On June 21, 2023 Aldeyra, a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated diseases, reported receipt of a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for the 505(b)(2) New Drug Application (NDA) of ADX-2191 (methotrexate for injection, USP), an investigational drug candidate, for the treatment of primary vitreoretinal lymphoma (PVRL) (Press release, Aldeyra Therapeutics, JUN 21, 2023, View Source [SID1234632816]). Although no safety or manufacturing issues with ADX-2191 were identified, the FDA stated that there was a "lack of substantial evidence of effectiveness" due to "a lack of adequate and well-controlled investigations" in the literature-based NDA submission. Based on prior discussions with the FDA, Aldeyra did not conduct any clinical trials of ADX-2191 in PVRL.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"While we appreciate the FDA’s position with respect to providing evidence from adequate and controlled trials, we do not currently believe that randomized clinical trials of ADX-2191 in PVRL, a rare and fatal cancer with no approved therapy, are feasible," stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra Therapeutics. "Given the current shortage of methotrexate, the lack of approved therapy for PVRL, and the desire to avoid potential safety risks associated with ocular injection of compounded formulations, we look forward to discussing with the FDA the potential for making ADX-2191 available to PVRL patients under an Expanded Access Program."

PVRL is a rare, high-grade, aggressive cancer, with a median survival of less than five years.1 Methotrexate, the compounded intravitreal injection of which is the standard of care for the treatment of PVRL,2 is currently in shortage, per the FDA Drug Shortages database. An Expanded Access Program allows for access to treatment options for serious diseases when other therapeutic options are not available. Aldeyra plans to discuss ADX-2191 for the treatment of PVRL with the FDA, including the potential to make ADX-2191 accessible to PVRL patients under an Expanded Access Program protocol.

ADX-2191 is also under development for the treatment of proliferative vitreoretinopathy and retinitis pigmentosa, both of which are rare, sight-threatening retinal diseases. Top-line results from a Phase 2 clinical trial of ADX-2191 in retinitis pigmentosa are expected to be announced in June of 2023. Additionally, Aldeyra plans to conduct a Type C meeting with the FDA in the second half of 2023 to discuss the completion of clinical development of ADX-2191 for the prevention of proliferative vitreoretinopathy.

On June 21, 2023 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), reported that the International Nonproprietary Name (INN) for AMP945 is narmafotinib (Press release, Amplia Therapeutics, JUN 21, 2023, View Source;[email protected] [SID1234632802]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The process for naming a drug compound requires an extensive process of review prior to approval. These reviews take into account similarity with other drug names, ease of pronunciation across multiple languages, language review for accidental meaning, and finally a period for parties to report an objection to the name. With these processes now complete the Company is now free to use the name narmafotinib for AMP945.

Amplia’s CEO and Managing Director, Dr Chris Burns, commented: "Approval of the name narmafotinib is an important step in the commercial development of the drug, signalling to potential partners and collaborators our commitment to the long-term development of this exciting agent.

On June 20, 2023 Alamar Biosciences, a company powering precision proteomics to enable the earliest detection of diseases, and Frazier Life Sciences, reported the launch of Attovia Therapeutics, a newly formed company focused on creating a pipeline of biotherapeutics in immune-mediated disease and cancer (Press release, Attovia Therapeutics, JUN 20, 2023, View Source [SID1234647438]). The company, based on Attobody, Alamar’s novel proprietary biparatopic nanobody platform, concurrently closed a $60 million Series A financing led by Frazier and joined by venBio and Illumina Ventures.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Attovia will receive an exclusive world-wide license to the Attobody platform and associated intellectual property and pipeline assets in the therapeutic field in exchange for equity and potential milestones and royalties. The company plans to use the proceeds from the Series A financing to achieve clinical proof-of-concept on its lead program in immune-mediated disease, nominate additional development candidates across the company’s core areas of focus in immunology and oncology, as well as continue to advance the core Attobody technology platform.

"By creating Attovia with Frazier and other investors, we can give the Attobody platform the dedicated attention and resources it deserves to fully realize its potential," said Yuling Luo, Ph.D., founder, chairman and CEO of Alamar and co-founder of Attovia. "Alamar scientists developed the Attobody platform to improve the affinity and specificity of antibodies and we are very excited to expand its applications to therapeutic development."

Tao Fu, M.S., M.B.A., co-founder of Attovia and Venture Partner at Frazier Life Sciences, will serve as Attovia’s chief executive officer and join the Board. Mr. Fu is a seasoned industry leader with over 25 years of executive experience in the pharmaceutical and biotech industries, including leadership roles at Zai Lab, Portola Pharmaceuticals, Bristol-Myers Squibb, and Johnson & Johnson. Attovia’s leadership team is fully rounded out by Petter Veiby, Ph.D., chief scientific officer; Hangjun Zhan, Ph.D., chief technology officer; and Zaneta Odrowaz, Ph.D., chief business officer.

"The ability to combine two nanobodies into a single biparatopic Attobody creates opportunities to fine-tune therapies for specific targets and to expand the target universe," said Mr. Fu. "I am excited to be partnering with a proven executive team, leading investors and an experienced board to develop medicines for some of the most prevalent disorders that still do not have ideal therapeutic solutions."

Jamie Topper, M.D. Ph.D., managing partner at Frazier Life Sciences and Aaron Royston, M.D., M.B.A., managing partner at venBio, will join the Attovia’s Board of Directors.

"Company creation is one of Frazier’s core strategies, and we are thrilled to partner with Alamar and other investors to launch Attovia," said Dr. Topper. "We believe Attovia is in a strong position to create a robust pipeline of first- and best-in-class drugs derived from the Attobody platform."

"Small format nanobodies have demonstrated recent success in delivering best-in-class efficacy in select immune-mediated disease as evidenced by Acelyrin, one of our early investments," said Dr. Royston. "We are thrilled to join the Attovia investor syndicate and to further advance the increasingly promising nanobody field."

About the Attobody Platform
The Attobody platform generates small format binders (referred to as "Attobodies") with ultra-high affinity, enhanced internalization and fast tissue penetration. These properties make Attobodies ideal binders for hard-to-drug targets such as G-protein-coupled receptors (GPCRs), and enable broad applicability across a number of modalities such as antibody-drug conjugate, radioconjugate or multi-specific biologics development. The biparatopic binding mode of Attobodies, combined with the high-throughput, evolution-driven method of discovering binders, significantly expands druggable epitope and target space. Attobodies do not require affinity maturation and can be engineered into a variety of valencies and half-life extension formats. Thus far, Alamar and Attovia have successfully generated Attobodies against a host of membrane, soluble and viral targets.

On June 20, 2023 Cidara Therapeutics, Inc. (NASDAQ: CDTX), a biotechnology company developing long-acting therapeutics designed to help improve the standard of care for patients facing serious diseases, reported that the company will deliver an oral presentation including highlights from its ongoing preclinical studies of CD421, a CD73-targeting drug-Fc conjugate (DFC), as well as the development of its novel DFCs from Cidara’s Cloudbreak platform, at the 2nd Annual Adenosine-Pathway Targeted Cancer Immunotherapy Summit in Boston, MA, which is being held June 20-22, 2023 (Press release, Cidara Therapeutics, JUN 20, 2023, View Source [SID1234636986]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased to be presenting preclinical data demonstrating the potential of CD421, as well as on the utility and flexibility of our Cloudbreak DFC platform," said Jeffrey Stein, Ph.D. president and chief executive officer of Cidara. "We believe CD421 combines the best attributes of small molecule and monoclonal antibody therapies and can serve as a potentially beneficial alternative treatment option for patients with cancer. We look forward to continuing to advance this candidate towards the clinic."

Oral presentation details are as follows:

Title: Cidara Drug-Fc-Conjugates (DFCs): A new approach to treatment of cancer
Presenter: James Levin, Ph.D., Senior Director, Preclinical Development at Cidara Therapeutics
Session Date/Time: Thursday, June 22, 2023 at 9:30 a.m. ET
Session Location: Boston, MA

To view the full schedule, visit the Adenosine Pathway Targeted Cancer Immunotherapy Summit website here.

About Cloudbreak DFCs
Cidara is developing a new generation of immunotherapeutic agents from its Cloudbreak platform that couple targeted small molecule and peptide drugs to a human antibody fragment (Fc). These highly potent, long-acting drug-Fc conjugates (DFCs) are designed to inhibit specific disease targets while simultaneously engaging the immune system. In addition to multiple oncology programs, Cidara is advancing its antiviral DFC CD388 through Phase 1 and Phase 2a clinical trials in partnership with Janssen for the universal prevention and treatment of influenza.

On June 20, 2023 Minghui Pharmaceutical, Inc., a leading clinical-stage biopharmaceutical company, reported that the first dosing has been completed in two phase 1 clinical studies evaluating MHB036C and MHB088C (Press release, Minghui Pharmaceutical, JUN 20, 2023, View Source [SID1234635431]). The studies aim to determine the maximum tolerated dose (MTD), the recommended phase 2 dose (RP2D), as well as assess the pharmacokinetics and preliminary efficacy of the ADCs in patients with selected types of advanced or metastatic solid tumors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

MHB036C and MHB088C, the two antibody-drug conjugates (ADCs) targeting TROP-2 or B7-H3, respectively, are generated through Minghui’s cutting-edge proprietary SuperTopoiTM ADC platform, which incorporates a highly potent topoisomerase (TOPO) 1 inhibitor linked through a cleavable linker. This novel payload significantly enhances the therapeutic potency of the ADCs, especially against cancer cells with moderate or low tumor-associated antigen expression.

Comprehensive in vitro and in vivo studies across a variety of cancer types demonstrated the exceptional efficacy of MHB036C and MHB088C, exhibiting 3 to 10 times more potent in killing tumor cells compared to their DXd counterparts. Additionally, preclinical GLP tox studies demonstrated an excellent safety profile, with no unique toxicities observed, particularly no severe pulmonary toxicities.

"We are delighted to announce the successful dosing of the first patient in our two ADC programs" stated Guoqing Cao, Ph.D., Chief Executive Officer at Minghui Pharmaceutical. "MHB036C and MHB088C epitomize the tremendous potential of Minghui’s SuperTopoiTM platform. These novel ADCs have undergone extensive research and development, showcasing remarkable efficacy and safety in preclinical studies. MHB036C and MHB088C hold great promise in the fight against various human solid tumors and we look forward to the results from the phase 1 studies, anticipated to conclude in early 2024. "

About MHB036C

MHB036C is an antibody drug conjugate (ADC) composed of a humanized anti-TROP-2 monoclonal antibody conjugated to Minghui’s proprietary DNA topoisomerase I inhibitor via a cleavable linker.

About MHB088C

MHB088C is an antibody drug conjugate (ADC) composed of a humanized anti-B7-H3 monoclonal antibody conjugated to Minghui’s proprietary DNA topoisomerase I inhibitor via a cleavable linker. The antibody has also shown more potent antigen binding and higher endocytosis efficiency.