On July 30, 2024 Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) (Processa or the Company), a clinical-stage pharmaceutical company focused on developing the next generation of chemotherapeutic drugs with improved efficacy and safety, reported that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for Next Generation Capecitabine (NGC-Cap), its lead product candidate (Press release, Processa Pharmaceuticals, JUL 30, 2024, View Source [SID1234645169]). The IND supports the initiation of a Phase 2 clinical trial in patients with advanced or metastatic breast cancer, which is expected to begin enrollment this quarter.

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"We are proud to achieve this significant milestone for NGC-Cap and look forward to entering the clinic for the treatment of advanced or metastatic breast cancer, where capecitabine is a standard of care. We previously demonstrated in our Phase 1b study that NGC-Cap is more potent than monotherapy capecitabine, providing up to 5-10 times more 5-fluorouracil exposure to cancer cells. This greater exposure resulted in a greater efficacy, with a safety profile better or similar to existing monotherapy with capecitabine," stated David Young, PharmD, Ph.D., President of Research and Development. "Initial data from the Phase 2 trial are expected mid-2025."

"Although capecitabine is among the most widely used chemotherapy drugs, particularly for the treatment of solid tumors, there remains the need for a more effective chemotherapy treatment with fewer or less-severe side effects," he added. "We believe that NGC-Cap can fulfill this need."

Breast cancer is the second most common cancer and a leading cause of cancer-related death. More than 2 million cases of breast cancer were diagnosed in 2022 with more than 665,000 deaths globally. The five-year survival rate for those diagnosed with metastatic disease is approximately 30%.

The Phase 2 study will be a global multicenter, open-label, adaptive design trial comparing two different doses of NGC-Cap to FDA-approved monotherapy capecitabine in approximately 60 to 90 patients with advanced or metastatic breast cancer. The trial is designed to evaluate the safety-efficacy profile of NGC-Cap versus monotherapy capecitabine, to determine the potential optimal dosage regimens of NGC-Cap as required by the FDA Project Optimus Initiative and to evaluate the possibility of personalizing NGC-Cap therapy. Processa expects to enroll the first patient into this trial in the third quarter of 2024.

About Capecitabine Administered with PCS6422 (NGC-Cap)

NGC-Cap combines the administration of PCS6422, the Company’s irreversible dihydropyrimidine dehydrogenase (DPD) enzyme inhibitor, with low doses of capecitabine. Capecitabine is the oral prodrug of 5-FU, and along with 5-FU is among the most widely used chemotherapy drugs, particularly for the treatment of solid tumors. When metabolized (after oral ingestion) it becomes 5-FU in the body, which, in turn, metabolizes to molecules called anabolites that actively kill duplicating cells, such as cancer cells, and to molecules called catabolites that only cause side effects. The presence of the DPD enzyme plays an integral role in the undesirable conversion of 5-FU to catabolites while simultaneously decreasing tumor exposure to 5-FU and it’s anabolites.

The NGC-Cap Phase 1b study evaluated ascending doses of capecitabine when combined with a fixed dose of PCS6422 in patients with advanced, relapsed or refractory progressive gastrointestinal tract cancer. These patients had to relapse from or fail all other treatments. NGC-Cap demonstrated greater 5-fluorouracil (5-FU) exposure and lower fluoro-beta-alanine (FBAL) exposure with a better or similar side effect profile compared with monotherapy capecitabine, as well as preliminary anti-tumor activity. In all evaluable patients who received one dose of PCS6422 and seven days of capecitabine, partial responses or stable disease was observed in 66.7% (8 out of 12) of patients with progression-free survival of approximately 5 to 11 months across these patients.

On July 30, 2024 LTZ Therapeutics, an immunotherapy-focused biotech company, reported the completion of the company’s Series A financing of over $20 million, to advance the development of its Myeloid Engager pipeline to treat cancer and autoimmune diseases (Press release, LTZ Therapeutics, JUL 30, 2024, View Source [SID1234645187]). This round was led by new investor Lapam Capital and includes new investment participation from GL Ventures. In addition, the company has received continued investment from K2 Venture Partners and Shunwei Capital. The closing of this round brings LTZ’s total funding to approximately $50 million since the company was founded in 2022.

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Proceeds from the financing will be used to accelerate development of LTZ’s pipeline, supporting the company’s Investigational New Drug (IND) process, initiating the Phase 1 clinical study of LTZ’s lead asset LTZ-301 as well as the IND-enabling work of its second asset, LTZ-232. The company will also use the new capital to further advance other programs in discovery and to expand its team.

"This round represents an important step forward in the development of our novel Myeloid Engager Platform for cancer and other conditions with unmet medical needs," said Robert Li, Ph.D., Founder and CEO of LTZ. "At the heart of our approach is the fusion of reverse translational science with a deep understanding of tumor microenvironment (TME) biology, especially myeloid biology. We’ve made significant progress this past year, not only validating the company’s platform in various preclinical test systems including cancer patient-derived organoid models, but also advancing our lead asset LTZ-301 from discovery to an IND-enabling stage. We are thankful for the ongoing support of our scientific team, advisory board and our syndicate of investors, making all of our accomplishments possible."

Macrophages appear to be one of the most prevalent immune cell populations in TME of various hematologic and solid tumors. Therefore, effectively engaging and activating macrophages to kill cancer cells represents significant therapeutic potential for patients. Based on reverse translational research to inform new discoveries and the emerging myeloid biology of TME in a broad range of cancer types, LTZ is developing its own novel Myeloid Engager Platform to primarily enhance the phagocytic function of monocytes and macrophages to foster anti-tumor immunity.

"LTZ comprises a highly skilled and passionate team that brings together a unique complement of expertise," said Zhihua Yu, Founding Partner at Lapam Capital. "We are excited about the reverse translational approach LTZ is taking to maximize the drugability of their assets. LTZ’s Myeloid Engager Platform and their pipeline have the potential to greatly impact the future of immunotherapy development, aiming to rebalance the immune system and improve patient outcomes. The preclinical results thus far carry promising clinical benefits and suggest applicability of LTZ’s model for a wide spectrum of cancer indications (liquid and solid) and autoimmune diseases, where the unmet need is incredibly high."

On July 30, 2024 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported that it has entered into an exclusive license agreement with Cortice Biosciences, Inc. ("Cortice") (Press release, CNS Pharmaceuticals, JUL 30, 2024, View Source [SID1234645151]). The Company will host a live webcast presentation to discuss the transaction on Tuesday, July 30, 2024 at 8:30 AM ET (details below). Additionally, CNS announced the launch of its new corporate branding and website, cnspharma.com.

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Under the terms of the Agreement, CNS Pharmaceuticals has obtained an exclusive license and the intellectual property rights to TPI 287, a potentially blood brain barrier permeable microtubule inhibitor, currently in development for the treatment of GBM, in exchange for an upfront payment of 616,698 shares of the Company’s common stock, as well as the possibility of future success-dependent milestone payments of cash or the Company’s common stock to Cortice. CNS Pharmaceuticals intends to advance the development of TPI 287 for an oncology indication in the United States, Canada, Mexico, and Japan, which is the territory covered by the Agreement (the "Territory"). Such development efforts will include, but may not be limited to, the prosecution and maintenance of existing and new intellectual property; preclinical and clinical development of TPI 287 including research, manufacturing, laboratory and clinical testing, regulatory filing, and marketing of TPI 287 in the Territory.

John Climaco, CEO of CNS Pharmaceuticals, stated, "For years, our team has searched for another drug candidate with the same high level of human data-supported therapeutic potential in GBM as Berubicin. The in-licensing of TPI 287 is a transformational step forward and we are prepared for the next stage to execute our vision of CNS Pharmaceuticals being the leading biopharma company developing drugs for this devastating and currently inescapably fatal disease."

"Our vision is anchored by our confidence in and commitment to our trial of Berubicin in patients with recurrent GBM. The 252 patients enrolled in its potentially pivotal trial will provide significant data about overall survival compared with Lomustine, the outcome of which will be made public in the first half of next year. Our highly experienced team that created and is executing this trial – currently one of the largest GBM studies being conducted anywhere in the world – makes us uniquely positioned to meet the challenge presented by this disease. The clinical network we have established is unparalleled by any other company’s GBM development program, and as a consequence, the TPI 287 program will require only limited capital resources prior to the release of Berubicin topline data. This will allow us to drive TPI 287 into potential registration studies in the most cost-effective manner possible. After negotiations spanning several years and following extensive scientific and clinical due diligence, we believe the highly compelling safety and efficacy data demonstrated by TPI 287 in over 350 patients to date makes it both the ideal complementary asset to Berubicin and the perfect next step in our Company’s strategic plan. Our work on bringing TPI 287 to patients begins immediately," added Mr. Climaco.

TPI 287 Key Highlights

· TPI 287 is an abeotaxane and has the same mechanism of action as other taxanes, e.g. paclitaxel (Taxol) and docetaxel, in which it stabilizes microtubules and inhibits cell division, causing apoptosis and cell death. While most taxanes are substrates for multi-drug resistant transporters, which maintain the blood brain barrier (BBB), similarly to Berubicin, TPI 287 has shown the potential to cross the BBB and treat CNS tumors.

· TPI 287 has been well tolerated in over 350 patients to date, including in clinical trials as a monotherapy and in combination with bevacizumab for the treatment of recurrent neuroblastoma and medulloblastoma, as well as refractory prostate cancer and melanoma, and in tauopathy disease, which can result in dementia.

· In a multicenter Phase 1 study evaluating TPI 287 in combination with bevacizumab in patients with recurrent GBM, results demonstrated an objective response rate of 60% and disease control rate of 96% in 23 subjects. Progression-free survival (PFS) of 5.5 months and overall survival (OS) of 13.4 months compare favorably to bevacizumab either as monotherapy or in combination with chemotherapy in similar patients yielding PFS of 2-4 months and OS of 6-9 months. The data from this study were recently published in a manuscript titled, "Phase 1 trial of TPI 287, a microtubule stabilizing agent, in combination with bevacizumab in adults with recurrent glioblastoma1," in Neuro-Oncology Advances.

· CNS Pharmaceuticals plans to engage the U.S. FDA and obtain feedback on the design of a study focused on the registration of TPI 287 in recurrent GBM, with the goal of initiating the study in 2025.

Samuel A. Goldlust, MD, Medical Director of Neuro-Oncology at Saint Luke’s Cancer Institute, a former investigator in the Company’s global study of Berubicin, as well as the principal investigator of studies of TPI 287 in GBM added, "The data seen to date with TPI 287 have been highly encouraging. There remains a tremendous unmet need for the GBM patient population, which I believe will require the development of a variety of effective therapeutic approaches. With the promising data demonstrated with both Berubicin and the synergies that TPI 287 has shown, I am excited for the Company to further explore and unlock the potential of TPI 287."

As previously announced in April 2024, the Company completed enrollment in its global potentially pivotal study evaluating Berubicin for the treatment of GBM. In December 2023 the Company announced the successful completion of its pre-planned interim futility analysis and received a recommendation from the independent Data Safety Monitoring Board (DSMB) to continue the study without modification. CNS Pharmaceuticals expects to report topline results from its potentially pivotal study of Berubicin in the first half of 2025.

"We also fully understand that the complexity and severity of GBM challenges scientists and clinicians to create novel treatment approaches for brain malignancies. As we have grown our expertise in the development of blood brain barrier permeable chemotherapeutics, we understand that multiple therapeutics may be required to effectively treat these diseases. Combinations of anthracyclines and taxanes that have shown activity for systemic disease may be more powerful as combination agents for the treatment of diseases metastatic to the brain. There is tremendous potential therapeutic synergy between Berubicin and TPI 287, and we are excited to expand our pipeline of drug candidates to offer patients with recurrent GBM an additional brain-penetrative chemotherapeutic option," added, Sandra Silberman, MD, PhD, Chief Medical Officer of CNS. "Having successfully completed enrollment in our Berubicin study, we have gained extensive experience and expertise in conducting late-stage registrational studies for recurrent GBM. That experience will now inform our clinical strategy for TPI 287 as we engage with key investigators at our active clinical sites. Our investigator network, which took years to build, can now be repurposed to save valuable time and resources, allowing us to expeditiously move forward with a similar potentially registrational study of TPI 287 in 2025."

Webcast Details

CNS Pharmaceuticals will host a live video webcast presentation with members of management and neuro-oncologist and Key Opinion Leader, Dr. Samuel Goldlust for investors, analysts, and other interested parties today, June 30, 2024 at 8:30 a.m. ET to discuss the transaction. Interested participants may register for the event here. The live webcast will be accessible on the Events page of the Investors section of the CNS website, cnspharma.com, and will be archived for 90 days.

On July 30, 2024 Replimune Group, Inc. (Nasdaq: REPL), a clinical stage biotechnology company pioneering the development of a novel class of oncolytic immunotherapies, reported that members from the Replimune management team will host investor meetings at the following two conferences (Press release, Replimune, JUL 30, 2024, View Source [SID1234645170]):

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BTIG Virtual Biotechnology Conference
Dates: August 5-6, 2024

2024 Wedbush PacGrow Healthcare Conference
Date: Tuesday, August 13, 2024

On July 30, 2024 Tempus AI, Inc. (NASDAQ: TEM), a leader in artificial intelligence and precision medicine, reported an expanded collaboration with Remix Therapeutics, a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address underlying drivers of disease (Press release, Tempus, JUL 30, 2024, View Source [SID1234645188]). The collaboration between Remix and Tempus began with the licensing of specific, de-identified data cohorts and has since expanded into a broader, strategic alliance.

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Remix is leveraging Tempus’ multimodal data to interrogate specific cohorts, including Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia (AML) with Tempus’ data analytics platform, Lens. The newly expanded scope of work also includes next-generation sequencing support for Remix’s Phase I trial for REM-422, the company’s potent, selective, oral small molecule messenger RNA (mRNA) degrader.

To support its research, Remix is utilizing Tempus’ xT and xR assays to capture DNA and RNA data, as well as tracking treatment response on an exploratory research basis with xM Monitor, the company’s circulating tumor DNA (ctDNA) assay, which detects and monitors changes in circulating tumor fraction to determine response to therapy for patients with advanced cancers.

"We’re excited to work with a biotech like Remix that understands and embraces the value that Tempus’ multimodal data can bring to their important work," said Ryan Fukushima, Chief Operating Officer of Tempus. "We were able to quickly expand our collaboration and provide Remix with an array of our offerings that are uniquely positioned to support them in achieving their research and development goals."

"Tempus’ multimodal data, analytics, and sequencing support will be invaluable tools as we advance our lead candidate, REM-422, into the clinic," said Dominic Reynolds, Ph.D., Chief Scientific Officer of Remix. "This collaboration provides us with robust resources and data to propel our research forward, ultimately advancing our goal of creating meaningful new treatment options for patients."