On January 8, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported that Raul Rodriguez, the company’s president and CEO, will present a company overview at the 43rd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2025 at 8:15 a.m. PT (11:15 a.m. ET) in San Francisco, CA (Press release, Rigel, JAN 8, 2025, View Source [SID1234649510]).

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To access the live webcast or archived recording, visit the Investor Relations section of the company’s website at www.rigel.com. Please connect to Rigel’s website prior to the start of the live webcast to allow for any software downloads.

On January 8, 2025 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, reported that the first patients have been enrolled in studies evaluating RP2 in two different settings: checkpoint naïve metastatic uveal melanoma; and second-line recurrent or metastatic hepatocellular carcinoma (HCC) (Press release, Replimune, JAN 8, 2025, View Source [SID1234649509]).

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"On the heels of our BLA submission for RP1 and designation as breakthrough therapy, we are pleased that the first patients have been enrolled in both the RP2 HCC clinical trial and the registration intended study of RP2 in metastatic uveal melanoma," said Sushil Patel, Ph.D., CEO of Replimune. "We are excited to explore the broader potential of the RPx platform and these RP2 clinical trials will play an important part of our future development plans."

RP2-202 Clinical Trial in Metastatic Uveal Melanoma
Uveal melanoma is a type of cancer that occurs in the tissues of the eye. Up to 50 percent of patients with uveal melanoma may develop metastatic disease. The most common site of metastasis for uveal melanoma is the liver and is estimated to occur in 90-95% of cases.1

"We are honored and excited to be able to offer this clinical trial to our patients with uveal melanoma, a group of patients for whom treatment options are very limited," said Dr. Justin Moser, an associate clinical investigator in the Cancer Research Division of HonorHealth Research Institute, where he specializes in uveal melanoma. "We hope that, by providing our patients with early access to treatments through clinical trials, that we will be able to help give them longer, higher-quality lives."

During ASCO (Free ASCO Whitepaper) 2024, results from an open-label, multicenter, Phase 2 study of RP2 alone or combined with nivolumab in a cohort of patients with uveal melanoma (n=17) were presented. RP2 administered as monotherapy or in combination with nivolumab demonstrated an overall response rate of 29.4%, with a disease control rate of 58.8%.

The RP2-202 trial (NCT06581406) is a randomized, phase 2/3 study that will enroll approximately 280 patients and evaluate RP2 in combination with nivolumab versus ipilimumab in combination with nivolumab in immune checkpoint inhibitor-naïve adult patients with metastatic uveal melanoma. The primary endpoints of the study are overall survival and progression free survival. Key secondary endpoints are overall response rate and disease control rate. For additional information about the RP2-202 clinical trial and to learn more about eligibility, please visit our clinical trials page here.

RP2-003 Clinical Trial in Hepatocellular Carcinoma
HCC is the third leading cause of cancer-related deaths in the world. Prognosis is generally poor with the majority of HCC cases diagnosed in the advanced stage. HCC comprises approximately 75 to 85 percent of primary liver cancer cases.

The RP2-003 trial (NCT05733598) is an open label trial that will enroll 30 patients and evaluate RP2 combined with the second-line therapy of atezolizumab and bevacizumab in patients with locally advanced unresectable, recurrent and/or metastatic HCC. The primary endpoint of the study is overall response rate (ORR) per modified RECIST 1.1 criteria. Key secondary endpoints are ORR per RECIST modified for HCC and duration of response. The study is being conducted under a collaboration and supply agreement with Roche. For additional information about the RP2-003 trial and to learn more about eligibility, please visit our clinical trials page here.

About RP2
RP2 is a derivative of RP1, Replimune’s lead product candidate that is based on a proprietary new strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response. RP2 additionally expresses an anti-CTLA-4 antibody-like molecule, as well as GALV-GP-R- and GM-CSF. RP2 is intended to provide targeted and potent delivery of these proteins to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic-immune-based efficacy on tumors and limiting off-target toxicity.

On January 8, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported that management will present at the 43rd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2025, at 7:30 am PT in San Francisco (Press release, Protara Therapeutics, JAN 8, 2025, View Source [SID1234649508]).

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A live webcast of the presentation can be accessed by visiting the Events and Presentations section of the Company’s website: View Source The webcast will be archived for a limited time following the presentation.

On January 8, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported a business outlook and outlined expected upcoming milestones (Press release, Moleculin, JAN 8, 2025, View Source [SID1234649507]).

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"We have positioned Moleculin to achieve value-driving milestones through the next several years, starting with the imminent beginning of enrollment for MIRACLE and the first interim read-out from that study later this year. Having a readout in the first year of a Phase 3 approval trial is, we believe, exceptional. Prior data support our belief that Annamycin is a potential game-changing asset for the treatment of AML, and we believe it can lead to significant value creation for Moleculin as we aggressively move forward with our clinical development plan. Not only has Annamycin appeared to be safer and more effective than currently prescribed anthracyclines, but we believe the recently announced preclinical data demonstrating significant activity of Annamycin in a Venetoclax-resistant AML model underscores the potential that Annamycin has ‘pipeline in a product’ potential and represents a much-needed treatment option for other patients who otherwise have very poor outcomes. Annamycin’s performance in Phase 2 has outperformed the response rates seen in billion-dollar assets in the AML space and AML remains among the highest unmet needs in healthcare. Simply stated, the bar for approval is low, and the potential reward for shareholders is substantial and we have never been more excited about the prospects for Annamycin," commented Walter Klemp, Chairman and CEO of Moleculin.

Clinical Development Update

Relapsed or Refractory Acute Myeloid Leukemia (AML)

The Company is currently advancing the development of Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") to a Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be global, including sites in the US, Europe and the Middle East.

The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting. The amended protocol allows for the unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). This early unblinding will yield 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects with just HiDAC. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

For Part B of the trial, approximately 244 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative. This increase from 240 to 244 subjects represents the statistical "cost" of the additional interim unblinding.

Important Development Program Highlights

Phase 3 MIRACLE trial builds off of positive results of Phase 1B/2 clinical trial evaluating AnnAraC for the treatment of subjects with AML as both first line therapy and for subjects who are refractory to or relapsed after induction therapy (MB-106):
Complete Remission (CR) rate was 50% and CRc (CR plus CRi (CR with incomplete recovery of blood counts)) rate was 60% for 2nd line subjects (n=10);
Median durability: ~8 months and increasing;
Median overall survival in MB-106 was 9.1 months for 0-6 prior lines of therapies (n=22) and 11.6 months for 2nd line subjects (n=10); and
Strong efficacy signal even where a prior venetoclax combination therapy has failed.
Received US Institutional Review Board (IRB) approval;
Leveraging expertise of Catalyst Clinical Research, a leading contract research organization (CRO), with our recent engagement with them to help conduct the MIRACLE trial; and
Accelerated planned unblinded data readout to H2 2025.
Expected Milestones for Annamycin AML Development Program

1Q – 3Q 2025 – Update on MIRACLE trial site selection/approvals by countries
1Q 2025 – First subject enrolled and treated in MIRACLE trial
2025 – Recruitment update for MIRACLE trial
2H 2025 – Data readout (n=45) unblinded efficacy/safety review
2H 2025 – 2026 – Impact of data readout (n=45) on regulatory pathway; Recruitment update
1H 2026 – Interim efficacy and safety data (n=~75-90) unblinded and Optimum Dose set for MIRACLE trial
2027 – Begin enrollment of 3rd line subjects in MIRACLE2
2027 – Enrollment ends in 2nd line subjects
2028 – Primary efficacy data for 2nd line subjects in MIRACLE
2028 – Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
Soft Tissue Sarcoma (STS) Lung Metastases

As previously announced, the Company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases. Subjects who had stable disease at the time of study discontinuation were followed for progression free response and overall survival. The study database is locked, and the clinical study report is being written and should be completed in early 2025 and will be released in detail at that time.

Expected Milestones for Annamycin STS Lung Mets Development Program

2025 – Final MB-107 data readout
2025 – Identify next phase of development / pivotal IIT (investigator-initiated-trial) program
Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).

On January 8, 2025 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that the National Medical Products Administration (NMPA) of China approved GARDASIL [Human Papillomavirus Quadrivalent (Types 6, 11, 16, and 18) Vaccine, Recombinant] for use in males 9-26 years of age to help prevent certain HPV-related cancers and diseases (Press release, Merck & Co, JAN 8, 2025, View Source [SID1234649506]). The approval makes GARDASIL the first HPV vaccine approved for use in males in China. GARDASIL is now indicated in China to prevent anal cancers caused by HPV Types 16 and 18, genital warts (condyloma acuminata) caused by HPV Types 6 and 11, and the following precancerous or dysplastic lesions caused by HPV Types 6, 11, 16, and 18: grade 1, grade 2, and grade 3 anal intraepithelial neoplasia (AIN).

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"The approval of GARDASIL for use in males 9-26 years old in China is a significant step forward in advancing public health," said Joseph Romanelli, president, Human Health International, Merck. "Since first approval, our HPV vaccines have helped protect over 50 million females in China from certain HPV-related cancers and diseases. With this expanded approval, we look forward to helping protect this new population of Chinese males from certain HPV-related cancers and diseases."

Indications for GARDASIL1

GARDASIL is a vaccine indicated in females 9 through 45 years of age. GARDASIL is indicated for the prevention of cervical, vulvar, vaginal, and anal cancers caused by HPV Types 16 and 18, precancerous or dysplastic lesions caused by HPV Types 6, 11, 16, and 18, and for the prevention of genital warts caused by HPV Types 6 and 11.

GARDASIL is indicated in males 9 through 26 years of age. GARDASIL is indicated for the prevention of anal cancer caused by HPV Types 16 and 18, and precancerous or dysplastic lesions caused by HPV Types 6, 11, 16, and 18, and for the prevention of genital warts caused by HPV Types 6 and 11.

GARDASIL does not eliminate the necessity for vaccine recipients to undergo screening for cervical, vulvar, vaginal, and anal cancers as recommended by a health care provider.

GARDASIL has not been demonstrated to provide protection against diseases caused by:

– HPV types not covered by the vaccine

– HPV types to which a person has previously been exposed through sexual activity

Not all vulvar, vaginal, and anal cancers are caused by HPV, and GARDASIL protects only against those vulvar, vaginal, and anal cancers caused by HPV Types 6, 11, 16 and 18.

GARDASIL is not a treatment for external genital lesions; cervical, vulvar, vaginal, anal, oropharyngeal and other head and neck cancers; or cervical intraepithelial neoplasia (CIN), vulvar intraepithelial neoplasia (VIN), vaginal intraepithelial neoplasia (VaIN), or anal intraepithelial neoplasia (AIN).

Vaccination with GARDASIL may not result in protection in all vaccine recipients.

Select Safety Information for GARDASIL

GARDASIL is contraindicated in individuals with hypersensitivity, including severe allergic reactions to yeast, or after a previous dose of GARDASIL.

Because vaccinees may develop syncope, sometimes resulting in falling with injury, observation for 15 minutes after administration is recommended. Syncope, sometimes associated with tonic-clonic movements and other seizure-like activity, has been reported following HPV vaccination. When syncope is associated with tonic-clonic movements, the activity is usually transient and typically responds to restoring cerebral perfusion. Safety and effectiveness of GARDASIL have not been established in pregnant women. The most common (≥1.0%) adverse reactions were headache, fever, nausea, dizziness; and injection-site pain, swelling, erythema, pruritus, and bruising. The duration of immunity of GARDASIL has not been established.

Dosage and Administration

GARDASIL should be administered intramuscularly in the deltoid or anterolateral area of the thigh.

For GARDASIL, a complete vaccination regimen for individuals 9 through 26 years of age consists of 3 doses at the following schedule: 0, 2 months, 6 months.