Blacksmith Medicines Announces $3.3M in Funding from NIAID to Support Clinical Trial Manufacturing of Novel Antibiotic

ON February 21, 2024 Blacksmith Medicines, Inc. (Blacksmith), a leading biopharma dedicated to discovering and developing medicines targeting metalloenzymes, reported the release of $3.3M from its contract with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), Department of Health and Human Services (HHS), to conduct IND-enabling and Phase 1 studies of FG-2101, its novel non-hydroxamate LpxC inhibitor, using both intravenous (IV) and oral administration for the treatment of urinary tract infections caused by multidrug-resistant Gram-negative bacteria (Press release, Blacksmith Medicines, FEB 21, 2024, View Source [SID1234643532]).

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The company’s recent successful completion of certain Base Period activities including preclinical toxicology studiestriggered the release of this award under its contract with NIAID (75N93022C00060). The total funding could be up to $17.2M over five years, depending on contract options exercised. The new funding will be used for GMP clinical material manufacturing and IND preparation/submission.

"With support from NIAID, we have successfully completed preclinical toxicology studies including 7-day and 14-day repeat dose assessments (non-GLP and GLP) and safety pharmacology evaluations including cardiovascular, respiratory and CNS assessments of our novel non-hydroxamate LpxC inhibitor FG-2101," said Andrew Tomaras, Ph.D., Senior Vice President of Blacksmith. "Next, with the achievement of these safety studies, we will initiate the preparation of an investigational new drug (IND) application as well as the manufacturing campaign of GMP material for upcoming clinical studies. We are extremely grateful to the NIAID team for their continued support for the candidacy of our potential first-in-class antibacterial agent."

About LpxC

LpxC, a zinc metalloenzyme, is an attractive and highly sought-after antibiotic target – it is conserved across Gram-negative bacteria and not found in Gram-positive bacteria or human cells. Inhibiting LpxC results in potent killing of Gram-negative bacteria with the presumed benefit of sparing Gram-positive bacteria such as those residing in the protective microbiome of the gut which help to deter opportunistic C. difficile infections.

Other LpxC inhibitors have been evaluated by biopharma in the past but chemistry limitations (e.g. hydroxamic acid) have yielded ineffective compounds that suffer from poor drug-like properties. Thus, there are no approved therapeutics targeting LpxC. Blacksmith, using its proprietary chemistry platform, has developed novel non-hydroxamate inhibitors of LpxC that are safe and effective in animal models of Gram-negative infection and are able to kill Gram-negative ‘superbugs’ where other antibiotics are ineffective.

About metalloenzymes and the Blacksmith platform

Metalloenzymes utilize a metal ion cofactor in the enzyme active site to perform essential biological functions. This diverse class of targets has historically been difficult to drug due to small molecule chemistry limitations that have plagued the industry. The Blacksmith metalloenzyme platform has solved this problem by leveraging the following:

A large proprietary fragment library of metal-binding pharmacophores (MBPs);
A comprehensive database containing a full characterization of the metalloenzyme genome including functions, metal cofactors, and associations to disease;
A first-of-its-kind metallo-CRISPR library of custom single guide RNAs;
An industry-leading metalloenzyme computational toolkit for docking, modeling and structure-based drug design; and
A robust and blocking intellectual property estate covering bioinorganic, medicinal, and computational chemistry approaches for metalloenzyme-targeted medicines.

Aleta Biotherapeutics and Cancer Research UK’s Centre for Drug Development Announce First Patient Dosed in ALETA-001 Phase 1/2 Clinical Trial in Patients with Relapsed/Refractory B-Cell Malignancies

On February 21, 2024 Aleta Biotherapeutics (Aleta), a clinical stage, immuno-oncology company with a CAR T-Cell Engager (CTE) platform that enables cell cancer therapies to work more effectively, and Cancer Research UK’s Centre for Drug Development, reported the first patient was dosed in a Phase 1/2 clinical trial (Press release, Aleta Biotherapeutics, FEB 21, 2024, View Source [SID1234640353]). This trial is evaluating the Company’s first-in-class biologic CAR T-Cell Engager, ALETA-001, for the treatment of patients with B-cell malignancies who are relapsed/refractory to CD19-targeting CAR T-cell therapy.

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Cancer Research UK’s Centre for Drug Development is sponsoring and conducting the Phase 1/2 clinical trial of ALETA-001, the lead agent in Aleta’s portfolio.

"It is very exciting and meaningful to have ALETA-001 now in the clinic. Clinical evaluation of ALETA-001 is a key milestone toward a much-needed treatment option for the many cancer patients whose CD19-targeted CAR T-cell therapies ultimately stop working. ALETA-001 restores and increases the effectiveness with which CAR T-cells can kill cancer cells, and we believe that it will enable more patients to successfully benefit from cell therapies," stated Dr. Paul Rennert, President and Chief Scientific Officer, Aleta Biotherapeutics.

The ALETA-001 Phase 1/2 clinical trial is an open-label, dose-expansion trial which will evaluate safety and tolerability, pharmacokinetic and pharmacodynamic effects, and early signals of clinical efficacy of ALETA-001 as a single agent. Chief Investigator Dr. Sridhar Chaganti is leading the trial from University Hospital Birmingham NHS Foundation Trust in Birmingham, UK, and will enroll patients who are relapsed/refractory following treatment with available CD19-targeting CAR T-cell therapies. For more information on this trial, please visit clinicaltrials.gov (NCT06045910)

Dr. Lars Erwig, Cancer Research UK’s Director of Drug Development, said, "A significant number of patients with blood cancers unfortunately relapse following CD19-directed CAR T-cell therapy. ALETA-001 is being developed to provide patients with these cancers a better chance for a successful treatment outcome. At Cancer Research UK, we are very excited to study the clinical potential of ALETA-001 to transform a patient’s treatment journey – which can possibly be lifesaving in many cases."

"Aleta-001 is a uniquely designed new molecule with a novel mechanism of action, coating tumor cells densely with the target antigen, thereby stimulating the tumor killing ability of activated CAR T-cells," commented Dr. Sridhar Chaganti, Chief Investigator, University Hospital Birmingham NHS Foundation Trust, Birmingham, UK. "This is an important new strategy being investigated to improve outcomes for patients with relapsed or refractory lymphomas who experience disease progression after CAR T-cell therapy and have a poor prognosis."

About CAR T-Cell Therapy Engager (CTE) ALETA-001

Aleta’s lead clinical stage program, ALETA-001, was designed specifically for the treatment of B-cell malignancies in patients who have received a CD19-directed CAR T-cell therapy and are at risk of treatment failure. Developed to improve the effectiveness of CD19-directed CAR T-cell therapies by increasing CD19 antigen density and restoring lost CD19 expression on the cancer cell, ALETA-001 contains the CD19 target protein which is further linked to an CD20 antibody domain. This allows CD19+/CD20+ cancer cells to be easily recognized and killed by CD19-directed CAR T-cells that were previously administered and are already circulating within a patient.

Aleta previously secured landmark clinical support and funding from Cancer Research UK for the ALETA-001 Phase 1/2 clinical trials, and ALETA-001 has received a UK Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). ILAP designation is granted to medicines that address life-threatening or seriously debilitating conditions, and where there exists a significant patient or public health need.

Oncoinvent to Present at Evercore ISI 2024 Emerging Biotech Conference

On February 21, 2024 Oncoinvent AS, a clinical stage company advancing alpha emitter therapy across a variety of solid cancers, reported that members of management will present at the Evercore ISI 2024 Emerging Biotech Conference on Wednesday, February 28, 2024 at 11:20 a.m. ET (Press release, Oncoinvent, FEB 21, 2024, View Source [SID1234640352]).

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Immune-Onc Therapeutics Announces Orphan Drug Designation Granted by US FDA for IO-202 (Anti-LILRB4) for the Treatment of Chronic Myelomonocytic Leukemia (CMML)

On February 21, 2024 Immune-Onc Therapeutics, Inc. ("Immune-Onc"), a clinical-stage biopharmaceutical company advancing novel therapies in immunology and oncology by targeting myeloid cell inhibitory receptors, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IO-202 for the treatment of chronic myelomonocytic leukemia (CMML) (Press release, Immune-Onc Therapeutics, FEB 21, 2024, View Source [SID1234640351]). IO-202 received Fast Track Designation for treatment of relapsed or refractory CMML in 2023. In addition, Fast Track and Orphan Drug Designations for IO-202 were granted by the FDA for the treatment of acute myeloid leukemia (AML) in 2022 and 2020, respectively.

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IO-202 is a first-in-class antagonist antibody with specific, high affinity binding to Leukocyte Immunoglobulin-Like Receptor subfamily B4 (LILRB4) and serves as a targeted therapy with broad potential in blood cancers, autoimmune and inflammatory diseases. IO-202 in combination with azacitidine (AZA) is currently in a Phase 1 dose expansion clinical trial (NCT04372433), enrolling newly diagnosed patients with CMML who have not received any hypomethylating agents (HMA). A dose escalation trial of IO-202 has been completed, showing clinical efficacy in relapsed or refractory AML and CMML, either as a monotherapy or in combination with AZA. IO-202 has been shown to be well tolerated in all patients treated to date.

"Although current therapeutic options for CMML can improve a patient’s quality of life, there is a high unmet need for effective disease-modifying approaches that are potentially curative," said Charlene Liao Ph.D., chief executive officer and board chair of Immune-Onc. "We are very proud that the FDA has granted IO-202 Orphan Drug Designation for the treatment of CMML. We look forward to continued collaborations with our investigators and the FDA as we work to bring this potentially important therapy to patients with hard-to-treat blood cancers."

The FDA grants Orphan Drug Designation to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation qualifies the sponsor for various development incentives of the Orphan Drug Act, including exemption of FDA application fees and tax credits for qualified clinical testing, and conveys seven years of marketing exclusivity for a drug approved to treat an orphan disease in the United States.

ABOUT CHRONIC MYELOMONOCYTIC LEUKEMIA (CMML)

CMML is a rare form of blood cancer, occurring in 4 of every 1,000,000 people in the United States each year, or about 1,100 annual cases1. CMML is characterized by the presence of a high monocyte count (>1×109/L peripheral monocytes with monocytes ≥ 10% of white blood count) along with dysplastic features in the bone marrow2. Current FDA approved therapies for CMML are all hypomethylating agents, including azacitidine, that attempt to control CMML without enhancement to overall survival.

ABOUT LILRB4 (also known as ILT3)

LILRB4, also known as ILT3, is an immune-modulatory transmembrane protein found on monocytes and monocyte-derived cells. LILRB4 is expressed on certain hematologic cancer cells, such as myelomonocytic leukemia blasts, and on certain pathogenic cells involved in autoimmunity and inflammatory processes.

About IO-202

IO-202 is a first-in-class antagonist antibody with specific, high affinity binding to LILRB4. IO-202 is a humanized IgG1 antibody with Fc effector function to kill LILRB4hi cells via antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). As such, IO-202 is a targeted therapy with broad potential in both blood cancers and autoimmune and inflammatory diseases.

IO-202 has completed the dose escalation part of the first-in-human, multicenter, open-label Phase 1 study in the U.S., and the data was presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in 2023. This Phase 1 trial has advanced to the dose expansion stage to evaluate IO-202 in combination with azacitidine (NCT04372433) in patients with newly diagnosed chronic myelomonocytic leukemia (CMML) who have not received any hypomethylating agents (HMA).

The U.S. Food and Drug Administration granted IO-202 Fast Track Designations for treatment for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) and for the treatment of patients with relapsed or refractory chronic myelomonocytic leukemia (CMML). The FDA has also granted Orphan Drug Designations to IO-202 for the treatment of AML and for the treatment of CMML.

Intensity Therapeutics to Present at the 2024 BIO CEO & Investor Conference

On February 21, 2024 Intensity Therapeutics, Inc. (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported that Lewis H. Bender, President and Chief Executive Officer, will present a company overview at the 2024 BIO CEO & Investor Conference on Monday, February 26, 2024, at the New York Marriott Marquis at 11:45am ET in the Uris Room (Press release, Intensity Therapeutics, FEB 21, 2024, View Source;investor-conference-302066806.html [SID1234640350]).

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Mr. Bender will also host in-person one-on-one meetings during the event. For more information about the 2024 BIO CEO & Investor Conference, please visit View Source