Priovant Therapeutics, Inc.

First patients have been dosed in CLARITY, a Phase 3 study evaluating brepocitinib in non-anterior non-infectious uveitis (NIU). Brepocitinib has also been granted Fast Track Designation from FDA for NIU. Study initiation follows successful Phase 2 study of brepocitinib in NIU (NEPTUNE) and productive End of Phase 2 Meeting with FDA. NEPTUNE 24-week wide field fluorescein angiography results were presented for first time at the EURETINA conference in Barcelona on September 20; clinically meaningful and dose-dependent improvement on retinal vascular leakage was observed from baseline to week 24, consistent with primary and secondary efficacy endpoints previously shared in the top-line readout. DURHAM, N.C., Sept. 23, 2024 /PRNewswire/ -- Priovant Therapeutics today announced that the first patients have been enrolled in CLARITY, a Phase 3 study evaluating brepocitinib in non-anterior non-infectious uveitis. Brepocitinib has also been granted Fast Track Designation for NIU by FDA. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Brepocitinib is a potential first-in-class dual selective inhibitor of TYK2 and JAK1 under evaluation for multiple highly morbid orphan autoimmune diseases. Brepocitinib is administered as an oral therapy dosed once daily. In a prior Phase 2 study evaluating brepocitinib in non-anterior NIU (NEPTUNE), a dose-dependent treatment benefit was observed on multiple endpoints of clinical significance. On the primary efficacy endpoint of Treatment Failure at six months, brepocitinib 45 mg showed the numerically greatest efficacy benefit observed to date of any therapy evaluated for NIU. Patient-level wide field fluorescein angiography (FA) results on retinal vascular leakage at six months were presented for the first time on September 20 at the EURETINA Congress in Barcelona, Spain. As measured through the Angiography Scoring for Uveitis Working Group (ASUWOG) grading system on wide field FA, brepocitinib 45 mg demonstrated a mean 4.4-point decrease in retinal vascular leakage while the 15 mg arm demonstrated a mean 0.5-point decrease. "FA is the gold standard measurement of posterior segment inflammation in uveitis," said Dilraj Grewal, MD, FASRS, Associate Professor of Ophthalmology, Vitreoretinal Surgery and Uveitis at Duke University and Associate Director of the Duke Reading Center, a comprehensive image reading center that specializes in systematic analysis of ophthalmic images captured in multicenter clinical trials. "The patient-level wide field FA results from the NEPTUNE trial show a clinically meaningful dose-dependent improvement on posterior segment inflammation from treatment with brepocitinib. With these results, the very strong clinical outcomes previously shared are now further supported by dose-dependent improvement on an important biomarker." CLARITY is a global multi-center Phase 3 program comparing brepocitinib 45 mg to placebo in patients with non-anterior NIU. CLARITY will consist of two sub-studies under a single protocol with 150 subjects in each sub-study (300 subjects total) randomized 1:1 to brepocitinib 45 mg and placebo, with a primary endpoint of Time to Treatment Failure. The study is closely modeled on the successful NEPTUNE Phase 2 study and will enroll subjects at sites in the United States, Europe, Asia, Australia, and South America. The initiation of CLARITY follows a productive End of Phase 2 Meeting with FDA. "The initiation of the CLARITY Study is an important milestone for the uveitis medical and patient communities," said Quan Dong Nguyen, MD, MSc, FAAO, FARVO, FASRS, CLARITY investigator and Professor of Ophthalmology at the Byers Eye Institute, and Professor of Medicine and Pediatrics at Stanford University School of Medicine. "Non-infectious uveitis is a devastating disease that, if inadequately managed, often leads to irreversible visual impairment or blindness. Current treatment options fail to meet many patients' needs, and brepocitinib's striking Phase 2 results from the NEPTUNE Study suggest that it has the potential to serve as a transformative therapy in controlling inflammation and improving visual acuity." Patient-level macular edema results at week 24 from the NEPTUNE Study will be presented at the American Academy of Ophthalmology (AAO) annual meeting October 18-21 in Chicago. Top-line 52-week Phase 2 data from NEPTUNE will also be available by the end of the year. In addition to NIU, brepocitinib is being evaluated in a fully enrolled Phase 3 study in dermatomyositis (VALOR). Top-line data from the VALOR Study is expected in the second half of 2025, with an NDA submission to follow if successful. About Priovant Priovant Therapeutics is a biotechnology company dedicated to developing novel therapies for autoimmune diseases with high morbidity and few available treatment options. The company's lead asset is brepocitinib, a dual selective inhibitor of TYK2 and JAK1. Through dual TYK2/JAK1 inhibition, brepocitinib is able to distinctively suppress key cytokines linked to autoimmunity—including type I IFN, type II IFN, IL-6, IL-12, and IL-23—with a single, targeted therapy. Brepocitinib is administered as a once-daily oral therapy. It has been dosed in over 1,400 subjects and has generated positive data in seven Phase 2 studies. Brepocitinib is currently being evaluated in Phase 3 programs for dermatomyositis and non-infectious uveitis. Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking, which are usually identified by the use of words such as "anticipate," "believe," "continue," "could," "estimate," "expect," "intends," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "would" and variations of such words or similar expressions. The words may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Our forward-looking statements include, but are not limited to, statements regarding our or our management team's expectations, hopes, beliefs, intentions or strategies regarding the future, and statements that are not historical facts, including statements about the clinical and therapeutic potential of our products and product candidates, the availability and success of topline results from our ongoing clinical trials and any commercial potential of our products and product candidates. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Contact: Daniel Herz-Roiphe Priovant Therapeutics [email protected] SOURCE Priovant Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Roivant is once again reaping the benefits of its bespoke business model. This time, it’s selling Dermavant to Organon for up to $1.2 billion. Most of that money — $950 million — is available via commercial milestones, according to Wednesday’s announcement . Organon is paying $175 million upfront plus another $75 million if Dermavant’s skin cream Vtama snags FDA approval to treat atopic dermatitis, a decision that’s expected by the end of the year. It will also pay tiered royalties on sales to Dermavant shareholders. “This is another example of Roivant’s ability to offer creative win-win collaborations,” Roivant CEO Matt Gline said in a statement. In the same release, Organon CFO Matthew Walsh said the milestone-heavy deal structure is meant to reinforce the company’s “commitment to disciplined capital allocation.” Vtama, which was first approved to treat plaque psoriasis in May 2022, brought in $18.4 million in sales in the first quarter , a 10% increase from the same period in 2023. Yet prescriptions have more than doubled, from nearly 200,000 as of August 2023 to over 430,000 by July. An approval in atopic dermatitis would likely provide a significant jolt to revenue potential, with Gline saying that Roivant is “excited for Vtama cream to benefit from Organon’s commercial scale.” In January , Dermavant shared updated data that found more than 80% of patients treated across two studies had a greater than 75% improvement on a skin-clearing endpoint. The deal marks the second acquisition of a Roivant property over the last year after it sold Telavant to Roche for $7.1 billion upfront in late 2023. Other subsidiaries like Priovant and its lead asset, brepocitinib, continue to progress. The company has brepocitinib in two Phase 3 studies for dermatomyositis and non-infectious uveitis, respectively, though recruitment in the NIU trial has yet to begin.

VALOR is the largest interventional dermatomyositis study ever conducted. Double-blind placebo-controlled study enrolled 241 subjects randomized 1:1:1 to brepocitinib 30 mg, brepocitinib 15 mg, and placebo. Data are expected in the second half of 2025, with potential dermatomyositis NDA submission to follow. Priovant also continues to rapidly progress development of brepocitinib for non-infectious uveitis, with additional data from the Phase 2 NEPTUNE study and initiation of an NDA-enabling Phase 3 program expected later this year. DURHAM, N.C., July 29, 2024 /PRNewswire/ -- Priovant Therapeutics today announced completion of enrollment of the Phase 3 VALOR study evaluating brepocitinib in dermatomyositis. The study enrolled 241 subjects across 90 sites in four continents, making it the largest interventional dermatomyositis trial ever conducted. Dermatomyositis (DM) is a multi-organ idiopathic inflammatory condition that affects approximately 40,000 adults in the United States. DM is characterized by debilitating muscle weakness and skin lesions. Approximately half of patients report falls and one third require mobility aids (e.g., canes, walkers, wheelchairs). Dermatomyositis rashes often affect large portions of a patient's body including the scalp and can be disfiguring and painful. In addition to skin and muscle manifestations, DM can also impact other organ systems, particularly through interstitial lung disease. The only approved therapies for DM are steroidal products and intravenous immunoglobulin (IVIg). Brepocitinib is a dual selective inhibitor of TYK2 and JAK1 administered orally once daily. Through dual TYK2/JAK1 inhibition, brepocitinib suppresses signaling of multiple pathogenic cytokines that are understood to drive DM disease activity. Brepocitinib has been dosed in over 1,400 subjects and generated positive results in seven Phase 2 studies across multiple autoimmune diseases. "Dermatomyositis is a highly morbid disease for which we have limited treatment options available," said Dr. Ruth Ann Vleugels, M.D., M.P.H., M.B.A., Heidi and Scott C. Schuster Distinguished Chair in Dermatology, Director of the Autoimmune Skin Disease Program and Connective Tissue Disease Clinics at Brigham and Women's Hospital, and Program Director for the Rheumatology-Dermatology Fellowship at Harvard Medical School. "Brepocitinib is a promising investigational therapy with the potential to transform standard of care treatment for dermatomyositis if approved. The rapid enrollment of the VALOR study reflects the myositis medical community's commitment to this crucial goal. We look forward to seeing the study's results next year and to ideally having an excellent therapeutic option for our patients with this debilitating disease." VALOR is a double-blind placebo-controlled study with subjects randomized 1:1:1 to brepocitinib 30 mg, brepocitinib 15 mg, and placebo. The primary endpoint in the VALOR study is the Total Improvement Score (TIS) at 52 weeks. The TIS is a registrational composite endpoint of six measures of DM disease activity. Secondary endpoints include additional measurements of skin and muscle disease, measurements of disease activity in other impacted organ systems, steroid-sparing benefit, and patient-reported quality of life outcomes. Data are anticipated in the second half of calendar year 2025, with a potential NDA submission to follow. "The dermatomyositis patient community is optimistic that the VALOR study will figure prominently in the history of our collective efforts to improve the lives of people living with dermatomyositis," said Lynn Wilson, President of Myositis Support and Understanding (MSU). "Completed enrollment of the VALOR study represents new medical possibilities and much needed hope for so many patients and families." Paula Eichenbrenner, Executive Director of The Myositis Association (TMA), added "People living with dermatomyositis experience an ongoing battle with the debilitating consequences of their disease, and novel treatment options are desperately needed. Successful enrollment of the VALOR study brings excitement, and we are grateful to the dermatomyositis community for embracing the opportunity to advance understanding of this complex condition." Priovant also continues to advance brepocitinib's development for treatment of non-infectious uveitis (NIU), another highly morbid autoimmune disease with significant unmet need. NIU is the fourth leading cause of blindness among the working-age population in the developed world. Primary endpoint data from the successful Phase 2 NEPTUNE study were recently presented at the American Society of Retina Specialists (ASRS) meeting, with additional 24-week results on vascular leakage and macular edema accepted as oral presentations at the upcoming EURETINA Congress September 19-22 and American Academy of Ophthalmology (AAO) annual meeting October 18-21. Priovant expects to share 52-week top-line results from the NEPTUNE study and initiate an NDA-enabling Phase 3 program by the end of 2024. "Priovant continues to make progress towards our goal of developing brepocitinib as a transformational therapy for highly morbid autoimmune diseases where the need for novel efficacious therapies is greatest," said Ben Zimmer, Priovant CEO. "We deeply appreciate the time and commitment of the patients, investigators, and site research staff who enable this important work. We look forward to providing further updates as we reach upcoming milestones in 2024 and 2025." About Priovant Priovant Therapeutics is a biotechnology company dedicated to developing novel therapies for autoimmune diseases with high morbidity and few available treatment options. The company's lead asset is brepocitinib, a dual selective inhibitor of TYK2 and JAK1. Through dual TYK2/JAK1 inhibition, brepocitinib is able to distinctively suppress key cytokines linked to autoimmunity—including type I IFN, type II IFN, IL-6, IL-12, and IL-23—with a single, targeted therapy. Brepocitinib is administered as a once-daily oral therapy. It has been dosed in over 1,400 subjects and has generated positive data in seven Phase 2 studies. Brepocitinib is currently being evaluated in a Phase 3 program for dermatomyositis and is entering a Phase 3 program for non-infectious uveitis. Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking, which are usually identified by the use of words such as "anticipate," "believe," "continue," "could," "estimate," "expect," "intends," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "would" and variations of such words or similar expressions. The words may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Our forward-looking statements include, but are not limited to, statements regarding our or our management team's expectations, hopes, beliefs, intentions or strategies regarding the future, and statements that are not historical facts, including statements about the clinical and therapeutic potential of our products and product candidates, the availability and success of topline results from our ongoing clinical trials and any commercial potential of our products and product candidates. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Contact: Daniel Herz-Roiphe Priovant Therapeutics [email protected] SOURCE Priovant Therapeutics