On June 6, 2024 Exicure, Inc. (Nasdaq: XCUR) has historically been an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. In September 2022, the Company reported a significant reduction in force, suspension of preclinical activities and halting of all research and development, and that the Company was exploring strategic alternatives to maximize stockholder value (Press release, Exicure, JUN 6, 2024, View Source [SID1234644182]).

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2023 Financial Results

Cash Position: Cash and cash equivalents were $0.8 million as of December 31, 2023, as compared to $8.6 million as of December 31, 2022. Subsequent to December 31, 2023, our cash and cash equivalents have decreased to approximately $0.2 million as of May 31, 2024. Subsequent to May 31, 2024, the Company received a $0.7 million loan from DGP Co., Ltd., a significant stockholder. The loan has a maturity of ten months from issuance and interest at a rate of 6.0% per annum is payable at maturity. The Company believes that its cash and cash equivalents are insufficient to continue to fund operations and additional funding is needed in the very near term.

Research and Development (R&D) Expense: Research and development expenses were $1.4 million for the year ended December 31, 2023, as compared to $19.8 million for the year ended December 31, 2022. The decrease in R&D expense for the year ended December 31, 2023 of $18.3 million reflects the stoppage of clinical, preclinical, and discovery program activities and a reduction in employee headcount with lower employee-related expenses and fewer discovery, preclinical, and clinical program activities resulting from the restructuring activities that the Company announced in December 2021 and September 2022.

General and Administrative (G&A) Expense: General and administrative expenses were $12.7 million for the year ended December 31, 2023, as compared to $10.9 million for the year ended December 31, 2022. The increase in G&A expense of $1.8 million for the year ended December 31, 2023 was mostly due to certain expenses that previously had been recorded as research and development expenses, such as office facilities, legal, and payroll related costs, that no longer met the criteria to be classified as research and development expenses due to the shift in our historical business operations discontinuing all research and development activities.

Net Loss: The Company had a net loss of $16.9 million for the year ended December 31, 2023, as compared to a net loss of $2.6 million for the year ended December 31, 2022. Although operating expenses were reduced by more than 50%, there were no revenues to offset these expenses resulting in an increase in net loss of $14.3 million.

Going Concern: Management believes that the Company’s existing cash and cash equivalents is not sufficient to continue to fund operations. The Company has already engaged in significant cost reductions, so our ability to further cut costs and extend the Company’s operating runway is limited. As a result, substantial additional financing is needed in very near term to pay expenses, fund the ongoing exploration of strategic alternatives and pursue any alternatives that may be identified. The Company needs to raise capital to fund its operations. There can be no assurance that such additional financing will be available and, if available, can be obtained on acceptable terms.

On June 6, 2024 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported that Dr. Neal Walker, Interim President & CEO of Aclaris, will participate in a fireside chat during the Goldman Sachs 45th Annual Global Healthcare Conference on Tuesday, June 11, 2024 at 11:20 AM ET in Miami, Florida (Press release, Aclaris Therapeutics, JUN 6, 2024, View Source [SID1234644181]).

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A webcast of the fireside chat may be accessed through the "Events" page of the "Investors" section of Aclaris’ website, www.aclaristx.com. The webcast will be archived for at least 30 days on the Aclaris website.

On June 6, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported additional details about the upcoming ENVISION virtual data event on Thursday, June 13, 2024, at 11:00 a.m. Eastern Time (Press release, UroGen Pharma, JUN 6, 2024, View Source [SID1234644178]). The event will focus on UGN-102 (mitomycin) for intravesical solution for patients with low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

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UroGen will provide 12-month durability of response results (secondary endpoint) from patients who exhibited a complete response (the primary endpoint) at three months following six weekly instillations of UGN-102. The program will also feature Key Opinion Leaders and a panel discussion on the treatment of LG-IR-NMIBC.

Please register for the webinar under the Events & Presentations section of the Company’s Investor Relations site (View Source).

Following the live audio webcast, a replay will be available on the Company’s website (View Source).

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting. If approved, UGN-102 would be the first non-surgical option approved for primary treatment of LG-IR-NMIBC, a subset of bladder cancer characterized by high recurrence rates and multiple surgeries.

On June 6, 2024 Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it has advanced into the Phase 1b portion of its Phase 1/2 proof-of-concept trial of revumenib, the Company’s highly selective, oral menin inhibitor, as a monotherapy in patients with relapsed or refractory (R/R) metastatic microsatellite stable (MSS) colorectal cancer (CRC) (Press release, Syndax, JUN 6, 2024, View Source [SID1234644177]). The Company’s decision was supported by the trial’s Independent Data Monitoring Committee (IDMC) following its recent pre-planned review of initial data from the Phase 1a portion of the trial.

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"In our first clinical trial exploring expansion of revumenib beyond hematological malignancies, initial results from the Phase 1a portion of the trial are encouraging," said Neil Gallagher, M.D., Ph.D., President, Head of Research and Development at Syndax. "We are particularly pleased to observe a compelling safety profile consistent with our existing revumenib dataset, as well as efficacy signals that include a 33% stable disease rate at 16 weeks as a monotherapy, which compares favorably with current standard-of-care and supports advancement into the Phase 1b portion. As we continue to focus on preparations for the potential launches of revumenib and axatilimab later this year, we look forward to continuing to explore the role revumenib could play in the treatment of R/R metastatic MSS CRC."

The Phase 1/2 trial (NCT05731947) is designed to assess the safety, tolerability, and anti-tumor activity of revumenib in patients with relapsed or refractory metastatic MSS CRC. The Phase 1a dose escalation portion of the trial enrolled a total of 19 patients who had a median of four prior therapies across three dose cohorts, including 163 mg, 226 mg, and 276 mg three times a day (TID). Revumenib was well-tolerated at all dose levels tested and the safety profile was consistent with the Company’s previously reported data. No Grade 3 or greater treatment-related adverse events (TRAEs) were observed and the most common TRAEs were decreased appetite, dysgeusia, nausea, and fatigue. In addition, the initial efficacy results provide early clinical support that revumenib may be able to impact disease progression in R/R patients with metastatic MSS CRC. At doses believed to achieve full target saturation, dose levels 2 and 3, 44% (4/9) of patients had stable disease at 8 weeks, and 33% (3/9) of patients had stable disease at 16 weeks. One patient with prolonged stable disease remained on study for 32 weeks. Based on the initial data, 276 mg TID was selected as the go-forward dose in the Phase 1b portion.

About Metastatic MSS CRC

Metastatic microsatellite stable (MSS) colorectal cancer (CRC) represents the second leading cause of cancer death in the U.S. with an estimated incidence in the relapsed or refractory (R/R) setting of over 55,000 patients per year. Activation of the Wnt/β-catenin signaling pathway is believed to be a key initiating step and growth driver for the majority of CRC tumors. The menin-MLL1 protein complex has been shown to regulate β-catenin activity and disrupting this complex through menin inhibition blocks growth of Wnt/β-catenin driven CRC tumors in preclinical models.

About Revumenib

Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged (KMT2Ar), also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). Positive topline results from the pivotal AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Pivotal data from the AUGMENT-101 trial in R/R NPM1 AML patients are expected in the fourth quarter of 2024. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted Breakthrough Therapy Designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement. The NDA filing for revumenib in R/R KMT2Ar acute leukemia is currently under Priority Review by the FDA under RTOR with a PDUFA action date of September 26, 2024.

On June 6, 2024 Replimune Group, Inc. (Nasdaq: REPL), a clinical stage biotechnology company pioneering the development of a novel class of oncolytic immunotherapies, reported the topline results from the primary analysis of the IGNYTE clinical trial of RP1 plus nivolumab in anti-PD1 failed melanoma (Press release, Replimune, JUN 6, 2024, View Source [SID1234644176]). The results by independent central review show one-third of patients receiving RP1 plus nivolumab responded to treatment, improving upon the investigator-assessed data presented at ASCO (Free ASCO Whitepaper) 2024, with all responses lasting greater than 6 months from baseline.

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"The overall strength of the IGNYTE data and safety profile further highlights the potential of RP1 in a difficult treatment setting with limited options for patients," said Sushil Patel, Ph.D., CEO of Replimune. "Based on these compelling results and recent FDA interactions, we are increasingly confident in our path forward. We have shared the results with the agency and plan to request a pre-BLA meeting, in advance of our intended BLA submission. With these data in hand, we are preparing for a commercial launch next year."

The anti-PD1 failed melanoma cohort from the IGNYTE clinical trial includes 140 patients who received RP1 plus nivolumab after confirmed progression while being treated with at least 8 weeks of prior anti-PD1 therapy (+/- anti-CTLA-4). The primary analysis by independent central review was triggered once all patients had been followed for at least 12 months.

The topline results show the overall response rate was 33.6% by modified RECIST 1.1 criteria, the primary endpoint as defined in the protocol, and 32.9% by RECIST 1.1 criteria, an additional analysis requested by the FDA. Responses from baseline were highly durable, with all responses lasting more than 6 months and median duration of response exceeding 35 months. The Company plans to submit the full primary analysis data from the anti-PD1 failed melanoma cohort including key secondary endpoint data and subgroups for presentation at an upcoming medical congress.

RP1 combined with nivolumab continues to be well-tolerated, with mainly Grade 1-2 constitutional-type side effects, observed. Treatment-related adverse events associated with RP1 in combination with nivolumab were predominantly Grade 1-2 constitutional type events (> 5% of patients), including fatigue, chills, pyrexia, nausea, influenza-like illness, injection-site pain, diarrhea, vomiting, headache, pruritis, asthenia, arthralgia, myalgia, decreased appetite, and rash, with a low incidence of Grade 3-5 events. Grade 4 events were one each of lipase increased, alanine aminotransferase increased, blood bilirubin increased, cytokine release syndrome, myocarditis, hepatic cytosis and splenic rupture. There were no Grade 5 events.

Conference Call Details
Replimune will host a conference call and webcast today at 8:00 a.m. ET. Listeners can register for the conference call via this link. Analysts wishing to participate in the question-and-answer session should use this link. The webcast and slides of the presentation can be accessed in the Investors section of the Company’s website at www.replimune.com. A replay of the webcast will be available on the Company’s investor website approximately two hours after the call’s conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.

About RP1
RP1 is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.