FDA Grants Latozinemab Breakthrough Therapy Designation for Frontotemporal Dementia Due to a Progranulin Gene Mutation (FTD-GRN)
- Latozinemab is the most advanced progranulin-elevating candidate in development for FTD-GRN
- The FDA granted Breakthrough Therapy Designation to latozinemab based on data from the INVOKE-2 Phase 2 clinical trial
- Latozinemab is currently being studied in the pivotal INFRONT-3 Phase 3 study
- None.
Insights
The FDA's Breakthrough Therapy Designation for latozinemab represents a significant milestone in the treatment of frontotemporal dementia with a progranulin gene mutation (FTD-GRN). This condition is a rare and rapidly progressing form of dementia and the designation reflects the potential of latozinemab to address a critical unmet medical need. The designation is based on the INVOKE-2 Phase 2 clinical trial data, which suggests that latozinemab may offer substantial improvement over existing therapies, of which there are currently none approved by the FDA.
From a medical research perspective, the underlying mechanism of action involves the elevation of progranulin levels, a protein implicated in cell survival and inflammation regulation. Latozinemab's targeting of sortilin to achieve this suggests a novel therapeutic approach, which could have broader implications for neurodegenerative diseases. The ongoing INFRONT-3 Phase 3 study will be crucial in determining the efficacy and safety profile of latozinemab, which will be pivotal for its potential market approval and subsequent adoption in clinical practice.
The Breakthrough Therapy Designation for latozinemab is likely to have a positive impact on the business prospects of Alector, Inc. and its partner GSK plc. This status can lead to expedited development and review processes, potentially reducing the time to market and associated costs. For investors, this development is significant as it could enhance the companies' valuation due to the anticipation of a new revenue stream, especially considering the lack of approved treatments for FTD-GRN.
However, it is important to note that the actual financial impact will depend on the results of the pivotal Phase 3 study, regulatory approval and the ability of Alector and GSK to successfully commercialize the drug. The rarity of the disease also means that the target market is relatively small, which could limit the peak sales potential, although orphan drug status often allows for premium pricing strategies.
FTD-GRN represents a niche but significant segment within the neurodegenerative disease market. The Breakthrough Therapy Designation for latozinemab by the FDA signals a strong therapeutic promise, potentially positioning Alector and GSK at the forefront of this market. The designation can also facilitate increased interest from investors, partnerships and possibly drive further research and development in related conditions.
Market dynamics for orphan drugs like latozinemab often involve high treatment costs and reimbursement challenges. It will be essential for Alector and GSK to navigate these challenges effectively. Market research will play a crucial role in understanding the size of the affected population, the potential market penetration rate and the competitive landscape, which currently lacks FDA-approved alternatives.
-Latozinemab is the most advanced progranulin-elevating candidate in development for FTD-GRN and has now become the first investigational medicine to receive a Breakthrough Therapy Designation for the treatment of FTD-GRN-
SOUTH SAN FRANCISCO, Calif., Feb. 07, 2024 (GLOBE NEWSWIRE) -- Alector, Inc. (Nasdaq: ALEC) and GSK plc (LSE/NYSE: GSK) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to latozinemab, an investigational human monoclonal antibody designed to block sortilin to elevate progranulin (PGRN) levels for the potential treatment of frontotemporal dementia with a progranulin gene mutation (FTD-GRN).
“In partnership with GSK, we are encouraged and excited by this FDA Breakthrough Designation. FTD-GRN is a rare and rapidly progressing neurodegenerative disease and one of the most common causes of early onset dementia," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. “With this designation, we look forward to continued productive conversations with the FDA, recognizing the unmet need for people living with FTD-GRN, a serious condition for which there are no FDA-approved treatment options available. Latozinemab, the most advanced progranulin-elevating candidate in clinical development for FTD-GRN, is currently being studied in the pivotal INFRONT-3 Phase 3 study, which achieved target enrollment in October 2023.”
The FDA granted latozinemab Breakthrough Therapy Designation for FTD-GRN based upon data from the INVOKE-2 Phase 2 clinical trial of latozinemab in FTD-GRN participants. The FDA's Breakthrough Therapy Designation is granted to expedite the development and review of drugs in the United States that are intended to treat a serious condition, when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).1
About Latozinemab
Latozinemab (AL001) is an investigational human monoclonal antibody designed to modulate progranulin (PGRN), a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, including frontotemporal dementia (FTD), Alzheimer’s disease, and Parkinson’s disease. Latozinemab aims to increase the level of PGRN in humans by inhibiting sortilin, a degradation receptor for PGRN. Latozinemab has received Orphan Drug Designation for the treatment of FTD as well as both Breakthrough Therapy and Fast Track designations for the treatment of FTD due to a progranulin gene mutation (FTD-GRN) from the U.S. Food and Drug Administration.
About Frontotemporal Dementia (FTD)
Frontotemporal dementia (FTD) is a rare neurodegenerative disease, but it is one of the most common causes of early onset dementia..2 It affects an estimated 50,000 to 60,000 people in the United States and roughly 110,000 in the European Union, with potentially higher prevalence in Asia and Latin America.3,4 There are multiple heritable forms of FTD, and FTD patients with a progranulin gene mutation (FTD-GRN) represent
Collaboration with GSK
In July 2021, Alector entered into a collaboration and license agreement with GSK (NYSE: GSK) to collaborate on the global development and commercialization of progranulin-elevating monoclonal antibodies, including latozinemab and AL101 (GSK4527226). Under the terms of the GSK agreement, Alector received
About Alector
Alector is a clinical-stage biotechnology company pioneering immuno-neurology, a novel therapeutic approach for the treatment of neurodegenerative diseases. Immuno-neurology targets immune dysfunction as a root cause of multiple pathologies that are drivers of degenerative brain disorders. Alector has discovered and is developing a broad portfolio of innate immune system programs, designed to functionally repair genetic mutations that cause dysfunction of the brain’s immune system and enable rejuvenated immune cells to counteract emerging brain pathologies. Alector’s immuno-neurology product candidates are supported by biomarkers and seek to treat indications, including Alzheimer’s disease and genetically defined frontotemporal dementia patient populations. Alector is headquartered in South San Francisco, California. For additional information, please visit www.alector.com.
About GSK
GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at gsk.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements regarding our product candidates, the expectation regarding the implications of Breakthrough Therapy Designation, planned and ongoing preclinical studies and clinical trials, expected milestones, including the timing of data from our INFRONT-3 trial, and expectations of our collaborations. Such statements are subject to numerous risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to risks and uncertainties as set forth in Alector’s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q, as well as the other documents Alector files from time to time with the Securities and Exchange Commission. These documents contain and identify important factors that could cause the actual results for Alector to differ materially from those contained in Alector’s forward-looking statements. Any forward-looking statements contained in this press release speak only as of the date hereof, and Alector specifically disclaims any obligation to update any forward-looking statement, except as required by law.
REFERENCES
- U.S. Food and Drug Administration (FDA). Breakthrough Therapy.
- The Association for Frontotemporal Degeneration (AFTD).
- Patient estimates based on internal forecasting analysis using published literature sources.
- E.U. estimates include EU5 countries only (Spain, Italy, France, U.K. and Germany).
- FTD Disorders Registry.
- Moore KM, Nicholas J, Grossman M, et al. Lancet Neurol. 2020 Feb; 19 (2).
Alector Contacts:
Alector
Katie Hogan
202-549-0557
katie.hogan@alector.com
1AB (media)
Dan Budwick
973-271-6085
dan@1abmedia.com
Argot Partners (investors)
Laura Perry
Argot Partners
212-600-1902
alector@argotpartners.com
FAQ
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