Apellis Pharmaceuticals Inc. (APLS:NASDAQ) today announced that it had achieved positive results in the company's Phase 3 PEGASUS study, which is evaluating pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH).
The firm advised in the report that the top-line data show that pegcetacoplan met the study's primary efficacy endpoint and demonstrated superiority compared to patients who were treated with eculizumab. The company noted that pegcetacoplan also showed promising results in key secondary endpoints specifically meeting non-inferiority on transfusion avoidance and absolute reticulocyte count.
The company's Chief Medical Officer Federico Grossi, M.D., Ph.D., commented, "Pegcetacoplan is the first and only investigational therapy to demonstrate superiority compared to eculizumab on hemoglobin levels. We are also excited to see that 85% of patients treated with pegcetacoplan were transfusion free...At Apellis, we are focused on developing groundbreaking therapies, and these results show that pegcetacoplan has the potential to transform the lives of people with PNH. We look forward to meeting with regulators in the first half of the year to discuss next steps."
Peter Hillmen, M.B., Ch.B., Ph.D., professor of experimental hematology at the University of Leeds and an investigator in the PEGASUS study, added, "The majority of patients with PNH currently receiving treatment with eculizumab have continuing anemia...The PEGASUS results show that pegcetacoplanhas the potential to become a new standard of care for patients with PNH."
In regards to the PEGASUS study, Apellis Pharma's Co-Founder and CEO Cedric Francois, M.D., Ph.D., remarked, "Going into the study, our most optimistic expectation was to see a 2 g/dL or more change in hemoglobin and a trend on the key secondary endpoints. Needless to say, we are thrilled with these results...These data give us strong confidence in the further development of pegcetacoplan as a targeted C3 inhibitor in geographic atrophy and other serious complement-driven diseases."
The firm explained in the report that "PNH is a rare, chronic, life-threatening blood disorder associated with abnormally low hemoglobin levels due to the destruction of oxygen-carrying red blood cells (hemolysis) and that persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea)."
The company identifies pegcetacoplan (APL-2) as "an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases." In addition to the study for use in PNH, the firm stated that it is also studying pegcetacoplan in several other clinical studies in geographic atrophy (GA), and C3 glomerulopathy and indicated that the FDA has granted Fast Track designation for pegcetacoplan in the treatment of both PNH and GA.
Apellis Pharmaceuticals is headquartered in Waltham, Mass., and is a global biopharmaceutical company that is engaged in pioneering targeted C3 therapies. The firm focuses in the areas of hematology, ophthalmology and nephrology.
Apellis Pharmaceuticals has a market capitalization of around $1.9 billion with approximately 63.89 million shares outstanding and a short interest of about 8.3%. APLS shares opened 35% higher today at $40.89 (+$10.70, +35.49%) over yesterday's $30.19 closing price and reached a new 52-week high price this morning of $42.48. The stock has traded today between $34.61 and $42.48/share and is currently trading at $37.50 (+$7.31, +24.21%).
[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 interview or the decision to write an article until three business days after the publication of the interview or 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.
