On November 15, 2022 Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod, reported that positive data from the Phase 1b clinical trial evaluating mocravimod in allogeneic hematopoietic cell transplantation patients has been published in Transplantation and Cellular Therapy (Press release, Priothera, NOV 15, 2022, View Source [SID1234624096]).

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The study assessed the safety and tolerability of mocravimod in patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) for hematological malignancies. The secondary objectives were to determine the pharmacokinetic profile of mocravimod in this patient group as well as to assess GvHD-free, relapse-free survival at 6 months after last treatment.

The study found that mocravimod can safely be added to standard treatment regimens in patients with hematological malignancies requiring allo-HCT. CD4+ T cells were more sensitive to mocravimod treatment than CD8+ T cells. Mocravimod resulted in a significant reduction of circulating lymphocyte numbers and had no negative impact on engraftment and transplant outcomes.

Mocravimod, a sphingosine 1 phosphate (S1P) receptor modulator which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of HCT.

A global Phase 2b/3 study assessing the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in acute myeloid leukemia (AML) patients undergoing allo-HCT is planned to start in the coming months. The trial design for this study will be published as an online abstract as part of the 2022 American Society of Hematology (ASH) (Free ASH Whitepaper) conference (December 10-13th).

Florent Gros, Co-Founder and CEO of Priothera, said: "The positive Phase 1b data reinforce the potential for mocravimod to improve survival outcomes for patients with hematological malignancies requiring HCT. We’re looking forward to building upon this foundation with our upcoming pivotal Phase 2b/3 trial and advancing our lead asset towards its next significant milestones."

Elisabeth Kueenburg, M.D., Chief Medical Officer at Priothera, commented: "Mocravimod’s mode of action has already been well established in autoimmune indications, and the Phase 1b trial shows that its potential also extends to hematology. We believe mocravimod has the potential to be a first-in-class therapy in maintaining graft-versus-leukemia responses, one of the most serious complications of allogeneic HCT, while preventing graft-versus-host disease."

References
S. Dertschnig et al. Mocravimod, a selective S1PR modulator in allogeneic hematopoietic stem cell transplantation for malignancy, Transplantation and Cellular Therapy.

Online publication: View Source

About Mocravimod
Mocravimod (also known as KRP203) is a synthetic, sphingosine 1-phosphate receptor (S1PR) modulator. This novel investigational drug has been assessed in Phase 1 and Phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies – commonly known as leukemias and lymphomas – led Priothera to further develop mocravimod for the treatment of blood cancers and the improvement of CAR-T cell therapy.

Mocravimod is being investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic cell transplantation (HCT). Allogeneic HCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages mocravimod’s unique mode of action to maintain the beneficial graft-versus leukemia (GVL) activity, while reducing tissue damage resulting from graft-versus-host disease (GVHD), both a consequence of allogeneic HCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

On November 15, 2022 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a pre-commercial-stage biopharmaceutical company focused on oncology, reported third quarter financial results and recent corporate and portfolio updates (Press release, BioLineRx, NOV 15, 2022, View Source [SID1234624095]).

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"The Company delivered outstanding performance during the third quarter and subsequent period. Last week’s FDA acceptance of our new drug application for APHEXDA (motixafortide) substantially advances our twin goals of delivering an important new therapy for the mobilization of stem cells in preparation for autologous transplantation in patients with multiple myeloma, and in parallel, transitioning to a commercial stage company," said Philip Serlin, Chief Executive Officer of BioLineRx. "Importantly, we took steps that allow us to rapidly commercialize APHEXDA, if approved, including securing financing, building out our U.S. operations, and progressing our launch strategy. We believe that APHEXDA has the potential to become the standard-of-care mobilizing agent for multiple myeloma patients."

"Additionally, working with our collaborators, we advanced motixafortide development programs for pancreatic cancer, reflecting motixafortide’s potential broad clinical utility. Finally, we anticipate sharing data from the Phase 1/2a trial of our solid tumor investigational immunotherapy AGI-134 prior to year-end. We believe we are well-positioned to execute across all of our programs and continue to aggressively plan for the potential launch of APHEXDA next year," Mr. Serlin concluded.

Recent Corporate Updates

Completed $40 million non-dilutive debt financing agreement with Kreos Capital and $15 million registered direct offering to support commercial launch of APHEXDA in the U.S.
Announced APHEXDA U.S. commercialization plan and named Holly May, President, BioLineRx USA
Portfolio Execution

Motixafortide (selective inhibitor of CXCR4 chemokine receptor)

Multiple Myeloma

Announced FDA acceptance of APHEXDA NDA in stem cell mobilization for autologous transplantation in multiple myeloma patients. PDUFA target action date set for September 9, 2023
Announced presentation of cost-effectiveness analysis of motixafortide versus plerixafor in stem cell mobilization for autologous transplantation in patients with multiple myeloma at the American Society of Hematology (ASH) (Free ASH Whitepaper) 64th Annual Meeting, which is being held December 10-13, 2022, in New Orleans, Louisiana
Pancreatic Ductal Adenocarcinoma (PDAC)

Began Phase 2b PDAC randomized clinical trial preparation activities with collaboration partner GenFleet. Anticipate clinical trial initiation in 2023. The collaboration agreement allows BioLineRx to retain global rights to motixafortide in all indications
Continued collaboration progress in Columbia University investigator-initiated Phase 2 study of motixafortide in combination with an anti-PD-1 and standard-of-care chemotherapy in first-line PDAC patients
Sickle Cell Disease & Gene Therapy

Announced presentation of clinical trial study design of novel stem cell mobilization regimen with motixafortide to support gene therapy development for sickle cell patients at the ASH (Free ASH Whitepaper) Annual Meeting, which is being held December 10-13, 2022, in New Orleans, Louisiana
AGI-134 (synthetic alpha-Gal glycolipid)

Solid Tumor Immunotherapy

Advanced biomarker analysis from the Phase 1/2a trial of AGI-134 in solid tumors and anticipate announcing results from Part 2 of the trial by year-end
Third Quarter 2022 Financial Results

Research and development expenses for the quarter ended September 30, 2022, were $4.4 million compared to $4.9 million for the same period in 2021; the decrease resulted primarily from lower expenses related to motixafortide NDA supporting activities, as well as lower expenses associated with the completed motixafortide GENESIS clinical trial, offset by an increase in payroll and related expenses
Sales and marketing expenses for the quarter ended September 30, 2022, were $1.3 million compared to $0.2 million for the same period in 2021; the increase resulted primarily from initiation of pre-commercialization activities related to motixafortide, as well as an increase in market research
General and administrative expenses for the quarter ended September 30, 2022, were $1.4 million compared to $1.0 million for the same period in 2021; the increase resulted primarily from an increase in share-based compensation and small increases across several G&A expenses
Net loss for the quarter ended September 30, 2022, was $6.8 million, compared to $5.7 million for the same period in 2021
As of September 30, 2022, the Company had cash, cash equivalents, and short-term bank deposits of $57.3 million and anticipates this will be sufficient to fund operations, as currently planned, into the first half of 2024
Conference Call and Webcast Information

BioLineRx will hold a conference call today, Tuesday, November 15 at 10:00 a.m. EST. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast. A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until November 17, 2022; please dial +1-888-295-2634 from the US or +972-3-925-5903 internationally.

On November 15, 2022 Kineta, Inc. ("Kineta" or the "Company"), a clinical-stage biotechnology company focused on developing next-generation immunotherapies that address cancer immune resistance, reported that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug application (IND) to evaluate its VISTA blocking immunotherapy, KVA12123 (formerly referred to as KVA12.1), as a potential treatment for patients with advanced solid tumors (Press release, Kineta, NOV 15, 2022, View Source;utm_medium=rss&utm_campaign=kineta-announces-fda-acceptance-of-investigational-new-drug-ind-application-for-kva12123-for-the-treatment-of-advanced-solid-tumors [SID1234624094]).

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Kineta is planning to conduct a Phase 1 ⁄ Phase 2 clinical study evaluating KVA12123 as a single agent and in combination with pembrolizumab in patients with advanced solid tumors. The objectives of the study are to evaluate the safety, tolerability, pharmacokinetics and anti-tumor responses of KVA12123 alone and in combination with pembrolizumab. Kineta expects to initiate the clinical trial in the fourth quarter of 2022.

"The FDA acceptance of the IND enables Kineta to initiate the Phase 1/Phase 2 clinical trial in cancer patients with advanced solid tumor and demonstrates Kineta’s ability to execute on our clinical objectives. This marks the next stage of our journey toward developing KVA12123 as a potential important new anti-cancer immunotherapy," said Shawn Iadonato, Ph.D., Chief Executive Officer of Kineta. "We believe KVA12123 has the potential to improve clinical responses in cancer patients as a monotherapy as well as in combination with currently approved immunotherapies. We look forward to working with our clinical partners to start this study in the United States later this year and to announce interim results anticipated in late 2023."

KVA12123 is expected to be a differentiated VISTA blocking immunotherapy to address the problem of immunosuppression in the tumor microenvironment (TME). It is a fully human engineered IgG1 monoclonal antibody that was designed to bind to VISTA through a unique epitope. KVA12123 is being developed as an intravenous infusion.

Kineta is developing KVA12123 in large clinical and commercial indications where existing checkpoint inhibitors (CPIs) perform poorly, there is a high unmet medical need and VISTA expression in the tumor microenvironment is high. KVA12123 may be an effective immunotherapy for many types of cancer, including lung, colorectal, ovarian, renal cell, and head and neck as well as other "cold" difficult-to-treat solid tumors.

On November 15, 2022 Massive Bio, a leader in AI-powered cancer clinical trial enrollment, reported a strategic partnership with Azra AI, a healthcare technology company that uses proprietary AI software to identify cancer diagnoses in real time and accelerate the patient care process (Press release, Massive Bio, NOV 15, 2022, View Source [SID1234624093]). Combined with Massive Bio’s ability to surface personalized clinical trial options for patients, the two companies working together provide early identification and precise treatment options, further improving cancer clinical care.

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Today, clinical care teams have limited bandwidth to review important diagnostics like patient pathology reports. Each report takes a clinician one to two minutes to read, delaying trial opportunities for patients with an urgent life-changing health situation. Azra AI’s technology, used in over 200 hospitals including HCA Healthcare, reads pathology reports in a fraction of a second, enabling clinicians to focus on the approximately 10 percent of positive pathology reports immediately to treat patients sooner and give them the best chances for survival.

"Our mission is to create hope and empower cancer patients by helping them find their best treatment options, which often requires urgent access to trials," said Selin Kurnaz, co-founder and CEO of Massive Bio. "We are excited to collaborate with Azra AI and expand our growing ecosystem of like-minded leaders who are as committed to transforming cancer clinical trials as we are. This is a monumental point in time where advanced technology is intersecting with medical science to meaningfully change lives."

Where Azra AI focuses on identification of cancer, Massive Bio’s platform provides cancer patients with relevant clinical trials using AI, empowering patients to find treatment options faster and enabling life sciences companies to conduct broader, more-inclusive, population-based recruitment rather than traditional site-specific recruitment. The companies’ collaboration has the potential to make an immediate impact on thousands of cancer patients – Azra AI’s technology touches nearly one in 10 cancer patients in the U.S. while Massive Bio recently announced onboarding more than 100,000 patients onto its trial matching platform.

By utilizing AI technology in cancer identification and trial matching, clinical care teams can improve healthcare access and equity by eliminating unconscious bias or human errors that can prevent successful patient enrollment. Further, healthcare teams can automatically operationalize the volumes of data collected to enhance the patient experience in ways they could not do before.

"We are eager to partner with Massive Bio to connect these two parts of the cancer journey," said founder and CEO of Azra AI, Chris Cashwell. "We can revolutionize the clinical trial process by automating the identification of potential patients early on using artificial intelligence. We are combing through millions of pathology reports and identifying cancer types more quickly. The data sent to Massive Bio’s platform closes the loop on serving that patient the best treatment options for their cancer. This enables our clinical teams to focus on cancer care and use the AI to offer the best patient experience."

On November 15, 2022 Nouscom, a clinical stage immuno-oncology company developing off-the-shelf and personalized immunotherapies, reported the first patient has been dosed in a randomized Phase 2 clinical trial evaluating NOUS-209 in combination with anti-PD1 checkpoint inhibitor (CPI) pembrolizumab versus pembrolizumab alone (Press release, NousCom, NOV 15, 2022, View Source [SID1234624092]). NOUS-209 is an off-the-shelf immunotherapy targeting 209 specific neoantigens for the treatment of Mismatch Repair/Microsatellite Instable High (dMMR/MSI-H) unresectable or metastatic gastric, colorectal and gastro-esophageal junction tumors.

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Nouscom is assessing the efficacy and safety of NOUS-209 in combination with pembrolizumab at multiple sites across Europe and the US (NCT04041310). The Phase 2 study will include two cohorts in dMMR/MSI-H unresectable and metastatic colorectal cancer (CRC):

A randomized cohort enrolling patients who are eligible for first line treatment of NOUS-209 plus pembrolizumab versus pembrolizumab alone;
A single arm cohort enrolling patients who have stopped responding to previous anti-PD1 and other approved therapies
Dr Michael J. Overman, Principal Investigator of the trial and Professor in the Department of GastroIntestinal Medical Oncology at the University of Texas MD Anderson Cancer Center, said: "The continued clinical development of NOUS-209 is critical as there remains a significant unmet need in the treatment of CRC, including overcoming tumor resistance to anti-PD1 immunotherapies. Data from the Phase 1 study presented at ASCO (Free ASCO Whitepaper) 20221 and published in Science Translational Medicine2 demonstrated how NOUS-209 induces neoantigen specific CD8+ T cells which infiltrate metastatic tumors and exert anti-tumor efficacy, providing hope for better treatment options for this patient population with difficult to treat cancers."

Dr Marina Udier, Chief Executive Officer of Nouscom, added: "The initiation of the Phase 2 study is another significant milestone this year for our company. Building on our published safety, immunogenicity and mechanism of action clinical data, the trial, together with the Phase 1 ‘cancer interception’ monotherapy study in Lynch Syndrome carriers, will allow us to demonstrate the efficacy of NOUS-209 and illustrate the power of our platform. We look forward to presenting interim results at key conferences during 2023."

References

ASCO Presentation: First clinical and immunogenicity results including all subjects enrolled in a phase I study of NOUS-209, an off-the-shelf immunotherapy, with pembrolizumab, for the treatment of tumors with a deficiency in mismatch repair/microsatellite instability (dMMR), Professor Marwan G. Fakih, M.D.
A.M. D’Alise et al. Adenoviral Based-Vaccine Promotes Neoantigen Specific CD8+ T Cell Stemness And Tumor Rejection, Science Translational Medicine; 14, 657, 2022
About NOUS-209

NOUS-209 is an off-the-shelf cancer immunotherapy for Microsatellite Instable High (MSI-H) tumors. MSI-H tumors are characterized by a defective DNA mismatch repair system, which generates highly immunogenic neoantigens called frame shift peptides (FSP) that are not present in healthy tissue.

NOUS-209 encodes for 209 shared FSP neoantigens, selected by Nouscom’s proprietary GENESIS (GE(netic)NE(oantigen)S(election)I(n)S(ilico)) algorithm. In published prospective validation studies, approximately 50 of the 209 neoantigens are expressed in any one patient’s tumor. These FSPs are cloned into Nouscom’s heterologous prime / boost viral vector platform of a Great Ape Adenoviral (GAd) and Modified Vaccinia Ankara (MVA) and potently generate FSP neoantigen specific CD8+ T cells, which have been shown to successfully infiltrate tumor microenvironments to exert anti-tumor activity.

NOUS-209 is being investigated in multi-center EU and US Phase 2 randomized clinical trials in patients with dMMR/MSI-H unresectable and metastatic colorectal cancer (CRC) (NCT04041310) in combination with checkpoint inhibitors (CPI) versus CPI alone and in patients who have stopped responding to previous anti-PD1 and other approved CPI therapies.