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Nykode Therapeutics Announces Issuance of Key Patent Covering Individualized Neoantigen Based Vaccines

On August 14, 2024 Nykode Therapeutics ASA (OSE: NYKD), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 12,059,459, entitled, "Therapeutic Anticancer Neoepitope Vaccine" (Press release, Nykode Therapeutics, AUG 14, 2024, View Source [SID1234645817]).

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The newly issued patent describes Nykode’s fully individualized neoantigen based vaccine, VB10.NEO, which is in development for the treatment of locally advanced or metastatic solid tumours. The 20 year expiration date of this patent is January 5, 2037. Related patents were previously granted to the company in Russia, India, and Australia.

Michael Engsig, CEO of Nykode, said, ‘We are very pleased with the granting of this important patent in the U.S., a key market for Nykode. Continuously expanding our patent protection is a core strategy for us. The granting of this patent is a testament to the world-class innovation taking place in Nykode’s labs. We remain dedicated to pushing the boundaries of what’s possible in medicine, bringing hope to patients and their families around the globe."

About VB10.NEO

VB10.NEO is a proprietary individualized neoantigen vaccine in development for the treatment of locally advanced or metastatic solid tumors under an exclusive, worldwide clinical collaboration with Genentech, a member of the Roche Group. The vaccine is designed to be produced on-demand according to the neoantigen profile of an individual patient. Neoantigens are proteins generated by tumor-specific mutations not present in normal tissues and are thus an attractive target for cancer immunotherapy as they may be recognized as foreign by the immune system.

Nykode is currently conducting a clinical study evaluating VB10.NEO: VB N-02, an open-label Phase 1b, dose-escalation study of the safety and antigen-specific immune responses elicited by VB10.NEO in combination with Roche’s checkpoint inhibitor atezolizumab in patients with locally advanced and metastatic tumors (NCT05018273).

Biomed Valley Discoveries Announces First Patient Dosed in Phase 2 Study of Its First-in-Class ERK 1/2 Inhibitor Ulixertinib in People with Histiocytosis

On August 13, 2024 Biomed Valley Discoveries (BVD), a clinical-stage biotechnology company guided by its founders’ intent of bringing hope for life to patients, reported that the first patient has been dosed in a Phase 2 study of ulixertinib, BVD’s highly selective, first-in-class ERK 1/2 inhibitor. The study will evaluate the safety and efficacy of ulixertinib monotherapy for the treatment of histiocytosis – a group of rare, cancer-like disorders characterized by an abnormal proliferation of a type of white blood cells called histiocytes – in patients ages 12 and older who have progressed on or cannot tolerate other therapies (NCT06411821).

Eli Diamond, M.D., neuro-oncologist and early drug development specialist at Memorial Sloan Kettering Cancer Center, is the lead principal investigator for this investigator-initiated trial (IIT). The IIT was initiated following the observation of durable efficacy signals in multiple histiocytosis patients who were administered ulixertinib under BVD’s compassionate use program, per the request and under the care of Dr. Diamond.

"There is a tremendous need for new treatment options for patients with histiocytosis, many of whom progress on or cannot tolerate existing treatments," said Dr. Diamond. "I am encouraged by the compassionate use cases in which ulixertinib provided meaningful clinical benefit for histiocytosis patients who were not responding to other therapies. Based on these observations, my collaborators and I look forward to further investigating ulixertinib in this patient population and believe it has the potential to offer a much-needed new treatment approach to overcome resistance mechanisms."

Histiocytosis can cause tumors throughout the body, fractures and bone lesions, mental deterioration, lung disease, hormonal abnormalities, and many other systemic symptoms. The MAPK signaling pathway – which comprises a sequential cascade of four key signaling nodes – RAS, RAF, MEK and ERK – is implicated in many cases of histiocytosis. There are limitations to the MAPK pathway treatments currently being deployed, including emergence of resistance to treatment. Direct inhibition of ERK, the final node of the MAPK pathway, represents an important potential strategy to overcome these challenges and limitations. There are currently no approved ERK inhibitors for histiocytosis or any additional indications.

"We are pleased that the ulixertinib compassionate use cases have provided actionable clinical insights to support the initiation of this study, which may result in significant benefits for a greater number of patients in need, and we applaud Dr. Diamond’s commitment and extensive efforts to find therapeutic solutions to help patients impacted by this difficult disease," said Brent Kreider, Ph.D., president of BVD. "We believe that treatment with our investigational first-in-class ERK inhibitor, ulixertinib, has the potential to bring new hope for life to patients with histiocytosis, in line with our mission and founders’ intent."

Ulixertinib is currently under investigation for more than 10 solid and liquid tumor indications, across both pediatric and adult indications, and as both a monotherapy and in combination with other cancer treatments. To learn more, visit biomed-valley.com/ulixertinib/.

(Press release, BioMed Valley Discoveries, AUG 13, 2024, View Source [SID1234665999])

RESULTS PRESENTATION FOR THE FULL YEAR ENDED 30 JUNE 2024

On August 13, 2024 CSL reported its results presentation for the full year ended 30 June 2024 (Presentation, CSL, AUG 13, 2024, View Source [SID1234647101]).

AN2 Therapeutics Reports Second Quarter 2024 Financial Results and Recent Business and Scientific Highlights

On August 13, 2024 AN2 Therapeutics, Inc. (Nasdaq: ANTX), a biopharmaceutical company focused on discovering and developing novel small molecule therapeutics derived from its boron chemistry platform, reported financial results for the quarter ended June 30, 2024 (Press release, AN2 Therapeutics, AUG 13, 2024, View Source [SID1234646195]).

"We reported results from the EBO-301 Phase 2/3 study in patients with treatment-refractory MAC lung disease last week and over the coming months will further analyze the data to determine next steps in NTM lung disease," said Eric Easom, Co-Founder, Chairman, President and CEO. "Despite this setback, we remain well positioned as a company – we have a boron chemistry platform with two development programs that are expected to advance into clinical trials in 2025. AN2-502998 is expected to enter Phase 1 with an aim to cure chronic Chagas disease, a potentially life-threatening illness that causes severe cardiac disease and where there are limited to no treatment options. We also plan to initiate a Phase 2 trial with epetraborole for the treatment of melioidosis, a deadly bacterial infection and global bioterrorism threat. Additionally, we have a pipeline of internally developed boron-based compounds in research targeting high unmet needs in infectious disease and oncology and we have the financial runway to allow us to achieve multiple inflection points over the next three years."

Second Quarter & Recent Business Updates:

Termination of Epetraborole Pivotal Phase 2/3 Clinical Study in TR-MAC Lung Disease
AN2 recently announced topline results from the Phase 2 part of the EBO-301 Phase 2/3 study evaluating epetraborole on top of an optimized background regimen in treatment-refractory MAC lung disease. The Phase 2 part of the study met its primary objective of demonstrating the potential validation of a novel patient-reported outcome (PRO) tool and a higher PRO-based clinical response rate in the epetraborole + OBR arm vs. placebo + OBR. However, sputum culture conversion at Month 6, a key secondary endpoint, was similar between treatment arms. Based on the topline data, the Company has terminated the Phase 2 and Phase 3 parts of the EBO-301 trial. Oral epetraborole 500 mg daily was generally well-tolerated and the study was not terminated due to safety concerns. The Company plans to further analyze the EBO-301 data to better understand the results and their impact on the ongoing development of epetraborole for nontuberculous mycobacteria (NTM) lung disease.

Published New Epetraborole Data in Antimicrobial Agents in Chemotherapy
In July, the company published a study in the journal Antimicrobial Agents in Chemotherapy, which highlighted the efficacy of epetraborole against M. abscessus in a mouse lung infection model. The study suggests that epetraborole could become an important therapy to address the high unmet need for effective oral treatment options for M. abscessus lung disease.

Selected Second Quarter Financial Results

Research and Development (R&D) Expenses: R&D expenses for the second quarter of 2024 were $12.1 million compared to $13.5 million for the same period during 2023 due to decreased chemistry manufacturing and controls activity and decreased expenses for completed Phase 1 clinical trials, partially offset by increased Phase 2/3 clinical trial costs, increased consulting and outside service costs and increased personnel-related expenses.
General and Administrative (G&A) Expenses: G&A expenses for the second quarter of 2024 were $3.7 million compared to $3.1 million for the same period during 2023 due to an increase in personnel-related expenses and professional and outside services.
Other Income, Net: Other income, net for the second quarter of 2024 was $1.4 million compared to $0.8 million for the same period during 2023 due to increased interest income based on higher interest rates and higher cash, cash equivalents and investment balances.
Net loss: Net loss for the second quarter of 2024 was $14.4 million, compared to $15.8 million for the same period during 2023.
Cash Position: The Company had cash, cash equivalents, and investments of $104.5 million at June 30, 2024, which is expected to fund operations through 2027.

NorthStar Medical Radioisotopes and Convergent Therapeutics, Inc. Expand Strategic Collaboration Around CONV01-α

On August 13, 2024 NorthStar Medical Radioisotopes, LLC, a global innovator in the development, production, and commercialization of radiopharmaceuticals used for therapeutic applications and medical imaging, and Convergent Therapeutics, Inc., a clinical stage radiopharmaceutical company, reported the signing of a strategic contract manufacturing services agreement (Press release, NorthStar Medical Radiostopes, AUG 13, 2024, View Source [SID1234645837]). Convergent’s lead asset CONV01-α is a prostate-specific membrane antigen (PSMA)-targeted monoclonal antibody linked to Ac-225 and is currently being investigated as a treatment for prostate cancer.

Under the terms of the expanded collaboration, NorthStar will:

Provide Convergent with its environmentally preferred, high purity non-carrier-added (n.c.a) Ac-225 for use in CONV01-α,
Utilize its new, state-of-the-art contract development and manufacturing facility to manufacture CONV01-α to serve patients in Convergent’s clinical trials, and
Perform research and development activities to support continued development of CONV01-α and other Convergent assets.
In March of this year, Convergent received U.S. Food and Drug Administration ("FDA") clearance for its investigational new drug ("IND") application for CONV01-α, a key milestone that enables Convergent to advance CONV01-α into Phase 2 studies and expand the scope of clinical development of CONV01-α as a monotherapy and in combination with other cancer therapies. The manufacturing supply agreement announced today will support these trials and, if U.S. FDA approved, the manufacturing of CONV01-α for commercial purposes.

"The last several years have seen an explosion of research and clinical trials in the area of radiotherapies, especially those using Ac-225," said Frank Scholz, Chief Executive Officer of NorthStar, "and yet today there are still far too few effective therapies for patients suffering from a variety of serious diseases including many cancers. Why? Because the processes to develop, manufacture and deliver therapeutically effective radiopharmaceuticals like Convergent’s CONV01-α to market – in particular at a scale able to meet patient demand for an approved, marketed medicine — are highly specialized and complex. At NorthStar, our passion is to reduce technological and operational barriers to help companies like Convergent Therapeutics not only secure a reliable source of Ac-225, but manufacture a finished, patient-ready drug product and continue to develop new and additional therapeutic indications for the patients who need new and better medicines. We are proud to be supporting Convergent and look forward to working with them to help make new medicines like CONV01-α therapeutic reality."

"We are excited to expand our partnership with NorthStar and the opportunity to leverage NorthStar’s unique position to scale both Ac-225 generation and manufacturing capacity," said Caitlyn Harvey, Head of Manufacturing at Convergent. "Convergent will begin enrolling patients into the CONVERGE-01 Phase 2 clinical trial in the next couple of months as we continue building on extensive clinical experience which will allow us to rapidly move into our Phase 3 study."

About CONV01-α

CONV01-α, Convergent’s alpha emitting radioantibody, combines the precision and pharmacokinetics of antibodies with the tumor-killing potential of alpha emitting radionuclides. Specifically, CONV01-α uses a humanized monoclonal antibody targeted at prostate-specific membrane antigen (PSMA) which is highly overexpressed in prostate cancer cells. Since PSMA is a validated target, several therapeutics are directed at this antigen and CONV01-α is differentiated by its use of both an antibody and alpha emitter. CONV01-α is linked to a powerful radionuclide called Ac-225, which releases alpha particles which kill cancer cells through DNA double strand breaks. Unlike other radioactive sources, alpha particles deliver high-energy radiation over very short distances, thereby minimizing radiation exposure to healthy neighboring cells and tissues. Pairing highly selective antibodies with such a powerful yet precise payload offers the ideal combination to treat many types of cancers.