NEW: No-Cost StreetSmart Live! Events. This type of intellectual capital is usually not made available to the general public. Learn More

Get the Latest Investment Ideas Delivered Straight to Your Inbox.

TICKERS: ARWR

Arrowhead Shares Point Higher on Positive Interim Data in Phase 2 Liver Disease Trial

Share on Stocktwits

Source:

Shares of Arrowhead Pharmaceuticals traded 48% higher after the firm reported positive interim results from its Phase 2 study of ARO-AAT in patients with alpha-1 antitrypsin deficiency liver disease.

Arrowhead Pharmaceuticals Inc. (ARWR:NASDAQ), which is focused on developing medicines that treat intractable diseases by silencing the genes that cause them, today announced "positive interim 24-week liver biopsy results in four subjects from AROAAT2002, an open-label Phase 2 clinical study of ARO-AAT, the company's second generation investigational RNA interference (RNAi) therapeutic being developed as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD)."

The company stated that the results obtained demonstrate meaningful pharmacodynamic effects by ARO-AAT that led to improvements in several relevant biomarkers. The firm indicated specifically that the results showed "substantial reductions in intra-hepatic mutant AAT protein (Z-AAT), both Z-AAT monomer and Z-AAT polymer, improvements in liver stiffness based on FibroScan and a decrease in alanine aminotransferase (ALT) and gamma-glutamyl transferase (GGT), both serum biomarkers of liver injury."

The company advised that in the pilot AROAAT2002 study of investigational ARO-AAT, serum and total intra-hepatic Z-AAT decreased in all four patients by up to 93% and 95%, respectively, following 24 weeks of treatment. In addition, the firm noted that all four patients showed reductions in ALT and GGT and three of four patients demonstrated reductions from baseline in intra-hepatic Z-AAT polymer, with a maximum reduction of 97%.

Arrowhead Pharmaceuticals Chief Medical Officer Javier San Martin commented, "While we had anticipated that 6 months of treatment with investigational ARO-AAT in the Phase 2 open label study would likely lead to substantial reductions in Z-AAT monomer, the improvements in additional clinically meaningful biomarkers, including reductions in Z-AAT polymer, improvements in FibroScan values, and decreases in ALT and GGT, were more substantial than we expected. These are very exciting results and provide us with increased confidence in the potential of this program. Based on these important data, we are actively assessing our clinical and regulatory path forward, including engaging with the U.S. Food and Drug Administration and other regulatory agencies, to identify areas where the program could potentially be streamlined and accelerated."

AROAAT2002 trial investigator Professor Pavel Strnad, M.D., of University Hospital Aachen in Germany, remarked, "These data are very encouraging and suggest that ARO-AAT may rapidly ameliorate liver injury. It is particularly reassuring to see the decrease in liver enzymes, which suggests that elevations are related to proteotoxic stress that could be addressed with ARO-AAT therapy rather than reflecting co-morbidities. In addition, no major lung events have occurred in this study to date, which indicates that RNAi-based reduction of Z-AAT in the liver has not negatively affected lung function during the treatment period."

Mark Brantly, M.D., scientific director of the Alpha-1 Foundation, added, "The Arrowhead ARO-AAT Phase 2 open label clinical trial is exciting for the Alpha-1 community as it brings forward an intervention for the liver disease associated with Alpha-1 Antitrypsin Deficiency. The interim result of this study demonstrates proof of principle that RNA interference is a promising therapy for the liver disease associated with Alpha-1 Antitrypsin Deficiency."

The company explained that the Phase 2 AROAAT2002 trial is a multi-dose pilot study to assess the responses of around 16 patients with AATD associated liver disease to ARO-AAT. The firm stated that ARO-AAT is also presently being evaluated in an ongoing Phase 2/3 SEQUOIA trial that commenced in August 2019.

Arrowhead Pharmaceuticals, headquartered in Pasadena, Calif., stated that its therapies utilize RNA chemistries and efficient modes of delivery to trigger RNA interference (RNAi) mechanisms in order to induce rapid, deep and durable knockdown of target genes. The firm explained that RNAi is a response characteristic present in living cells that affects the production of a specific protein by inhibiting the expression of a specific gene. Arrowhead claimed that its RNAi-based therapeutics take advantage of this natural gene silencing pathway.

Arrowhead Pharmaceuticals started the day with a market capitalization of around $3.5 billion with approximately 102.3 million shares outstanding and a short interest of about 9.2%. ARWR shares opened 18.5% higher today at $40.05 (+$6.25, +18.49%) over yesterday's $33.80 closing price. The stock has traded today between $40.05 and $53.12 per share and is currently trading at $49.90 (+$16.10, +47.63%).

[NLINSERT]

Disclosure:
1) Stephen Hytha compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.
2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.
3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.
4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.
6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.




Learn More about Streetwise Reports Live

Want to read more about Biotechnology / Pharmaceuticals investment ideas?
Get Our Streetwise Reports Life Sciences Report Newsletter Free and be the first to know!

A valid email address is required to subscribe