Graft vs Host Disease

HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced that it has achieved the first research milestone criterion in a collaboration with Alexion, AstraZeneca Rare Disease (“Alexion”) to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease. (Related release is here). The achievement of a predefined research milestone triggers a milestone payment to JCR. JCR recently strengthened its collaboration with Alexion to address unmet needs by initiating a second research collaboration in December 2023 to apply the same technology to oligonucleotide therapeutics. (Related release is here). The milestone payment has a minor impact on JCR’s consolidated financial results for this fiscal year ending on March 31, 2024. About the J-Brain Cargo® Platform Technology J-Brain Cargo® is a proprietary blood-brain barrier (BBB)-penetrating technology to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for a lysosomal storage disorder (LSD) is IZGARGO® (INN: pabinafusp alfa) for the treatment of Mucopolysaccharidosis (MPS) type II (Hunter Syndrome). Based on the same platform technology, JR-141(INN: pabinafusp alfa), JR-171 for the treatment of MPS I (INN: lepunafusp alfa) and JR-441 for the treatment of MPS IIIA have advanced into global clinical trials. JCR intends to start clinical trials with four additional programs from its LSD pipeline by FY2028. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is redefining expectations and expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 49-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, Fabry disease, acute graft-versus host disease, MPS II, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II (Hunter syndrome), MPS III A and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical progress at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. Together we soar. For more information, please visit Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.

ROCKVILLE, Md., March 19, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced that it plans to host a conference call on Monday, March 25, 2024, at 8:30 a.m. ET to discuss its financial results for the full year ended December 31, 2023 and provide a corporate update. Individuals may participate in the live call via telephone by dialing 1-877-451-6152 (domestic) or 1-201-389-0879 (international) and using the conference ID: 13744453. Participants are asked to dial-in 15 minutes before the start of the call to register. Investors and the public can access the live and archived webcast of this call via the “News & Media” section of the company’s website, , under “Events” or by clicking here, up to 90 days after the call. About Theriva™ Biologics, Inc. Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include: (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at .

DALLAS--(BUSINESS WIRE)-- Forte Biosciences, Inc. ( ) (NASDAQ: FBRX), a clinical-stage biopharmaceutical company focused on autoimmune and autoimmune-related diseases, today announced its 2023 results and provided a business update. 2023 Business Highlights “Forte achieved a major milestone by advancing FB-102 into the clinic and beating the timelines we targeted in our third quarter business update. The single ascending dose (SAD) portion of the FB-102 phase 1 trial was successfully completed and dosing has begun in the multiple ascending dose (MAD) cohorts.” said Paul Wagner, Ph.D., Chairman and Chief Executive Officer of Forte Biosciences. “I am deeply appreciative of the talented Forte team that made this achievement possible. I also want to thank our investors who believe in the potential of FB-102 and in the Forte team as we continue to advance this exciting program. We look forward to providing more clinical updates on FB-102 over the course of this year.” 2023 Operating Results Research and development expenses were $21.9 million for the year ended December 31, 2023, compared to $5.6 million during the same period in 2022. The increase of $16.3 million was primarily due to a net increase of approximately $9.9 million in manufacturing expense, a net increase of approximately $6.0 million in preclinical and clinical expenses as our FB-102 program entered the clinic, and a net increase in payroll and related expenses of approximately $0.4 million primarily due to an increase in headcount. General and administrative expenses were $10.6 million for the year ended December 31, 2023, compared to $8.3 million for the same period in 2022. The increase of $2.3 million was primarily due to an increase in professional and legal expenses of $2.6 million, an increase in other expenses of $0.4 million, including rent and personnel expenses, partially offset by a reduction in stock-based compensation expense of $0.7 million. Net losses per share were ($1.00) and ($0.80) for the years ended December 31, 2023 and 2022, respectively. Forte ended 2023 with approximately $37.1 million in cash and cash equivalents. Forte had approximately 36.3 million shares of common stock outstanding as of December 31, 2023. CONSOLIDATED BALANCE SHEETS (in thousands except share and par value data) December 31, 2023 December 31, 2022 Assets Current assets: Cash and cash equivalents $ 37,125 $ 41,100 Prepaid expenses and other current assets 1,202 411 Total current assets 38,327 41,511 Property and equipment, net 109 — Other assets 544 486 Total assets $ 38,980 $ 41,997 Liabilities and stockholders' equity Current liabilities: Accounts payable $ 1,424 $ 1,153 Accrued liabilities 2,242 2,026 Total current liabilities 3,666 3,179 Commitments and contingencies (Note 6) Stockholders’ equity: Common stock, $0.001 par value: 200,000,000 shares authorized as of December 31, 2023 and December 31, 2022; 36,335,105 and 21,000,069 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively 36 21 Additional paid-in capital 153,794 125,841 Accumulated other comprehensive income 4 — Accumulated deficit (118,520 ) (87,044 ) Total stockholders’ equity 35,314 38,818 Total liabilities and stockholders’ equity $ 38,980 $ 41,997 CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (in thousands, except share and per share amounts) Year Ended December 31, 2023 2022 Operating expenses: Research and development $ 21,862 $ 5,594 General and administrative 10,624 8,302 Total operating expenses 32,486 13,896 Loss from operations (32,486 ) (13,896 ) Interest income 1,124 162 Other expense, net (114 ) (145 ) Net loss $ (31,476 ) $ (13,879 ) Per share information: Net loss per share - basic and diluted $ (1.00 ) $ (0.80 ) Weighted average shares and pre-funded warrants outstanding, basic and diluted 31,571,039 17,383,531 Comprehensive Loss: Net loss $ (31,476 ) $ (13,879 ) Unrealized gain on available-for-sale securities 4 — Comprehensive loss $ (31,472 ) $ (13,879 ) Additional details on our 2023 financial results can be found in Forte’s Form 10-K as filed with the SEC on March 18, 2024. You can also find more information in the investor relations section of our website at . About Forte Forte Biosciences, Inc. is a clinical-stage biopharmaceutical company that is advancing its product candidate, FB-102, which is a proprietary molecule with potentially broad autoimmune and autoimmune-related applications including in such indications as graft-versus-host disease, vitiligo and alopecia areata. Forward-Looking Statements Forte cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negatives of these terms or other similar expressions. These statements are based on the Company’s current beliefs and expectations. Forward looking statements include statements regarding the Company’s beliefs, goals, intentions and expectations regarding its product candidate, FB-102 and the therapeutic potential of FB-102. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation: risks related to Forte’s ability to obtain sufficient additional capital to continue to advance Forte’s product candidate, FB-102; uncertainties associated with the clinical development and regulatory approval of Forte’s product candidate, FB-102, including potential delays in the commencement, enrollment and completion of clinical trials; the risk that results from preclinical studies may not be predictive of results from clinical trials; risks associated with the failure to realize any value from FB-102 in light of inherent risks and difficulties involved in successfully bringing product candidates to market; and additional risks, uncertainties, and other information affecting Forte’s business and operating results is contained in Forte’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 18, 2024, and in its other filings with the Securities and Exchange Commission. All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Forte undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Source: Forte Biosciences, Inc.