diligently to fast-track development and commercialization of our cellular immune-oncology therapeutic products, while providing the highest quality and safety standards for our patients.”
Avalaon GloboCare (NASDAQ:AVCO) is teaming up with some of the brightest minds in CAR-T and CRISP based technologies including Dr. Yen-Michael Hsu, M.D., Ph.D., Director of the Immunologic Monitoring and Cellular Products Laboratory at the UPMC Hillman Cancer Center and Dr. Shuguang Zhang at MIT’s Media La to rapidly develop potentially lifesaving technological advancements with major commercial applications. Recently some of the smartest money on Wall Street has been extremely bullish on this specific niche including Catherine Wood at ARK invest, in a recent interview she stated that her ARKG (ARK Genomic) ETF has the most potential upside over the next five years as the fund has been deploying billions to companies that produce or enable CRISPR, another Targeted Therapeutics. Over the past few weeks microcap stocks have been under more than usual pressure resulting in Avalon GloboCare NASDAQ (AVCO) to be trading at their 52 week low at just around S1.00 a share. I strongly believe Avalon’s pipe line along with a market cap of less than 100 million creates a highly asymmetric risk reward opportunity.