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Alpine Immune Sciences to Collaborate with Merck on Immuno-Oncology Study to Evaluate ALPN-202 in Combination with KEYTRUDA®
“We are extremely pleased to collaborate with Merck, one of the world’s leading immuno-oncology companies,” said Stanford Peng, M.D., Ph.D., President and Head of R&D of Alpine. “Our prior preclinical studies demonstrated that the combination of ALPN-202 and a PD-1 inhibitor can be particularly advantageous, and this collaboration will greatly enable our ability to pursue this opportunity in the clinic. This study, in conjunction with NEON-1, ALPN-202’s ongoing first-in-human monotherapy trial, will provide insights across a broad spectrum of cancers and lines of therapy.”
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Intellia’s positive interim results make stock market friendly towards gene editing
On June 26th, 2021, Intellia Therapeutics (Intellia; Cambridge MA) announced positive interim results from the phase I dose-escalation trial of its gene-editing program, NTLA-2001, being tested as a treatment for transthyretin (ATTR) amyloidosis. ATTR amyloidosis occurs when liver cells generate mutated TTR proteins that misfold, leading to the accumulation of protein adducts as amyloids. An increase in amyloid formation leads to systemic complications such as neuropathy and cardiomyopathy. NTLA-2001 is designed as a single-dose gene therapy for ATTR amyloidosis to knock out the mutated TTR gene in liver cells
Interim trial data from six patients reveal a mean reduction of 87% in serum TTR levels by day 28 of treatment, with a maximum serum reduction of 96%. This compares favorably with current pharmaceutical treatments for the disease- RNA-silencing therapy from Alnylam and protein stabilizing small molecule therapy from Pfizer, which achieve around 80% reduction. The most well-received (by the stock market) results from the interim data are possibly the sustained lack of side effects, including liver toxicity and immune system overdrive, in ATTR patients. Secondly, the systemic administration of the gene therapy, as opposed to viral vector-based site-specific delivery used by other CRISPR companies such as Editas, indicates easier clinical adoption of the therapy.
Since Intellia’s announcement, the stock market speculation has pivoted from cautious to overtly positive across the spectrum of gene-editing companies, increasing share prices of Intellia by 45% along with others such as Editas, Beam Therapeutics, and CRISPR Therapeutics by 10-15%. Intellia’s market capitalization rose to USD 11 billion from the previous USD 9 billion in a single trading session. A lot of this confidence comes from the relief that gene editing can be safe. There has been a general queasiness about tinkering with genes and their potential unwanted (and possibly irreversible) ramifications that has led even the FDA to take a cautious approach to sanctioning gene-editing based human trials. The Intellia trials have recruited patients from New Zealand and Luxemburg, and not the USA. Intellia’s results are now speculated to open doors for FDA approvals to gene editing. The lack of risk now associated with gene editing only augments its potential to achieve life-long treatments to diseases with unmet needs, including cancer.
Another compelling aspect of the single-dose therapy is the reduction of cost to treatment that is attractive to health insurers. Alnylam’s Onpattro requires three weekly infusions by a clinician, Pfizer’s Vyndamax is a daily-once oral medication costing USD 25,000 annually. NTLA-2001 requires no healthcare infrastructure beyond the single injection, for lifelong treatment. Backing from insurers will give an edge to gene therapy products over competitors, despite the latter’s longer market presence. Interestingly the share price of Anlnylam is down by 10%, since the NTLA-2001 news, while Regeneron- Intellia’s partner for commercializing NTLA-2001- has seen a 4% increase in share price, amid the speculation that Regeneron may eventually acquire Intellia’s gene-editing pipeline and that Alnylam’s ATTR offering will be faced out by Intellia’s program.
In terms of revenue, NTLA-2001 is poised to be a blockbuster speculated to generate upwards of USD 15 billion annually for Intellia. ATTR amyloidosis has been a profitable market for companies generating between USD 306 million (Alnylam’s Onpattro) to USD 429 million (Pfizer’s Vyndamax) annually. One only hopes that NTLA-2001 continues being safe and effective, even against patients with previous exposure to other ATTR therapies (not evaluated in the clinical trials).
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