Zunsemetinib

- Multiple Catalysts in Immuno-Inflammatory Indications Anticipated in 2025 and 2026 - - Expected Cash Runway Extended Through the First Half of 2028 - - Phase 2 Results Received to Date from Chinese Partner CTTQ Provide Clinical Evidence of Enhanced Potency of Bosakitug (ATI-045) and Opportunity for Development Partnerships - - Investigational New Drug (IND) Application for Bispecific Antibody ATI-052 Cleared by U.S. Food and Drug Administration - WAYNE, Pa., May 08, 2025 (GLOBE NEWSWIRE) -- Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, today announced its financial results for the first quarter of 2025 and provided a corporate update. “We are entering into a potentially transformative multi-year period for Aclaris, with important milestones throughout our business that we believe will position us for future growth,” stated Dr. Neal Walker, Chief Executive Officer and Chair of the Board of Directors of Aclaris. “Ensuring successful and timely execution of our clinical programs is our priority, and given the realities of today’s financial market environment, it’s imperative that we continue to do so in a manner that efficiently utilizes our capital. As such, we’ve announced that further global development of bosakitug - our uniquely potent anti-TSLP monoclonal antibody - in respiratory indications will be dependent on partnerships. Regarding our internal programs, we expect to initiate new clinical trials with bosakitug; ATI-2138, our highly selective oral ITK/JAK3 inhibitor; and ATI-052, our potential best-in-class bispecific anti-TSLP/IL-4R antibody, for which we recently received IND clearance from the FDA. Work is also ongoing toward next-generation small molecule kinase inhibitors and biologic antagonists of immuno-inflammatory pathways to further expand our pipeline and continue our innovative work in I&I.” “Importantly, we have the cash we believe we need to execute our plan,” continued Dr. Walker. “Our expected cash runway now extends through the first half of 2028, and we will continue to practice rigorous financial stewardship with a goal of extending our runway further by exploring additional non-dilutive opportunities.” First Quarter 2025 Highlights and Recent Updates Pipeline: Bosakitug (ATI-045): Investigational Anti-TSLP monoclonal antibody Aclaris Expects to Initiate Enrollment in Placebo-Controlled Two-Arm Phase 2 Trial in Atopic Dermatitis (AD) in the Second Quarter of 2025: Based on the potency of bosakitug observed in preclinical and clinical studies to date, including the compelling results of the Phase 2a single arm trial in AD, Aclaris intends to initiate a two-arm placebo-controlled Phase 2 trial of bosakitug in approximately 90 patients with moderate-to-severe AD in the second quarter of 2025. This trial is designed to evaluate the Company’s anti-TSLP therapeutic in a placebo-controlled setting in a time- and cost-efficient manner.Results from CTTQ’s Phase 2 Trials in Respiratory Indications Support Further Development: The totality of the results received to date from Aclaris’ regional partner, Chia Tai Tianqing Pharmaceutical Group, Co., Ltd. (CTTQ) from their Phase 2 trials of bosakitug in Chinese patients with chronic rhinosinusitis with nasal polyps (CRSwNP) and severe asthma provide additional clinical evidence of the enhanced potency of bosakitug. CTTQ has announced that they are conducting Phase 3 clinical trials of bosakitug in both indications and are currently conducting a Phase 2 trial in chronic obstructive pulmonary disease (COPD). CTTQ is responsible for the disclosure and presentation of the results of their clinical trials.Aclaris Intends to Seek Partners to Develop Bosakitug in Respiratory Indications: Aclaris’ clinical focus for bosakitug will remain on dermatological immuno-inflammatory indications. Further global (excluding China) development in respiratory indications is dependent on entering into potential partnerships. ATI-2138: Investigational oral covalent ITK/JAK3 inhibitor Top Line Results from Phase 2a Trial in AD Expected in June 2025: Dosing is complete in the single arm Phase 2a open-label trial of ATI-2138 designed to investigate the safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics of ATI-2138 in patients with moderate-to-severe AD. The Company expects to report top line results in June 2025. Potential target indications under consideration include alopecia areata (AA) and vitiligo. ATI-052: Investigational bispecific anti-TSLP/IL4R monoclonal antibody Announced Clearance of ATI-052 Investigational New Drug (IND) Application and Plan to Initiate Phase 1 Trial: Aclaris announced that the U.S. Food and Drug Administration cleared its IND application for ATI-052 in April 2025. The Company expects to initiate the placebo-controlled Phase 1a/1b trial evaluating single and multiple ascending doses of ATI-052, followed by a proof-of-concept portion in an undisclosed indication, in the second quarter of 2025. (press release here) Next Generation Kinase and Cytokine Signaling Pathway Inhibitor Development: Confirmed Plan for Next IND Submission: Preclinical work is ongoing to develop next-generation ITK inhibitors, which the Company expects to provide the basis for new INDs starting in 2026.Development of Novel Bispecific Antibodies Targeting Certain Cytokine Pathways Underway: Preclinical work is ongoing to develop next-generation bispecific antibodies utilizing the bosakitug anti-TSLP binding region paired with binding fragments targeting other undisclosed cytokine signaling pathways. Corporate: Expected Cash Runway Extends Through the First Half of 2028 and Fully Funds Preclinical and Clinical Development Plans: During the first quarter of 2025, Aclaris took certain steps to focus its pipeline investments while extending its cash runway further. In doing so, the Company continues to prioritize clinical execution and achievement of development milestones, all of which Aclaris expects to be funded by the Company’s current cash runway. Aclaris ended the first quarter of 2025 with $190.5 million in cash, cash equivalents, and marketable securities providing balance sheet strength to execute on its development plan. Aclaris' cash management policy is focused on capital preservation and holding a portfolio of securities with short-term maturities. The investment portfolio is composed of fixed income securities, primarily high-credit quality corporate debt and U.S. government securities.Injunction Against Sun Pharmaceuticals Lifted, Providing Aclaris with a Potential Additional Opportunity for Non-Dilutive Financing: In 2023, Aclaris granted Sun Pharma exclusive rights under certain patents for, among other things, the use of deuruxolitinib, Sun Pharma’s JAK inhibitor, to treat AA. The agreement included an upfront payment of $15.0 million, regulatory and commercial milestones, and royalties. In 2024, Incyte Corporation was granted a preliminary injunction against Sun Pharma, blocking Sun Pharma from launching its product LEQSELVI®. In April 2025, a U.S. Appeals Court lifted the injunction. As Aclaris has successfully executed similar financings in the past, the Company may seek to monetize this financial asset to provide additional non-dilutive financing.Provided Update on Senior Leadership: Jesse W. Hall, M.D. has been appointed as Chief Medical Officer. Dr. Hall brings decades of experience in all phases of drug development, from early development through global regulatory filings and approvals, Phase IV post-marketing surveillance obligations, and commercial launch support to Aclaris. He most recently served as Chief Medical Officer for AltruBio where he was responsible for leadership of all clinical and medical functions. (press release here) First Quarter 2025 Financial Results As of March 31, 2025, Aclaris had cash, cash equivalents and marketable securities of $190.5 million compared to $203.9 million as of December 31, 2024. The Company believes that its cash, cash equivalents and marketable securities will be sufficient to fund its operations through the first half of 2028, without giving effect to any potential business development transactions or financing activities. Net loss was $15.1 million for the first quarter of 2025 compared to $16.9 million for the first quarter of 2024. Total revenue was $1.5 million for the first quarter of 2025 compared to $2.4 million for the first quarter of 2024. The decrease was primarily driven by the sale of a portion of royalty payments under the Company’s agreement with Eli Lilly and Company to OCM IP Healthcare Portfolio IP, an investment vehicle for Ontario Municipal Employees Retirement System (OMERS), in July 2024. Research and development (R&D) expenses were $11.6 million for the quarter ended March 31, 2025 compared to $9.8 million for the prior year period. The increase was primarily driven by expenses related to the Company’s bosakitug program, specifically preclinical and clinical development expenses associated with startup activities for a Phase 2 trial in AD. The increase was partially offset by a reduction in development expenses for zunsemetinib and lepzacitinib and lower compensation-related expenses. General and administrative (G&A) expenses were $6.1 million for the quarter ended March 31, 2025 compared to $6.8 million for the corresponding prior year period. The decrease was primarily due to a reduction in personnel expenses as a result of lower headcount and lower termination benefits. About Aclaris Therapeutics, Inc. Aclaris Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a pipeline of novel product candidates to address the needs of patients with immuno-inflammatory diseases who lack satisfactory treatment options. The company has a multi-stage portfolio of product candidates powered by a robust R&D engine. For additional information, please visit www.aclaristx.com and follow Aclaris on X (formerly Twitter) at @AclarisTx and on LinkedIn. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “anticipate,” “believe,” “expect,” “intend,” “may,” “plan,” “potential,” “will,” and similar expressions, and are based on Aclaris’ current beliefs and expectations. These forward-looking statements include expectations regarding its plans for its development programs for bosakitug, ATI-2138 and ATI-052, including the anticipated timing for the initiation of its Phase 2 trial of bosakitug in AD and its Phase 1 trial of ATI-052, the timing for reporting the results of its Phase 2a trial of ATI-2138 in AD, and its plans to explore additional future indications for ATI-2138 including AA and vitiligo, its plans for its preclinical development programs and timing of IND submissions, its plans to monetize the Sun Pharma financial asset, and the sufficiency of its cash, cash equivalents and marketable securities to fund its operations through the first half of 2028. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Aclaris’ reliance on third parties over which it may not always have full control, Aclaris’ ability to enter into strategic partnerships on commercially reasonable terms, the uncertainty regarding the macroeconomic environment and other risks and uncertainties that are described in the “Risk Factors” section of Aclaris’ Annual Report on Form 10-K for the year ended December 31, 2024, and other filings Aclaris makes with the U.S. Securities and Exchange Commission from time to time. These documents are available under the “SEC Filings” page of the “Investors” section of Aclaris’ website at www.aclaristx.com. Any forward-looking statements speak only as of the date of this press release and are based on information available to Aclaris as of the date of this release, and Aclaris assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Aclaris Therapeutics Contacts: Kevin BalthaserChief Financial Officer(484) 329-2178kbalthaser@aclaristx.com Will RobertsSenior Vice PresidentCorporate Communications and Investor Relations(484) 329-2125wroberts@aclaristx.com Aclaris Therapeutics, Inc.Condensed Consolidated Statements of Operations(unaudited, in thousands, except share and per share data) Three Months Ended March 31, 2025 2024 Revenues: Contract research $445 $657 Licensing 1,010 1,741 Total revenue 1,455 2,398 Costs and expenses: Cost of revenue(1) 506 809 Research and development(1) 11,584 9,845 General and administrative(1) 6,139 6,844 Licensing 1,010 1,031 Revaluation of contingent consideration 300 2,800 Total costs and expenses 19,539 21,329 Loss from operations (18,084) (18,931) Other income: Interest income 2,166 1,990 Non-cash royalty income 833 — Total other income 2,999 1,990 Net loss $(15,085) $(16,941)Net loss per share, basic and diluted $(0.12) $(0.24)Weighted average common shares outstanding, basic and diluted 122,390,303 71,074,858 (1) Amounts include stock-based compensation expense as follows: Cost of revenue $219 $252 Research and development 1,185 (29)General and administrative 2,131 1,866 Total stock-based compensation expense $3,535 $2,089 Aclaris Therapeutics, Inc.Selected Consolidated Balance Sheet Data(unaudited, in thousands, except share data) March 31, December 31, 2025 2024 Cash, cash equivalents and marketable securities $190,525 $203,896Total assets $198,094 $220,327Total current liabilities $21,514 $31,596Total liabilities $54,028 $64,773Total stockholders' equity $144,066 $155,554Common stock outstanding 108,265,529 107,850,124 Aclaris Therapeutics, Inc.Selected Consolidated Cash Flow Data(unaudited, in thousands) Three Months Ended March 31, 2025 2024 Net loss $(15,085) $(16,941)Depreciation and amortization 128 243 Stock-based compensation expense 3,535 2,089 Revaluation of contingent consideration 300 2,800 Changes in operating assets and liabilities (1,935) (9,006)Net cash used in operating activities $(13,057) $(20,815)

12月20日，Aadi Bioscience（NASDAQ：AADI）宣布从药明生物、多禧生物引入3项处于临床前阶段的ADC药物的全球独家许可权。根据协议条款，Aadi将向药明生物和多禧生物支付4400万美元首付款，最高达2.65亿美元的开发里程碑付款和5.4亿美元的商业里程碑付款，以及个位数比例的销售提成。总计合作金额高达8.49亿美元，约合人民币62亿元。 受此消息影响，Aadi Bioscience股价在盘后大涨70%+。 无独有偶，三天后，RAPT Therapeutics（NASDAQ:RAPT）宣布从济民可信引入单克隆抗体RPT904的海外权益，济民可信将获得3500万美元的首付款，以及最高可获得6.725亿美元里程碑付款及特许权使用费，RAPT当日股价暴涨110%+。 而自2024年下半年以来，已有多家美股上市的小市值药企通过引入中国资产，带来了市值暴涨： 2024年8月，Instil Bio（NASDAQ:TIL）宣布从宜明昂科引进PD-L1/VEGF双抗、CTLA-4抗体的大中华区外全球权益，Instil Bio支付5000万美元预付款和近期里程碑（其中预付款1000万美元），不超过21亿美元里程碑金额，以及个位数至低双位数比例的销售分成； 2024年11月，Aclaris Therapeutics（NASDAQ：ACRS）宣布从博奥信引入BSI-045B和BSI-502 达成全球（除大中华区外）独家授权协议。博奥信将获得超过4000万美元的首付款，以及Aclaris 19.9%的股权。此外，博奥信还可以收到超过9亿美元的开发及销售里程碑付款以及单位数比例的销售分成。 下半年，四家市值不足1亿美元的小药企，为何纷纷引入中国资产？ 1 步入发展困境的Small-cap Biotech 众所周知，生物医药行业研发失败率很高，在美股上市的许多明星Biotech公司也经历了各种失败，引入中国资产的Aadi Bioscience、RAPT Therapeutics、Aclaris Therapeutics、Instil Bio都曾是各自领域的潜力之星，但却面临各自的发展困境。 商业化前景暗淡的Aadi Bioscience。专注于精准肿瘤领域的Aadi由Abraxane（注射用紫杉醇）和白蛋白技术平台的发明者Neil Desai博士创建，旨在为具有mTOR通路驱动程序改变的癌症患者带来转化疗法。目前，Aadi的主要产品Fyarro已于2021年11月获得FDA批准上市，用于治疗不可切除的局部晚期或转移性恶性血管周围上皮样细胞肿瘤（PEComa）成年患者，今年前三季度销售额为1880万美元，而获批以来的累计销售额仅为5220万美元，商业化前景十分暗淡。 被FDA hold住研发的RAPT Therapeutics。RAPT曾是CCR4领域的明星玩家，受到诸多机构的追捧，但在2024年2月，FDA突然宣布暂停Zelnecirnon治疗哮喘的2a期试验，RAPT股价暴跌70%，最终彻底放弃了Zelnecirnon，而公司临床中管线只剩下癌症方向的CCR4抑制剂tivumecirnon。然而，同一领域的安进的CCR4抗体Mogamulizumab已经独占市场多年，显然难以超越对手，公司股价也不断创新低。 接连失败的Aclaris Therapeutics。自免药物公司Aclaris Therapeutics在2023年3月宣布ATI-450治疗化脓性汗腺炎2a期临床试验失败，接着在同年11月宣布被寄予厚望的ATI-450治疗类风湿性关节炎的2b期临床研究宣告失败，股价当日暴跌86%。 2024年初，Aclaris Therapeutics发布了JAK1/3抑制剂ATI-1777治疗轻度至重度特应性皮炎的临床2b期数据，虽然试验达到主要疗效终点，但投资者对其他“没有达到统计学意义”数据并不买账，股价再次单日暴跌25%。 被迫出租生产设施的Instil Bio。Instil Bio原先是一家TIL领域的明星biotech，但随着临床管线的折戟，股价从二十多美元一路跌至不足1美元，2023年底，被迫进行20股并1股以维持上市地位，目前长期不足1亿美元。今年7月，Instil Bio将其位于Tarzana CA的生产设施租给阿斯利康，年租金753万美元。 虽然市场对这些公司的发展已经不再看好，但毕竟作为曾经的明星企业，曾获得许多知名机构的投资，即使经历了各种失败，但手上现金依然充足，其金额甚至超过公司市值，如在引入中国资产前，Aadi有8000万美元美元现金、Instil Bio拥有1.61亿美元现金、Aclaris有1.73亿美元现金、RAPT有9790万美元现金，为license-in 资产提供支持。 2 Summit Therapeutics的成功之鉴 今年下半年以来，美股上市小药企疯狂引入中国资产，则是源于Summit Therapeutics（NASDAQ: SMMT）成功之鉴。 Summit成立于2003年，并于2015年在纳斯达克上市，致力于抗感染药物和遗传罕见病药物的开发。曾研发出一代药王依鲁替尼的Pharmacyclics（NASDAQ:PCYC)（被艾伯维以210亿美元收购）的大股东Duggan向Summit投入7500万美元，成为Summit最大股东。 Duggan投资Summit的原因是因为彼时他对抗感染药物的十分看好，而当时Summit核心产品、用于治疗艰难梭菌感染的抗生素ridinilazole刚发布了优异的临床二期数据。在此之前，Duggan已经拥有两次失败的抗生素药企投资失败经历。 然而，Summit在抗生素领域的研究进展并不顺利，2021年12月，公司发布了ridinilazole临床三期数据。结果显示，相较于已有的标准疗法，ridinilazole的优势并不显著，且未达主要终点。 由于核心产品的失败，Summit股价持续下跌，2022年12月初，公司股价跌至每股0.7美元，是一家典型的失败Biotech。 2022年12月，Summit获得康方生物PD-1/VEGF双抗依沃西的海外权益，不到两年时间，公司股价已经从0.785美元/股上涨到18美元/股，上涨了23倍。Summit也从一家市值仅6亿美元的小公司，变成市值130多亿美元的中型公司。 令人惊叹的成长曲线自然吸引效仿者，AADI、RAPT、Aclaris在宣布引入中国资产后，股价在一周内分别上涨了50%+、100%+与125%+，更为夸张的是Instil bio，在一个月内股价涨超6倍。 3 美股小药企或将开启中国资产淘金潮 与先行者Summit Therapeutics使用自有资金引入中国资产不同，下半年以来，美股上市的小型生物医药公司开始与投资机构联手引入中国资产，同步宣布进行PIPE融资（ Private Investment in Public Equity ）： Aclaris Therapeutics宣布完成8000万美元的PIPE融资，融资由Vivo Capital领投，Forge Life Science 、Rock Springs、RA Capital、Adage Capital、Decheng Capital、Logos Capital、Samsara BioCapital跟投； Aadi Bioscience宣布完成1亿美元的PIPE融资，融资由Ally Bridge领投，OrbiMed、Invus、Kalehua Capital、普方生物联合创始人Tae Han、Avoro Capital、KVP Capital、Acuta Capital跟投； RAPT Therapeutics宣布完成1.5亿美元的PIPE融资，融资由The Column Group和TCGX领投，BVF、Deep Track、OrbiMed、Perceptive 、Medicxi、Redmile、Foresite Capital和RTW跟投。 鉴于宣布引入资产后的良好表现，越来越多的投资机构对license-in的PIPE融资感兴趣，一些资金不怎么充裕的美股小市值生物医药上市公司也有机会引入中国资产，Small-cap Biotech来华淘金或许会成为新浪潮。 同写意媒体矩阵，欢迎关注↓↓↓