Clinical-stage genome editing company Intellia Therapeutics Inc. (NTLA:NASDAQ) and biotechnology firm Regeneron Pharmaceuticals Inc. (REGN:NASDAQ), announced "positive interim data from an ongoing Phase 1 clinical study of their lead in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis."
The company advised that the Phase 1 trial being led and conducted by Intellia Therapeutics is designed to evaluate the safety and efficacy of NTLA-2001 in individuals who have been diagnosed with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
The firm stated that "NTLA-2001 is the first CRISPR/Cas9-based therapy candidate to be administered systemically, via intravenous infusion, for precision editing of a gene in a target tissue in humans." Intellia explained further that "NTLA-2001 is designed to inactivate the TTR gene in liver cells to prevent the production of misfolded transthyretin (TTR) protein, which accumulates in tissues throughout the body and causes the debilitating and often fatal complications of ATTR amyloidosis."
The company indicated that the interim data that it released today was collected from the first six ATTRv-PN patients in the Phase 1 study who were members of two single-ascending dose cohorts in the trial being carried out in the U.K. and New Zealand. At present, the trial calls for enrollment of 38 adult patients between the ages of 18 and 80.
The firm said that single doses of 0.1 mg/kg or 0.3 mg/kg of NTLA-2001 were given to the participants with reductions in serum TTR levels measured from baseline through 28 days. The company reported that patient who received the 0.1 mg/kg dosage of NTLA-2001 showed mean reductions of 52% in serum TTR and that those who received a higher dosage of 0.3 mg/kg demonstrated 87% reduction in serum TTR with one patient who showed a 96% reduction. The firm noted that the results compared extremely favorable to the current standard of care for ATTRv-PN that usually yields an average of just 80% reductions in TTR.
The firm noted that later this year, after it is able to identify the most effective recommended dose in the dose-escalation portion of the study, it expects to begin a single-dose expansion cohort in part 2 of the Phase 1 study. The company said that when it finishes the Phase 1 trial, it intends to move to pivotal studies for indications for polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.
Intellia Therapeutics' President and CEO John Leonard, M.D., commented, "These are the first ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR. The interim results support our belief that NTLA-2001 has the potential to halt and reverse the devastating complications of ATTR amyloidosis with a single dose…Solving the challenge of targeted delivery of CRISPR/Cas9 to the liver, as we have with NTLA-2001, also unlocks the door to treating a wide array of other genetic diseases with our modular platform, and we intend to move quickly to advance and expand our pipeline. With these data, we believe we are truly opening a new era of medicine."
Regeneron Pharmaceuticals' President and Chief Scientific Officer George D. Yancopoulos, M.D., Ph.D., stated, "This is exciting early data both for people living with this devastating disease and for the entire scientific community working to maximize the potential of genetics-based medicines through cutting-edge research and technologies…Thanks to large-scale human genetics research, there have been many new genetic targets identified and confirmed to have an impact on human health. Combining this knowledge with the precision and enhanced convenience of a single CRISPR infusion unlocks new possibilities in treating – and potentially even curing – life-threatening and historically difficult-to-address diseases."
Julian Gillmore, M.D., Ph.D., professor of medicine at the National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, U.K., and the Phase 1 study's national coordinating investigator, remarked, "ATTR amyloidosis is a progressive and fatal disease that usually requires chronic, lifelong treatment. These interim Phase 1 data support NTLA-2001 as the only one-time treatment either on the market or in development...As the first-ever systemically administered CRISPR therapy candidate, NTLA-2001 shows strong potential to stop the production and accumulation of the misfolded TTR protein by inactivating the TTR gene at the root of the disease. This approach could deliver life-changing, lifelong benefits to patients with all forms of ATTR amyloidosis, who continue to experience debilitating symptoms and complications of disease while on the standard of care."
Intellia pointed out that "based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first curative treatment for ATTR amyloidosis, adding that NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body."
The company stated that "ATTR amyloidosis is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold." The firm wrote that about 50,000 people globally have ATTRv amyloidosis and around 200,000-500,000 people worldwide have ATTRwt amyloidosis.
Intellia Therapeutics is based in Cambridge, Mass., and described its business as "a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology." The firm stated that it is pursuing two primary approaches in order to fully take advantage of the transformative potential of the CRISPR/Cas9 technology. These include in vivo programs where CRISPR is administered intravenously in precision editing of disease-causing genes directly in target tissues and ex vivo programs where CRISPR is used to by removing, re-engineering and re-infusing the patient's own cells to treat autoimmune diseases and cancer.
Regeneron, headquartered in Tarrytown, N.Y., is a leading biotechnology company with a market capitalization of around $58.6 billion that has earned approval for nine FDA-approved treatments and has many other product candidates in its development pipeline. The firm's approved medicines and those in development are designed to help patients with allergic, cardiovascular eye, infectious, inflammatory and rare diseases, cancer, pain and hematologic conditions.
Intellia Therapeutics started the day with a market cap of around $6.1 billion with approximately 68.15 million shares outstanding and a short interest of about 8.9%. NTLA shares opened almost 58% higher today at $140.15 (+$51.32, +57.77%) over Friday's $88.83 closing price and reached a new 52-week high this morning of $144.40. The stock has traded today between 121.66 and $144.40 per share and is currently trading at $132.73 (+$43.90, +49.42%).
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