Allopurinol

Event will take place on Wednesday, June 26, 2024 in New York CARMIEL, Israel, June 13, 2024 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell based protein expression system, today announced that it will host an in-person investor day at the Lotte Palace New York Hotel in New York on Wednesday, June 26, 2024 at 8:30 a.m. Eastern Daylight Time (EDT). The investor day will feature presentations by the following key opinion leaders: Aleš Linhart, DSc, FESC (Charles University, Prague), who will discuss the treatment landscape for Fabry disease and perspectives on Elfabrio® (pegunigalsidase alfa), a plant cell-expressed PEGylated recombinant α-Galactosidase-A enzyme approved for adult patients with Fabry disease. Naomi Schlesinger, MD (University of Utah), who will discuss the current treatment landscape for uncontrolled gout and top-line results from the First-in-Human Phase I single ascending dose clinical trial of PRX-115, a plant cell-expressed recombinant PEGylated uricase. In addition, the event will include a corporate overview and strategy presentation by Dror Bashan, Protalix's President and Chief Executive Officer. A live question and answer session will follow the formal presentations. To register for the event, please click here. About Aleš Linhart, DSc, FESC Aleš Linhart, DSc, FESC is currently heading the Department of Cardiovascular Medicine of the First Medical Faculty and General University in Prague, Czech Republic and serves as vice-dean for international affairs of the First Faculty of Medicine of Charles University in Prague. He obtained his MD degree at Charles University in Prague, received his training in cardiology and vascular medicine at General University Hospital in Prague and Broussais Hospital in Paris, France. In 2004 he was appointed professor at Charles University in Prague. His research focuses mainly on Fabry disease, metabolic cardiomyopathies, noninvasive cardiac imaging, and atherosclerosis. He is member of several scientific societies, namely Czech and European Society of Cardiology (ESC). He served as the chairman of the Working Group on Myocardial and Pericardial Diseases of the ESC and is the immediate past president of the Czech Society of Cardiology. He authored or co-authored more than 450 scientific peer-reviewed papers, 85 book chapters, and three monographs. About Naomi Schlesinger, MD Naomi Schlesinger, MD is the Harold J, Ardella T, and Helen T Stevenson Presidential Endowed Chair of Rheumatology, Professor and Chief Division of Rheumatology, Spencer Fox Eccles School of Medicine, University of Utah, Salt Lake City, UT. She is a previous Chief of the Division of Rheumatology at Rutgers-Robert Wood Johnson Medical School and Director of Rutgers Robert Wood Johnson Medical School Gout Center. Dr. Schlesinger is a previous President of the NJ Rheumatology Association and current President of the Utah Rheumatology Association and was the recipient of the 2015 Rheumatologist of The Year award by the Arthritis Foundation - NJ Chapter. Dr. Schlesinger is a noted authority in the field of gout, having published many papers regarding the diagnosis, treatment, and better understanding of the pathogenesis of gout. Dr. Schlesinger's research has won recognition, including the work titled: Efficacy of canakinumab (ACZ885), a fully human anti-Interleukin (IL)-1beta monoclonal antibody, in the prevention of flares in gout patients initiating allopurinol therapy, which was selected as one of the five highest-ranking abstracts that will likely shape our treatment paradigms for years to come in the 2010 ACR/ ARHP Annual Scientific Meeting and the work titled: Erectile dysfunction is common among gout patients, which was selected (one of 13) for inclusion in the official 2014 EULAR Press Conference from over 4000 abstracts. Other pioneering work includes the treatment of gout flares with topical ice, seasonality of gout, diagnosing gout using ultrasound, understanding the pathogenesis of bone erosions in gout, and the importance of anti-inflammatory treatment in gout. Dr. Schlesinger serves as the American College of Rheumatology (ACR) abstract Co-Chair on metabolic and crystal arthropathies, a member of the ACR Global Engagement Special Committee (GESC), and a member of the ACR Annual Meeting Planning Committee (AMPC). She is a Co-Director of the International Gout and Hyperuricemia Center, Third Affiliated Hospital of Sun Yat-sen University, Guangzhou, China. She serves as a consultant to pharmaceutical companies in the field of gout. About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, was approved by both the FDA and the European Medicines Agency in May 2023. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio. Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX-115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; PRX-119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Investor Contact Mike Moyer, Managing Director LifeSci Advisors 617 308 4306 [email protected] Logo: SOURCE Protalix BioTherapeutics, Inc.

Fortress‘ late-stage pipeline continues to advance and may generate up to three regulatory approvals on NDAs and BLAs in the next 12 months and potentially a fourth BLA filing as early as 2025 FDA accepted New Drug Application filing for DFD-29 to treat inflammatory lesions and erythema of rosacea in adults; PDUFA goal date of November 4, 2024 MIAMI, May 15, 2024 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue, today announced financial results and recent corporate highlights for the first quarter ended March 31, 2024. Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, “We achieved first quarter year-over-year product revenue growth of 7%, which was driven by greater than 20% year-over-year growth in our flagship products, Qbrexza® and Accutane®. In the first quarter of 2024, the U.S. Food and Drug Administration (“FDA”) accepted the New Drug Application (“NDA”) filing for DFD-29 and set a Prescription Drug User Fee Act (“PDUFA”) goal date of November 4, 2024. If approved, DFD-29 has the potential to be the only oral, systemic therapy to address inflammatory lesions and erythema (redness) from rosacea, differentiating it as a potential best-in-class solution for the millions of patients suffering from rosacea. We also dosed the first patient in a multi-center Phase 2 study for Triplex for control of cytomegalovirus (“CMV”) in patients undergoing liver transplantation and received grant funding from the National Institutes of Health (“NIH”) to further advance cell and gene therapy candidates for the potential treatment of adults living with HIV and children with Menkes disease. Looking ahead, our expansive portfolio of development-stage programs across multiple areas, including oncology, dermatology, and rare diseases, holds the potential for up to three NDA and Biologics License Application (“BLA”) regulatory approvals within the next 12 months and potentially a fourth BLA filing as early as 2025. Additionally, we anticipate multiple data readouts this year, including topline data from the Phase 1b/2a clinical trial of AJ201 to treat spinal and bulbar muscular atrophy (“SBMA”), data from the Phase 1b clinical trial of dotinurad for the treatment of gout and hyperuricemia and topline Phase 2 clinical data of Triplex, a CMV vaccine for adults co-infected with HIV and CMV. This sustained progress underscores the strength of Fortress’ business model, centered on acquiring and advancing assets that address unmet medical needs and enhance long-term value for shareholders through product revenues, equity holdings and royalties.” Recent Corporate Highlights1: Regulatory Milestones and Updates In March 2024, the FDA accepted the NDA for DFD-29 (Minocycline Hydrochloride Modified Release Capsules, 40 mg) and set a PDUFA goal date of November 4, 2024. We submitted the NDA to the FDA seeking approval for DFD-29 for the treatment of inflammatory lesions and erythema of rosacea in adults in January 2024. Both double blinded, randomized controlled DFD-29 Phase 3 clinical trials achieved their co-primary and all secondary endpoints with subjects completing the 16-week treatment with no significant safety issues. DFD-29 demonstrated statistical superiority compared to both Oracea capsules and placebo for Investigator’s Global Assessment (IGA) treatment success and the reduction in the total inflammatory lesion count in both clinical trials. Additionally, DFD-29 showed significantly superior reduction in Clinicians Erythema Assessment compared to placebo in both of the Phase 3 clinical trials. DFD-29 is currently in development at our partner company, Journey Medical Corporation (Nasdaq: DERM) (“Journey Medical”). The CUTX-101 rolling NDA submission is ongoing and is expected to be completed by our partner, Sentynl Therapeutics, Inc. in 2024. Cyprium, our subsidiary company that developed CUTX-101, will retain 100% ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101. We submitted a BLA to the FDA for cosibelimab, our investigational anti-PD-L1 antibody, as a treatment for patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or radiation, in January 2023. In December 2023, the FDA issued a complete response letter (“CRL”) for the cosibelimab BLA. The CRL solely cited findings that arose during a multi-sponsor inspection of a third-party contract manufacturing organization as approvability issues to address in a resubmission. The CRL did not state any concerns about the clinical data package, safety or labeling for the approvability of cosibelimab. We intend to seek to address the feedback in a potential BLA resubmission, which is currently targeted for mid-year. Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc. (Nasdaq: CKPT) (“Checkpoint”). Based on its public statements, AstraZeneca plc has estimated that it expects the FDA to accept its BLA submission of CAEL-101 (anselamimab) to treat AL amyloidosis for review as early as 2025. Clinical Updates The Phase 2 clinical trial of Triplex, a CMV vaccine, for adults co-infected with HIV and CMV is now fully enrolled with topline data anticipated in the fourth quarter of 2024. The study aims to show that vaccination with Triplex can safely elicit a CMV-specific immune response and reduce asymptomatic CMV replication in a population of people with HIV on suppressive antiretroviral therapy. The study will also evaluate whether this intervention might reduce chronic inflammation and immune activation, as compared to placebo, and thus, potentially reduce related mortality and morbidity. In May 2024, we announced that the first patient was dosed in a multi-center, placebo-controlled, randomized Phase 2 study of Triplex for control of CMV in patients undergoing liver transplantation. The trial is funded by a grant from the NIH’s National Institute of Allergy and Infectious Diseases of the (NIH/NIAID) that could provide over $20 million in non-dilutive funding. Triplex is currently in development at our subsidiary company, Helocyte, Inc. A Phase 1b clinical trial in patients with gout and hyperuricemia is ongoing in the U.S. to confirm the comparability of U.S. patients’ response to dotinurad (urate transporter (URAT1) inhibitor) with data generated in Japan, and to assess drug-drug interactions, if any, with allopurinol. We expect to announce data from this trial in mid-2024. Dotinurad is currently in development at our subsidiary company, Urica Therapeutics, Inc. (“Urica”). Commercial Product Updates Journey Medical’s total revenues for the first quarter ended March 31, 2024 were $13.0 million, an increase of $0.8 million, or 7%, compared to total net revenues of $12.2 million for the first quarter ended March 31, 2023. General Corporate: In January 2024, Fortress raised gross proceeds of approximately $11.0 million in a registered direct offering priced at-the-market under Nasdaq rules. In January 2024, Checkpoint raised gross proceeds of approximately $14.0 million in a registered direct offering, and Avenue raised approximately $5.0 million gross proceeds from warrant exercise transactions. Financial Results: As of March 31, 2024, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $85.8 million, compared to $83.4 million as of December 31, 2023, an increase of $2.5 million during the quarter. Fortress’ consolidated cash, cash equivalents and restricted cash, totaling $85.8 million as of March 31, 2024, includes $45.6 million attributable to Fortress and the private subsidiaries, $3.2 million attributable to Avenue, $11.2 million attributable to Checkpoint, $1.7 million attributable to Mustang Bio and $24.1 million attributable to Journey Medical. Fortress’ consolidated cash, cash equivalents and restricted cash, totaled $83.4 million as of December 31, 2023, which included $42.2 million attributable to Fortress and private subsidiaries, $1.8 million attributable to Avenue, $4.9 million attributable to Checkpoint, $7.0 million attributable to Mustang Bio and $27.4 million attributable to Journey Medical. Subsequent to the end of the first quarter, in May 2024, Avenue raised approximately $4.4 million in gross proceeds from warrant exercise transactions and Mustang raised approximately $4.0 million in gross proceeds from a public offering of common stock and warrants. Fortress’ consolidated net revenue totaled $13.0 million for the first quarter ended March 31, 2024, all of which was generated from our marketed dermatology products. This compares to consolidated revenue totaling $12.4 million for the first quarter of 2023, which included $12.2 million in revenue generated from our marketed dermatology products. Consolidated research and development expenses including license acquisitions totaled $24.8 million for the first quarter ended March 31, 2024, compared to $39.5 million for the first quarter ended March 31, 2023. Consolidated selling, general and administrative costs were $17.9 million for the first quarter ended March 31, 2024, compared to $25.3 million for the first quarter ended March 31, 2023. Consolidated net loss attributable to common stockholders was $(17.7) million, or $(1.03) per share, for the first quarter ended March 31, 2024, compared to net loss attributable to common stockholders of $(23.5) million, or $(3.47) per share for the first quarter ended March 31, 2023. All share and per share information has been retroactively adjusted to give effect to the Company’s October 2023 1-for-15 reverse stock split for all periods presented, unless otherwise indicated. About Fortress Biotech Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 20 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Fortress’ portfolio is being commercialized and developed for various therapeutic areas including oncology, dermatology, and rare diseases. Fortress’ model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand and advance the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, Nationwide Children’s Hospital and Sentynl. For more information, visit . Forward-Looking Statements Statements in this press release that are not descriptions of historical facts are “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. The words “anticipates,” “believes,” “can,” “continue,” “could,” “estimates,” “expects,” “intends,” “may,” “might,” “plans,” “potential,” “predicts,” “should,” or “will” or the negative of these terms or other comparable terminology are generally intended to identify forward-looking statements. These forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include risks relating to: our growth strategy, financing and strategic agreements and relationships; our need for substantial additional funds and uncertainties relating to financings; our ability to identify, acquire, close and integrate product candidates successfully and on a timely basis; our ability to attract, integrate and retain key personnel; the early stage of products under development; the results of research and development activities; uncertainties relating to preclinical and clinical testing; our ability to obtain regulatory approval for products under development; our ability to successfully commercialize products for which we receive regulatory approval; our ability to secure and maintain third-party manufacturing, marketing and distribution of our and our partner companies’ products and product candidates; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein. Company Contact: Jaclyn Jaffe Fortress Biotech, Inc. (781) 652-4500 ir@fortressbiotech.com Media Relations Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com FORTRESS BIOTECH, INC. AND SUBSIDIARIES Unaudited Condensed Consolidated Balance Sheets ($ in thousands except for share and per share amounts) March 31, December 31, 2024 2023 ASSETS Current assets Cash and cash equivalents $ 83,774 $ 80,927 Accounts receivable, net 9,799 15,222 Inventory 10,580 10,206 Other receivables - related party 324 167 Prepaid expenses and other current assets 12,071 10,500 Total current assets 116,548 117,022 Property, plant and equipment, net 6,128 6,505 Operating lease right-of-use asset, net 16,462 16,990 Restricted cash 2,063 2,438 Intangible assets, net 19,473 20,287 Other assets 3,971 4,284 Total assets $ 164,645 $ 167,526 LIABILITIES AND STOCKHOLDERS’ EQUITY (DEFICIT) Current liabilities Accounts payable and accrued expenses $ 76,379 $ 73,562 Income taxes payable 843 843 Common stock warrant liabilities 689 886 Operating lease liabilities, short-term 2,601 2,523 Partner company convertible preferred shares, short-term, net 4,021 3,931 Partner company installment payments - licenses, short-term, net 3,000 3,000 Other short-term liabilities 163 163 Total current liabilities 87,696 84,908 Notes payable, long-term, net 61,420 60,856 Operating lease liabilities, long-term 17,619 18,282 Other long-term liabilities 1,847 1,893 Total liabilities 168,582 165,939 Commitments and contingencies Stockholders’ equity (deficit) Cumulative redeemable perpetual preferred stock, $0.001 par value, 15,000,000 authorized, 5,000,000 designated Series A shares, 3,427,138 shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively, liquidation value of $25.00 per share 3 3 Common stock, $0.001 par value, 200,000,000 shares authorized, 19,375,343 and 15,093,053 shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively 19 15 Additional paid-in-capital 733,290 717,396 Accumulated deficit (710,287 ) (694,870 ) Total stockholders' equity attributed to the Company 23,025 22,544 Non-controlling interests (26,962 ) (20,957 ) Total stockholders' equity (deficit) (3,937 ) 1,587 Total liabilities and stockholders' equity (deficit) $ 164,645 $ 167,526 FORTRESS BIOTECH, INC. AND SUBSIDIARIES Unaudited Condensed Consolidated Statements of Operations ($ in thousands except for share and per share amounts) Three Months Ended March 31, 2024 2023 Revenue Product revenue, net $ 13,030 $ 12,165 Collaboration revenue — 181 Revenue - related party — 35 Other revenue — 48 Net revenue 13,030 12,429 Operating expenses Cost of goods sold - product revenue 6,816 6,449 Research and development 24,839 35,276 Research and development - licenses acquired — 4,230 Selling, general and administrative 17,941 25,341 Total operating expenses 49,596 71,296 Loss from operations (36,566 ) (58,867 ) Other income (expense) Interest income 833 1,036 Interest expense and financing fee (2,602 ) (4,296 ) Gain (loss) on common stock warrant liabilities (667 ) 6,678 Other income (expense) (21 ) 304 Total other income (expense) (2,457 ) 3,722 Net loss (39,023 ) (55,145 ) Net loss attributable to non-controlling interests 23,606 33,608 Net loss attributable to Fortress $ (15,417 ) $ (21,537 ) Net loss attributable to common stockholders $ (17,731 ) $ (23,545 ) Net loss per common share attributable to common stockholders - basic and diluted $ (1.03 ) $ (3.47 ) Weighted average common shares outstanding - basic and diluted 17,151,945 6,792,376 1 The development programs depicted in this press release include product candidates in development at Fortress, at Fortress’ private subsidiaries (referred to herein as “subsidiaries”), at Fortress’ public subsidiaries (referred to herein as “partner companies”) and at entities with whom one of the foregoing parties has a significant business relationship, such as an exclusive license or an ongoing product-related payment obligation (such entities referred to herein as “partners”). The words “we”, “us” and “our” may refer to Fortress individually, to one or more of our subsidiaries and/or partner companies, or to all such entities as a group, as dictated by context.