Masofaniten

Combination of masofaniten plus enzalutamide continues to be well tolerated with deep and durable reductions in PSA in Phase 1 dose escalation in patients with mCRPC, including 81% of patients achieving PSA90, 69% of patients achieving PSA90 in less than 90 days, and 63% of patients achieving PSA <0.2ng/mL Phase 2 masofaniten plus enzalutamide combination dose expansion study ongoing Cash runway sufficient to fund operations beyond 2025 SOUTH SAN FRANCISCO, California and VANCOUVER, Canada, Feb. 13, 2024 /PRNewswire/ - ESSA Pharma Inc. ("ESSA", or the "Company") (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, today provided a corporate update and reported financial results for the fiscal first quarter ended December 31, 2023. "As we enter 2024, we are focused on advancing the investigation of masofaniten in combination with second-generation antiandrogen agents, with the goal of improving clinical outcomes for patients battling prostate cancer," said David Parkinson, MD, President and CEO of ESSA. "We are extremely pleased with the data reported to date from the Phase 1 dose escalation study evaluating masofaniten combined with enzalutamide in patients with mCRPC naïve to second generation anti-androgens where we continue to observe compelling, deep and durable reductions in prostate-specific antigen ("PSA"), along with an encouraging 16.6 month median time to PSA progression. We look forward to reporting updated Phase 1 dose escalation data during 2024. In addition, we continue to advance enrollment in both Phase 1 arms evaluating masofaniten in combination with apalutamide or abiraterone acetate, respectively, and look forward to reporting preliminary data during the second half of 2024." "On the financial front, we are bolstered by our strong cash position that we expect will provide sufficient runway to fund our planned operations through several data readouts and beyond 2025. We are excited to execute on our key 2024 objectives which we believe will meaningfully advance the investigation of masofaniten and look forward to providing updates on our progress throughout the year," concluded Dr. Parkinson. First Quarter Fiscal 2024 and Recent Highlights Masofaniten Combination Studies Reported Phase 1 dose escalation data at multiple medical meetings from the four cohorts of its ongoing Phase 1/2 study evaluating masofaniten in combination with enzalutamide in patients with metastatic castration-resistant prostate cancer ("mCRPC") naïve to second-generation antiandrogens but may have been treated with chemotherapy in the metastatic castration-sensitive setting. These data were presented at the 2024 ASCO Genitourinary Cancers Symposium, the 2023 Prostate Cancer Foundation Scientific Retreat and at the European Society for Medical Oncology 2023 Congress. As of the most recent data presentation, the results demonstrated that the combination continues to be well tolerated at the dose levels tested through up to 25 cycles of dosing in some patients. Deep and durable reductions in PSA were observed across evaluable patients for efficacy in all dosing cohorts (n=16). Across all dosing cohorts, 88% of patients achieved PSA50, 81% of patients achieved PSA90, 69% of patients achieved PSA90 in less than 90 days, and 63% of patients achieved PSA <0.2ng/mL. While the data for time to PSA progression are still maturing with a current median follow up of 11.1 months, the median time to PSA progression is 16.6 months. ESSA expects to report updated data from the Phase 1 dose escalation study during the second half of 2024. Enrollment is underway in the Phase 2 portion of the Phase 1/2 study evaluating the combination of masofaniten and enzalutamide compared to enzalutamide monotherapy in patients with mCRPC naïve to second-generation antiandrogens but who may have been treated with chemotherapy in the metastatic castration-sensitive setting. The Phase 2 portion of the study is an open-label randomized study comparing 160 mg once-daily of single agent enzalutamide to the combination of masofaniten with enzalutamide, and is expected to enroll approximately 120 patients. The recommended Phase 2 combination dose was identified as masofaniten 600 mg twice-daily combined with enzalutamide 160 mg once daily. The study is currently enrolling at approximately 25 sites in the USA, Canada and Australia. Expansion to European sites is in progress. ESSA plans to provide guidance for timing of the public disclosure of initial data once the Phase 2 portion has been underway for several months. Initiated two additional masofaniten combination arms as part of the ongoing Phase 1 masofaniten study. One arm will evaluate masofaniten in combination with abiraterone acetate and prednisone in patients with either metastatic castration-sensitive prostate cancer or mCRPC while the second arm will evaluate masofaniten in combination with apalutamide in patients with non-metastatic castration-resistant prostate cancer after 12 weeks of masofaniten single agent. ESSA expects to report preliminary data from these Phase 1 arms during the second half of 2024. An investigator-sponsored neoadjuvant study was also initiated evaluating neoadjuvant use of the combination of masofaniten and darolutamide compared to darolutamide monotherapy in high-risk patients undergoing prostatectomy. Masofaniten Monotherapy Study On track to complete the Phase 1b masofaniten monotherapy study evaluating masofaniten in patients with late-line mCRPC, which is currently ongoing. The initial results from the study were reported at the 2023 American Society of Clinical Oncology Genitourinary Cancers Symposium, which demonstrated that masofaniten monotherapy was well-tolerated, achieved clinically significant exposures, and showed preliminary signals of anti-tumor activity in a subset of patients. ESSA plans to present the complete Phase 1a and 1b monotherapy results in 2024 at a medical conference. Summary Financial results (Amounts expressed in U.S. dollars) Net Loss. ESSA recorded a net loss of $6.0 million for the first quarter ended December 31, 2023 compared to $6.7 million for the first quarter ended December 31, 2022. The decrease in the first quarter was primarily attributed to an increase in investment income over the prior period of $0.5 million. Research and Development ("R&D") expenditures. R&D expenditures for the first quarter ended December 31, 2023 were $5.4 million compared to $5.3 million for the first quarter ended December 31, 2022, and include non-cash costs related to share-based payments $526,241 for the first quarter ended 2023 compared to $791,192 for the first quarter ended 2022. Reduced expenditure on manufacturing for the period was offset by additional investment in the clinical trial work on masofaniten. General and Administration ("G&A") expenditures. G&A expenditures for the first quarter ended December 31, 2023 were $2.2 million compared to $2.5 million for the first quarter ended December 31, 2022 and include non-cash costs related to share-based payments of $277,177 for the first quarter ended 2023 compared to $772,419 for the first quarter ended 2022. The overall expenditure is largely comparable to the Company's additional spend on professional fees to update Company's documents for its shelf prospectus and ATM program. Liquidity and Outstanding Share Capital As of December 31, 2023, the Company had available cash reserves and short-term investments of $142.1 million. The Company's cash position is expected to be sufficient to fund current and planned operations beyond 2025. On November 6, 2023, the Company announced that it had entered into an Open Market Sale AgreementSM (the "ATM Sales Agreement") with Jefferies LLC, effective as of November 3, 2023. Under the ATM Sales Agreement, ESSA may, within the period that the ATM Sales Agreement is in effect, sell its common shares from time to time for up to $50.0 million in aggregate sales proceeds. No offers or sales of common shares will be made in Canada, to anyone known by Jefferies LLC to be a resident of Canada or on or through the facilities of any stock exchange or trading markets in Canada. As of December 31, 2023, the Company had 44,163,647 common shares issued and outstanding. In addition, as of December 31, 2023, there were 2,920,000 common shares issuable upon the exercise of prefunded warrants at an exercise price of $0.0001. About ESSA Pharma Inc. ESSA is a clinical-stage pharmaceutical company focused on developing novel and proprietary therapies for the treatment of patients with prostate cancer. For more information, please visit , and follow us on Twitter and LinkedIn. Forward-Looking Statement Disclaimer This release contains certain information which, as presented, constitutes "forward-looking information" within the meaning of the Private Securities Litigation Reform Act of 1995 and/or applicable Canadian securities laws. Forward-looking information involves statements that relate to future events and often addresses expected future business and financial performance, containing words such as "anticipate", "believe", "plan", "estimate", "expect", and "intend", statements that an action or event "may", "might", "could", "should", or "will" be taken or occur, or other similar expressions and includes, but is not limited to, statements regarding the Company's plans to report updated data from its studies, the Company's advancement and evaluation of masofaniten, the timing of the Company's studies, enrollment in the Company's studies, the presentation of Phase 1a and 1b monotherapy results, sales of common shares pursuant to the ATM Sales Agreement and the Company's expected cash runway. Forward-looking statements and information are subject to various known and unknown risks and uncertainties, many of which are beyond the ability of ESSA to control or predict, and which may cause ESSA's actual results, performance or achievements to be materially different from those expressed or implied thereby. Such statements reflect ESSA's current views with respect to future events, are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by ESSA as of the date of such statements, are inherently subject to significant medical, scientific, business, economic, competitive, political and social uncertainties and contingencies. In making forward looking statements, ESSA may make various material assumptions, including but not limited to (i) the accuracy of ESSA's financial projections; (ii) obtaining positive results of clinical trials; (iii) obtaining necessary regulatory approvals; and (iv) general business, market and economic conditions. Forward-looking information is developed based on assumptions about such risks, uncertainties and other factors set out herein and in ESSA's Annual Report on Form 10-K dated December 12, 2023, under the heading "Risk Factors", a copy of which is available on ESSA's profile on EDGAR at and on SEDAR+ at , and as otherwise disclosed from time to time on ESSA's EDGAR and SEDAR+ profiles. Forward-looking statements are made based on management's beliefs, estimates and opinions on the date that statements are made and ESSA undertakes no obligation to update forward-looking statements if these beliefs, estimates and opinions or other circumstances should change, except as may be required by applicable United States and Canadian securities laws. Readers are cautioned against attributing undue certainty to forward-looking statements. Contacts ESSA Pharma, Inc. David Wood, Chief Financial Officer 778.331.0962 [email protected] Investors and Media: Argot Partners 212.600.1902 [email protected] ESSA PHARMA INC. CONSOLIDATED BALANCE SHEETS Amounts in thousands of United States dollars ESSA PHARMA INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS Amounts in thousands of United States dollars, except share and per share data SOURCE ESSA Pharma Inc

▎药明康德内容团队编辑本期看点1. 礼来（Eli Lilly and Company）公布其基因疗法AK-OTOF的积极早期临床结果，一次给药后，先天失聪患儿在30天内听力获得恢复。2. 第二代抗雄激素药物EPI-7386联用恩杂鲁胺（enzalutamide）治疗转移性去势抵抗性前列腺癌（mCRPC）患者的1/2期临床试验结果积极，88%患者的前列腺特异性抗原（PSA）减少≥50%（PSA50）。3. 广谱合成聚合物抗感染药物RECCE 327用于治疗糖尿病足感染（DFI）的早期临床试验达到主要终点。药明康德内容团队整理AK-OTOF：公布1/2期临床试验的新数据礼来的全资子公司Akouos宣布，其1/2期临床试验AK-OTOF-101取得了积极的初步临床结果。AK-OTOF是一种治疗因耳铁蛋白基因（OTOF）突变而导致感音神经性听力丧失患者的基因疗法。AK-OTOF利用AAVAnc80（一种对内毛细胞具有高转导效率的衣壳）和一个强大、普遍存在的启动子，实现了只在目标内毛细胞中才能观察到的耳铁蛋白的表达，其表达水平有可能恢复高敏锐度的生理性听力。Akouos同时开发了专门用于耳蜗内给药的给药装置，可通过微创手术方法在整个耳蜗内给予AK-OTOF。此疗法具有成为治疗内耳疾病潜在“first-in-class”疗法的可能。在该研究中，第一位接受AK-OTOF治疗的受试者是一名11岁的儿童，出生时就患有重度听力损失。在接受AK-OTOF治疗后的30天内，该患者的听力就得到了恢复。在第30天的检查中，该患者所有测试频率的听力都得到了恢复，达到65至20分贝听力级（hearing level，即人耳在某一频率的听阈比正常听阈高出的分贝）的阈值，某些频率的听力在正常范围内。手术给药过程和AK-OTOF疗法的耐受性都很好，没有严重不良事件的报告。第二例受试者的的初步数据将在2024年耳鼻喉科研究协会（ARO）中冬会议上公布。Masofaniten（EPI-7386）：公布1/2期临床试验的新数据ESSA Pharma公布了其第二代抗雄激素药物EPI-7386与恩杂鲁胺联合治疗转移性去势抵抗性前列腺癌患者的1/2期临床试验的最新数据。EPI-7386是一种高选择性的雄激素受体N-末端结构域的口服小分子抑制剂。此次公布的研究结果显示，该组合疗法的耐受性良好，无论患者先前的化疗状态如何，患者的PSA都有快速、深度且持久的降低。在所有队列中，88%（14/16）的患者达到了PSA50，81%（13/16）患者的PSA减少≥90%（PSA90），69%（11/16）的患者在90天内达到PSA90，63%（10/16）患者达到PSA<0.2 mg/mL的水平。中位随访时间为11.1个月时，PSA病情进展的中位时间为16.6个月。RECCE 327（R327）：公布1/2期临床试验数据Recce Pharmaceuticals公司公布了其抗感染候选药物RECCE 327（R327）用于治疗糖尿病足感染患者的积极早期临床结果。R327是Recce Pharmaceuticals公司开发的一种广谱合成聚合物抗感染药物，旨在解决严重或危及生命的细菌感染，包括脓毒症。研究表明，该化合物能够迅速且不可逆地关闭细菌细胞能量ATP的产生，使细菌的蛋白质合成减少和细胞膜去极化，在较高浓度下甚至可以导致细胞裂解。R327已被FDA授予快速通道资格。该研究达到了解决/治愈DFI中细菌感染的主要终点。所有患者对R327局部治疗的耐受性都很好，能解决轻度皮肤和软组织DFI，包括耐多药革兰氏阳性和革兰氏阴性病原体的感染。▲接受R327治疗的DFI患者的情况概览（图片来源：参考资料[4]）STX-001：IND申请获得FDA许可Strand Therapeutics宣布，美国FDA已批准其多机制合成的自我复制mRNA疗法STX-001的IND申请，将启动首个人体试验。STX-001是基于Strand公司实体瘤平台研发的首款药物，它能够直接在肿瘤细胞内表达所需的任何治疗性蛋白质。新闻稿称，STX-001能够提供治疗剂量的IL-12，其疗效优于传统的mRNA疗法。STX-001与PD-1/PD-L1检查点抑制剂联用时还能诱导持久的抗肿瘤反应并增强疗效。此外，STX-001的自我复制mRNA技术还可诱导免疫原性癌细胞死亡，并促进T细胞和NK细胞在肿瘤微环境中的招募和活化。该公司计划在2024年年初将STX-001推进到临床试验中。VCA-894A：IND申请获得FDA许可Vanda Pharmaceuticals公司宣布，FDA已经批准了其新型反义寡核苷酸（ASO）疗法VCA-894A的IND申请，该药将用于治疗由IGHMBP2基因中的隐性剪接位点变异引起的2S型腓骨肌萎缩症（CMT2S）患者。IGHMBP2突变在CMT2S的表现中起着关键作用，可能是由于α-运动神经元的缺失，进而导致周围神经系统恶化。ASO具有调节基因表达的能力，可用于罕见病的个体化治疗。VCA-894A能够通过特异性靶向IGHMBP2的一个隐性剪接位点变体发挥作用。CX-2051、CX-801：IND申请获得FDA许可CytomX Therapeutics公司宣布，其靶向EpCAM的条件激活型抗体偶联药物（ADC）候选疗法CX-2051和条件激活型免疫治疗细胞因子CX-801的IND申请已获得FDA批准。CX-2051是一种条件激活型ADC，其使用的细胞毒性有效载荷是喜树碱的衍生物。喜树碱是一种拓扑异构酶-1抑制剂，这类药物已在ADC领域针对多个靶点显示出强大的临床抗癌活性。CX-2051已在包括结直肠癌在内的多个临床前模型中显示出强大的临床前活性和耐受性。干扰素α2b（IFNα2b）是一种免疫治疗细胞因子，在转移性黑色素瘤、肾癌和膀胱癌等多种癌症类型中已显示出临床活性，并获得了监管部门的批准。与其他细胞因子相比，IFNα2b在激活抗肿瘤免疫反应方面可能更胜一筹。CX-801是一种双重掩蔽、条件激活型IFNα2b，有可能成为多种肿瘤类型联合治疗的基石，包括传统的免疫肿瘤学敏感和不敏感（冷）肿瘤。CX-2051已获准在已知表达EpCAM的实体瘤中启动1期剂量递增研究，CX-801已获准在黑色素瘤、肾癌和头颈部鳞状细胞癌等实体瘤中启动1期剂量递增研究。该公司计划于2024年上半年启动这两项研究。Naronapride：IND申请获得FDA许可Renexxion Ireland公司宣布，FDA批准了naronapride的IND申请，用于治疗对质子泵抑制剂（PPI）无反应的有症状的胃食管反流病（GERD）患者。Naronapride是一种潜在“best-in-class”的口服、局部作用的泛促胃动力药，它通过调节肠壁腔面的两个有效靶点——激动5-HT4受体和拮抗D2受体发挥作用，可用于上消化道和下消化道具有未竟需求的适应症。Naronapride的设计旨在使其在肠道腔内具有局部活性和最小的可吸收性，从而提高疗效和安全性。目前，该公司已经完成了4项针对naronapride的积极的2期研究，naronapride用于治疗慢性特发性便秘和胃食管反流病的3期研究也已准备就绪。大家都在看药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看）[1] Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting. 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Retrieved January 25, 2024, from https://www.prnewswire.com/news-releases/onward-therapeutics-announces-enrollment-of-phase-1-clinical-trial-of-a-novel-bispecific-antibody-ot-a201-targeting-two-immune-checkpoints-302042048.html[9] Renexxion Ireland Ltd. Announces FDA Clearance of Investigational New Drug (IND) Application for Naronapride to Treat PPI-Non-Responsive Symptomatic GERD. Retrieved January 25, 2024, from https://www.globenewswire.com/news-release/2024/01/24/2815101/0/en/Renexxion-Ireland-Ltd-Announces-FDA-Clearance-of-Investigational-New-Drug-IND-Application-for-Naronapride-to-Treat-PPI-Non-Responsive-Symptomatic-GERD.html[10] CytomX Therapeutics Announces FDA Clearance of IND Applications for CX-2051, a Probody® Antibody Drug Conjugate (ADC) Targeting EpCAM and CX-801, a Conditionally Activated Interferon Alpha-2b. Retrieved January 25, 2024, from https://www.globenewswire.com/news-release/2024/01/24/2815209/37704/en/CytomX-Therapeutics-Announces-FDA-Clearance-of-IND-Applications-for-CX-2051-a-Probody-Antibody-Drug-Conjugate-ADC-Targeting-EpCAM-and-CX-801-a-Conditionally-Activated-Interferon-Al.html[11] Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease. Retrieved January 25, 2024, from https://www.globenewswire.com/news-release/2024/01/25/2817385/0/en/Ultragenyx-Announces-Completion-of-Dosing-Across-Stage-1-Cohorts-in-Pivotal-Phase-1-2-3-Cyprus2-Study-Evaluating-UX701-Gene-Therapy-for-the-Treatment-of-Wilson-Disease.html[12] ESSA Pharma Presents Updated Phase 1 Masofaniten (EPI-7386) Clinical Data at the 2024 ASCO Genitourinary Cancers Symposium. Retrieved January 25, 2024, from https://www.prnewswire.com/news-releases/essa-pharma-presents-updated-phase-1-masofaniten-epi-7386-clinical-data-at-the-2024-asco-genitourinary-cancers-symposium-302044194.html免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新