Arrowhead Pharmaceuticals Initiates Expanded Access Program for Plozasiran and Announces Upcoming Presentation of Clinical Data
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The initiation of an Expanded Access Program (EAP) for plozasiran by Arrowhead Pharmaceuticals is a significant step for patients with familial chylomicronemia syndrome (FCS), a rare and potentially life-threatening genetic disorder. The EAP allows patients who are not eligible for clinical trials to gain access to investigational treatments. This is particularly important for conditions like FCS, where treatment options are limited. The program's success could lead to increased goodwill towards the company and potentially expedite the drug's approval process if outcomes are positive.
Moreover, the presentation of the SHASTA-2 Phase 2 clinical data at a reputable conference such as the American College of Cardiology signals the company's progress in its research and development pipeline. The data will be scrutinized by the medical community and could influence the stock market's perception of the company's potential for future growth. Positive results could lead to an increase in stock value, while any negative outcomes could have the opposite effect.
Arrowhead's announcement regarding the EAP and the upcoming presentation of Phase 2 data is likely to be closely monitored by investors. The EAP could be seen as a strategic move to generate early real-world data and may positively impact the company's image in the eyes of both patients and investors. If the Phase 2 data presented is compelling, it could lead to increased investor confidence in the company's pipeline and potentially attract further investment.
It's important to note that the costs associated with EAPs are typically significant and the company will need to manage these expenses carefully. Investors should consider the balance between the potential long-term benefits of early access programs and the short-term financial implications. The pharmaceutical sector is highly competitive and Arrowhead's ability to navigate the clinical and regulatory landscape will be critical for its continued success and stock performance.
Arrowhead Pharmaceuticals' engagement in both the EAP and the presentation of new clinical data is indicative of a company actively working to establish a strong position within the lipid disorder treatment market. Familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia are niche but high-need areas and Arrowhead's focus on these could carve out a significant market niche. The EAP, while not directly revenue-generating, may provide early access to treatment for patients, which can enhance the company's reputation and potentially lead to a more favorable regulatory review.
The presentation of Phase 2 data at a major cardiology conference also serves as an important validator of the company's scientific credibility. Should the data be positive, it may lead to increased interest and investment in Arrowhead's broader portfolio, potentially impacting the stock positively. Conversely, any concerns raised by the data could have a detrimental effect. Stakeholders will be keen to understand the broader implications of the clinical findings for the company's market potential and competitive standing.
- Expanded Access Program initiated to make investigational plozasiran available to patients with familial chylomicronemia syndrome
- New SHASTA-2 Phase 2 clinical data in patients with severe hypertriglyceridemia to be presented at ACC.24
“Arrowhead is committed to supporting patients living with various lipid disorders and the physicians who treat them. To that end, we are pleased that Dr. Daniel Gaudet, Professor of Medicine at Université de
Arrowhead is committed to bringing new investigational medicines to patients with serious diseases as quickly and efficiently as possible. The company’s EAP is a potential pathway for patients with serious or immediately life-threatening diseases or conditions to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
The plozasiran EAP is for individuals living with FCS. As with any investigational medicine that has not been approved by regulatory authorities, investigational plozasiran may or may not be effective in treating your diagnosis or condition, and there may be risks associated with its use. If you are a patient or caregiver wishing to know more about this plozasiran EAP for FCS, please discuss this EAP and all treatment options with your treating physician. If you are a treating physician and are seeking information about the plozasiran EAP or would like to request access for a patient, please contact EAP@arrowheadpharma.com.
Details about the ACC.24 presentation are listed below.
American College of Cardiology 73rd Annual Scientific Session & Expo – April 6-8, 2024
Title: Plozasiran (ARO-APOC3), An Investigational RNAi Therapeutic, Demonstrates Profound and Durable Reductions in APOC-3 and Triglycerides (TG) in Patients With Severe Hypertriglyceridemia (SHTG), SHASTA-2 Final Results
Date/Time: April 7, 2024, 9:00 a.m. EDT
Presenter: Daniel Gaudet MD, PhD
Session: Late-Breaking Clinical Trials II
Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website after the oral presentation concludes.
About Plozasiran
Plozasiran, previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of Apolipoprotein C-III (APOC3) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. The goal of treatment with plozasiran is to reduce the level of APOC3, thereby reducing triglycerides and restoring lipids to more normal levels.
In multiple clinical studies, investigational plozasiran demonstrated reductions in triglycerides and multiple atherogenic lipoproteins in patients with familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (SHTG), and mixed dyslipidemia (MD). Plozasiran has demonstrated a favorable safety profile to date with treatment emergent adverse events reported that reflect the comorbidities and underlying conditions of the study populations. Plozasiran is currently being investigated in the Phase 3 PALISADE clinical study in patients with FCS, which is on schedule to be completed in the middle of 2024. Phase 2 studies in patients with SHTG and MD, SHASTA-2 and MUIR respectively, are complete and additional Phase 3 studies are planned to begin shortly.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
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ir@arrowheadpharma.com
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Source: Arrowhead Pharmaceuticals, Inc.
