On July 30, 2024 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported that management will be attending the following upcoming investor conferences (Press release, Delcath Systems, JUL 30, 2024, View Source [SID1234645153]):

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BTIG Virtual Biotechnology Conference
Date: Tuesday, August 6, 2024
Location: Virtual

Canaccord Genuity’s 44th Annual Growth Conference
Date: Wednesday, August 14, 2024
Presentation Time: 9:30 am Eastern Time
Location: Boston, MA

On July 30, 2024 Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) ("Day One" or the "Company"), a biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported its second quarter 2024 financial results and highlighted recent corporate achievements (Press release, Day One, JUL 30, 2024, View Source [SID1234645152]).

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"We had an outstanding quarter across all facets of our business," said Jeremy Bender, Ph.D., chief executive officer of Day One. "Demand for OJEMDA led to strong early launch performance following our first approval, and we made significant progress advancing our programs and pipeline, including the addition of DAY301, a potential first-in-class ADC targeting PTK7 that we expect to be in the clinic in the coming months."

Program Highlights

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OJEMDA received U.S. Food and Drug Administration (FDA) accelerated approval in April 2024. It is the first and only FDA approved therapy for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

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Day One provided updated duration of treatment data from the registrational Phase 2 FIREFLY-1 trial investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pLGG. For the 77 patients enrolled on Arm 1, which was the dataset used to assess OJEMDA’s efficacy, the median duration of treatment is now 23.7 months, with some patients being on treatment out to 32 months. Additional analyses will be presented at future medical conferences.

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Day One and Ipsen entered into an exclusive licensing agreement to commercialize tovorafenib outside of the U.S. in July 2024. Under the agreement, Day One will receive approximately $111 million upfront in cash and equity investment at a premium with up to approximately $350 million in additional launch and sales milestone payments as well as tiered double-digit royalties starting in mid-teens percentage on net sales. Ipsen secured commercialization rights to tovorafenib outside of the U.S.

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Day One entered into an exclusive licensing agreement with MabCare Therapeutics for its novel ADC targeting protein-tyrosine kinase 7 (PTK7) in June 2024. The Company expects to dose the first patient in the Phase I portion of the Phase 1/2a clinical trial of DAY301 in the fourth quarter of 2024 or first quarter of 2025.

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Day One presented a poster on tovorafenib demonstrating reversibility of changes in growth velocity observed in the Phase 2 FIREFLY-1 clinical trial at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. These data were also shared at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO).

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Day One made the decision to close the pimasertib program in July 2024, including the FIRELIGHT-1 trial evaluating it in combination with tovorafenib. Resources will be redirected to the DAY301 program and results will be shared at a future medical meeting or publication.

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The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial evaluating tovorafenib as a front-line therapy in patients aged 6 months to 25 years with pLGG continues to enroll patients in the United States, Canada, Europe, Australia and Asia, with more than 100 sites activated.

Corporate Highlights and Upcoming Milestones

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Day One announced it entered into a definitive agreement for an oversubscribed private placement of its securities for total gross proceeds of approximately $175.0 million in July 2024.

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The Company sold the rare pediatric disease Priority Review Voucher awarded by the FDA upon OJEMDA’s approval for total cash proceeds of $108.0 million in May 2024, representing a gain on sale.

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Commercial operations veteran John Stubenrauch joined Day One in July 2024 as Chief Technology Officer. Dr. Stubenrauch, PhD, MBA, was most recently Chief Operating Officer at Nutcracker Therapeutics and brings more than 25 years of experience developing and commercializing medicines, including ADCs, as well as a broad range of product modalities.

Second Quarter 2024 Financial Highlights

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Cash Position: The Company’s cash, cash equivalents and short-term investments totaled $361.9 million as of June 30, 2024.

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Product Revenue, Net: OJEMDA net product revenues were $8.2 million for the second quarter of 2024, the first partial quarter of the U.S. launch.

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R&D Expenses: Research and development expenses were $92.1 million for the second quarter of 2024 compared to $32.2 million for the second quarter of 2023. The increase was primarily due to the MabCare Therapeutics license agreement upfront payment of $55.0 million, increased clinical trial activities related to tovorafenib, and additional employee compensation costs.

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SG&A Expenses: Selling, general and administrative expenses were $30.2 million for the second quarter of 2024 compared to $17.1 million for the second quarter of 2023. The increase was primarily due to additional employee compensation costs, commercial launch activities, and increased professional service expenses to support company growth.

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Net Loss: Net loss totaled $4.4 million for the second quarter of 2024 with non-cash stock-based compensation expense of $13.0 million, compared to $45.9 million for the second quarter of 2023 with non-cash stock-based compensation expense of $9.5 million.

Upcoming Events

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2024 Wedbush PacGrow Healthcare Conference, August 12-14, 2024

Conference Call

Day One will host a conference call and webcast today, July 30 at 8:00 a.m. Eastern Time. To access the live conference call by phone, dial 877-704-4453 (domestic) or 201-389-0920 (international), and provide the access code 13745150. Live audio webcast will be accessible from the Day One Investors & Media page. To ensure a timely connection to the webcast, it is recommended that participants register at least 15 minutes prior to the scheduled start time. An archived version of the webcast will be available for replay on the Events & Presentations section of the Day One Investors & Media page for 30 days following the event.

About OJEMDA

OJEMDA (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases.

OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.

For more information, please visit www.ojemda.com.

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 Chemomab Therapeutics Ltd. (Nasdaq: CMMB) ("Chemomab" or the "Company"), a clinical stage biotechnology company developing innovative therapeutics for fibro-inflammatory diseases with high unmet need, reported that it had closed a private placement that resulted in gross proceeds of approximately $10 million to the Company, before deducting offering expenses payable by the Company (Press release, Chemomab, JUL 30, 2024, View Source [SID1234645150]).

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Pursuant to the terms of the transaction, the Company sold a total of 8,097,167 ADSs and pre-funded warrants in lieu of ADSs. The purchase price per ADS was $1.235. The private placement did not include warrant coverage. Both existing investors and new investors participated in the private placement, including HBM Healthcare Investments, OrbiMed and Sphera Biotech Master Fund LP.

Oppenheimer & Co. Inc. acted as Capital Markets Advisor to the Company for the private placement. Other advisors to the Company in the private placement included LifeSci Capital and Maxim Group.

The offer and sale of the securities sold in the private placement were made in a transaction not involving a public offering and the securities have not been registered under the Securities Act of 1933, as amended, and may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements. As part of the private placement, the Company and the investors entered into a registration rights agreement pursuant to which the Company has agreed to file a registration statement with the Securities and Exchange Commission (the "SEC") registering the resale of the ADSs, including ADSs issuable upon exercise of the Pre-Funded Warrants, issued in the private placement.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On July 30, 2024 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported positive topline data from the ongoing Phase 2 clinical trial (EudraCT No.: 2020-002195-12; NCT04526899) in patients with unresectable stage III or IV melanoma whose disease had progressed following anti-PD-(L)1-containing treatment (Press release, BioNTech, JUL 30, 2024, View Source [SID1234645149]). The randomized trial evaluates the clinical activity and safety of the investigational mRNA cancer immunotherapy BNT111 in combination with Libtayo (cemiplimab), an anti-PD-1 monoclonal antibody being developed by Regeneron, and assesses the two single agents alone.

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The trial met its primary efficacy outcome measure, demonstrating a statistically significant improvement in ORR in patients treated with BNT111 in combination with cemiplimab as compared to historical control in this indication and treatment setting. Both randomized monotherapy arms showed clinical activity. The ORR in the cemiplimab monotherapy arm was in line with the historical control of anti-PD-(L)1 or anti-CTLA-4 treatments in this patient group. The treatment was well tolerated and the safety profile of BNT111 in combination with cemiplimab in this trial was consistent with previous clinical trials assessing BNT111 in combination with anti-PD-(L)1-containing treatments. The Phase 2 trial will continue as planned to further assess the secondary endpoints, which were not mature at the time of the primary analysis.

"These Phase 2 results mark a significant step towards our vision of personalized cancer medicine. We envision mRNA as a centerpiece in future treatment paradigms for cancer, helping to address unmet medical needs, such as for patients with anti-PD-(L)1 refractory or resistant melanoma," said Prof. Özlem Türeci, M.D., Chief Medical Officer and Co-Founder at BioNTech. "These data are a proof of concept for us in three dimensions: First, for our decade-long improved mRNA cancer vaccine technology that uses uridine mRNA chemistry, a non-coding backbone that is engineered for optimal translational performance and our proprietary lipoplex formulation for delivery. Second, for our computational approaches for selecting suitable tumor antigens for our cancer indication-specific FixVac platform candidates. Third, for our strategy to combine synergistic modalities, in this case BNT111 with an established immune checkpoint treatment."

BioNTech and Regeneron plan to present data from this trial at a forthcoming medical conference. Further, the companies also intend to submit these data for publication in a peer reviewed scientific journal.

BNT111 is based on BioNTech’s fully owned FixVac platform that utilizes a fixed combination of four mRNA-encoded, tumor-associated antigens designed to trigger an innate and tumor-antigen-specific immune response against cancer cells expressing one or more of the respective tumor antigens. In 2021, BNT111 in combination with cemiplimab received Fast Track designation by the FDA for the treatment of anti-PD-1-refractory/relapsed, unresectable Stage III or IV melanoma. In the same year, the FDA granted Orphan Drug designation for BNT111 the treatment of stage IIB through IV melanoma.

About BioNTech’s oncology mRNA platforms
BioNTech has developed a range of mRNA platforms to establish a novel class of therapeutics and vaccines aimed at improving the health of people worldwide. In oncology, BioNTech utilizes five mRNA platforms. Each platform is designed with the aim to address unique challenges in oncology. BioNTech’s fully owned FixVac (Fix Combination Vaccine) platform candidates target specific cancer indications focusing on tumor-associated antigens which are shared by many cancer patients, while iNeST (Individualized Neoantigen Specific Immunotherapy) platform candidates are personalized immunotherapies tailored to the patient’s individual tumor profile. Both platforms utilize BioNTech’s proprietary optimized uridine mRNA ("uRNA") technology and the lipoplex ("LPX") delivery technology that the scientific founders and the researchers at BioNTech have pioneered over decades of scientific discoveries and technological advancements. These technologies are optimized for immunotherapy applications aiming to boost the immunostimulatory effect of the investigational immunotherapies and to trigger targeted immune responses against cancer cells expressing one or more of the respective encoded tumor antigens. Currently, six programs based on the Company’s FixVac and iNeST platforms are being evaluated in randomized Phase 2 trials in various solid tumor indications.

In addition to the FixVac and iNeST platforms, BioNTech is leveraging mRNA to deliver the building plan for targeted antibody, cytokine or immunomodulating protein approaches directly to the patient based on the Company’s RiboMab, RiboCytokine and Intratumoral Immunotherapy platforms aiming to help the body to produce its own therapeutic.

About advanced melanoma
Melanoma is amongst the leading causes of cancer-related deaths globally, responsible for roughly 58,000 deaths yearly. 1 Anti-PD-1 refractory/relapsed unresectable Stage III or IV melanoma is an aggressive form of melanoma, which remains particularly lethal. Current standard of care includes checkpoint inhibitor therapies that substantially improve the life expectancy of patients with melanoma.2,3 Despite advances in treatment, a high proportion of patients exhibit resistance to approved therapies, leading to limited options for those who progress on targeted or immunotherapy.4 The 5-year survival rate for patients with distant metastatic melanoma is approximately 35%.5 This underscores the significant unmet medical need.

About BNT111
BNT111 is an mRNA-based off-the-shelf cancer immunotherapy candidate for intravenous administration encoding a fixed set of four non-mutated melanoma-associated antigens (NY-ESO-1, MAGE-A3, tyrosinase, and TPTE) delivered as uridine mRNA-lipoplex formulation. Over 90% of patients with cutaneous melanomas express at least one of these antigens.6 Data of the Lipo-MERIT Phase 1 clinical trial have shown that BNT111 alone or in combination with blockade of the checkpoint inhibitor PD-1 induces novel antigen-specific anti-tumor immune responses and enhances pre-existing immune responses against the encoded melanoma-associated antigens in checkpoint inhibitor-experienced patients with unresectable melanoma.7 Further, these data have shown that the candidate can prime and activate T cells against the vaccine antigens that persisted for more than one year under continuous monthly vaccination.7 BNT111 is one of three clinical-stage FixVac product candidates within BioNTech’s development pipeline. The candidate is currently being evaluated in a Phase 2 clinical trial in combination with cemiplimab, in patients with anti-PD-1 refractory/relapsed unresectable Stage III or IV melanoma.

About the BNT111-01 trial
The BNT111-01 trial (EudraCT No.: 2020-002195-12; NCT04526899) is an open-label, randomized Phase 2 trial evaluating the efficacy of BNT111 and cemiplimab and the contribution of the single components in patients with anti-PD-1/PD-L1-refractory or relapsed, unresectable Stage III or IV cutaneous melanoma. Conducted across approximately 60 sites in 7 countries, this multi-site trial aims to demonstrate anti-tumor activity and ORR of the combination therapy as well as each agent alone. Additional endpoints include duration of response (DOR), disease control rate (DCR), overall survival (OS), safety, and tolerability. Patients were randomized in a 2:1:1 ratio to Arm 1 (BNT111 + cemiplimab), Arm 2 (BNT111 monotherapy), and Arm 3 (cemiplimab monotherapy), with up to 24 months of active treatment. More information on this trial can be found at clinicaltrials.gov or www.clinicaltrialsregister.eu.