Highlights & Summary
Intro
September was a busy month for the next-generation therapeutics field, full of new clinical trial updates and partnership announcements. Other key developments include a number of regulatory updates.
Cell Therapy
- Beam Therapeutics Announces First Patient Dosed for T-ALL/T-LL | Clinical Trial
- Seraxis Announces Closing of Second VC Tranche and Successful Completion of Preclinical Studies | Financing
- Atara Biotherapeutics Announces Plans to Submit Tab-cel BLA in Q2 2024 Following FDA Agreement on Comparability | Regulatory
- NexImmune Announces Workforce Reduction to Enable Continued AIMTM Nanoparticle Platform Development Strategy | Personnel
Gene Therapy
- All-In Summit Hosts Siren Biotechnology CEO for Talk | Company Creation
Oligo Therapy
- Launch of Phase 1 Trial for Replicate Bioscience's srRNA Vaccine | Clinical Trial
- Moderna Has Positive Phase 3 Results for Flu Vaccine | Clinical Trial
Antibody-Drug Conjugates
- Vincerx Pharma Doses First Patient with VIP943 | Clinical Trial
- Samsung Biologics Joins Forces with AimedBio | Partnership
- Nurix Announces Strategic Collaboration with Seagen | Partnership
Cell Therapy
Beam Therapeutics Announces First Patient Dosed for T-ALL/T-LL | Clinical Trial
Beam Therapeutics treats the first patient with BEAM-201, a quad-edited CAR-T cell therapy, in patients with relapsed/refractory T-ALL/T-LL. The ongoing Phase 1/2 trial of BEAM-201 is a multicenter, open-label study evaluating safety and efficacy in patients. This represents the first instance of a patient in the U.S. receiving a base editing therapeutic.
Acepodia Announces Nobel Laureate Carolyn Bertozzi, Ph.D., as Chief Scientific Advisor | Personnel
Biotech firm Acepodia has appointed Nobel Laureate Dr. Carolyn Bertozzi as Chief Scientific Advisor. Dr. Bertozzi's click chemistry research is foundational to Acepodia's Antibody-Cell Conjugation (ACC) platform. She was awarded the 2022 Nobel Prize in Chemistry for her innovative approach in bioorthogonal chemistry. As part of her role, she will lead Acepodia's Scientific Advisory Board.
Seraxis Announces Closing of Second VC Tranche and Successful Completion of Preclinical Studies | Financing
Seraxis has closed the second segment of its initial VC round, raising over $50 million in equity. This round saw contributions from Frazier Life Sciences, Polaris Partners, Eli Lilly, and the JDRF T1D Fund, following the achievement of preclinical milestones for Seraxis’ pancreatic organoids, SR-02. Seraxis produces pancreatic organoids using a stem cell line from a human pancreatic islet. As SR-02 approaches IND filing, Ted Hibben, a seasoned business development expert, has joined the company as Chief Corporate Development Officer to steer commercial endeavors in diabetes therapy.
Atara Biotherapeutics Announces Plans to Submit Tab-cel BLA in Q2 2024 Following FDA Agreement on Comparability | Regulatory
Atara Biotherapeutics announced progress in the FDA regulatory pathway for tab-cel. Both parties have agreed on analytical comparability between manufacturing versions, permitting data pooling from different processes for the BLA. Atara plans to submit the tab-cel BLA in Q2 2024, incorporating recent trial data from the ALLELE study. Tabelecleucel, an EBV-specific T-cell immunotherapy, treats EBV+ PTLD, a rare and severe malignancy post-transplant. In Europe, tabelecleucel is marketed as EBVALLO.
NexImmune Announces Workforce Reduction to Enable Continued AIMTM Nanoparticle Platform Development Strategy | Personnel
NexImmune, cut its workforce by about 53% on September 5, 2023. This decision by the Board and management aims to lower expenses and prolong the company's financial runway. Despite this reduction, NexImmune CEO, Kristi Jones, highlighted their progress in pre-clinical data and the value of their nanoparticle technology in directing T cell function. As part of these changes, CFO John Trainer also left the company on September 2, 2023, but will continue to offer advisory services through a consulting agreement.
Gene Therapy
Astellas Declines Option to Taysha’s GAN Program | Pipeline
Astellas, who previously paid $20M upfront and invested $30M in Taysha to secure options to two programs, has declined to exercise the option for TSHA-120, an AAV9 treatment for giant axonal neuropathy (GAN). This comes after Astellas saw the meeting minutes with regulators post Phase 2 completion. Regulators were skeptical of endpoint design and other features of the proposed pivotal trial. Given a complicated path forward, Taysha is deprioritizing the program to focus exclusively on TSHA-102, their program for Rett Syndrome.
Otsuka Pharmaceutical Co. Partners with ShapeTX on Eye Programs | Partnership
In a collaboration valued at over $1.5 billion, Otsuka Pharmaceutical and ShapeTX are partnering to develop intravitreally-delivered adeno-associated viruses (AAVs) for treating ocular diseases. ShapeTX's AI-driven AAVid capsid discovery platform will optimize AAV variants for precise targeting, attempting to reduce clinical safety risks. Shape had previously established a partnership with Roche.
Catapult Creates Conglomerate to Improve AAV Analytical Technologies | Manufacturing
Catapult (CGT Catapult) is partnering with Rentschler Biopharma and Refeyn Ltd. for a two-year project aimed at improving adeno-associated virus (AAV) manufacturing. The collaboration will focus on developing automated AAV gene therapy manufacturing platforms and integrating it into Rentschler Biopharma's existing process. This will be in an attempt to address high-throughput sampling and analysis of production lots. The project is supported by Innovate UK's grant for digitalization and automation in medicine R&D and manufacturing.
All-In Summit Hosts Siren Biotechnology CEO for Talk | Company Creation
The 2023 All-In Summit hosted former UCSF Professor and Siren Biotechnology CEO, Dr. Nicole Paulk, during their 3-day conference. Siren officially came out of stealth earlier this year with the goal of using AAV to deliver immune-modulating factors to tumors in an effort to make them more susceptible to anti-cancer medicines. The company’s core technology comes from the lab that Dr. Paulk led at UCSF.
Oligo
Ginkgo Bioworks and Pfizer Partner for RNA Therapy Discovery | Partnership
Ginkgo Bioworks has partnered with Pfizer to discover RNA-based drug candidates. Pfizer will utilize Ginkgo's specialized RNA technology to progress the exploration and development of innovative RNA molecules in key research areas. Ginkgo stands to receive an initial payment and may also be eligible for milestone payments totaling up to $331 million across these programs.
Launch of Phase 1 Trial for Replicate Bioscience's srRNA Vaccine | Clinical Trial
Replicate Bioscience has commenced a Phase 1 trial for its RBI-4000 vaccine, a pioneering rabies prevention solution utilizing self-replicating RNA (srRNA) technology. This is the first human subject dosed with this next-generation technology. CEO Nathaniel Wang, Ph.D., believes this approach as valuable for also addressing other intricate infectious diseases, oncology, and autoimmune conditions.
Moderna Has Positive Phase 3 Results for Flu Vaccine | Clinical Trial
Moderna recently unveiled promising results from its Phase 3 trial of the flu vaccine, mRNA-1010. This vaccine exhibited superior HAI titers compared to Fluzone HD. The company also provided updates on the vaccine pipelines, including plans for a Phase 3 study of neoantigen therapy in conjunction with Keytruda® for non-small cell lung cancer. Moderna is poised to launch up to 15 products in the next few years, potentially introducing new indications for mRNA therapies.
Khosla Ventures Leads Seed Funding for mRNA Biotech | Financing
Rejuvenation Technologies, a small biotech startup, has raised $10.6 million in a seed round led by Khosla Ventures. Rejuvenation's approach involves employing mRNA within lipid nanoparticles to instruct cells to produce a protein, thereby elongating telomeres. Their primary drug candidates are tailored for treating diseases such as idiopathic pulmonary fibrosis.
ADCs
Vincerx Pharma Doses First Patient with VIP943 | Clinical Trial
The company has administered the first patient in its Phase 1 clinical trial for VIP943, targeting relapsed/refractory AML, MDS, and B-cell ALL. VIP943 is an innovative ADC candidate with a KSP inhibitor payload, designed to enhance efficacy and reduce toxicity in CD123+ hematologic malignancies, with preliminary safety data expected in 2024. According to DeciBio’s TheraTrack there are ~4 unique anti-CD123 ADC assets in development for the treatment of hematologic tumors.
Samsung Biologics Joins Forces with AimedBio | Partnership
Samsung Biologics has made an equity investment in AimedBio, a Korean biotech company specializing in antibody-drug conjugate (ADC) technology for severe neurological diseases. This investment includes a research collaboration to develop ADC technologies and a contract development agreement for AimedBio’s monoclonal antibody therapy. Samsung Biologics is expanding into ADCs and is constructing a dedicated ADC manufacturing facility, aiming for completion in 2024.
Nurix Announces Strategic Collaboration with Seagen | Partnership
The two companies have partnered to develop Degrader-Antibody Conjugates, with Nurix receiving a $60 million upfront payment and potential milestone payments of up to approximately $3.4 billion. The collaboration aims to combine targeted protein degradation and antibody-drug conjugation technologies to create innovative cancer therapies.
AstraZeneca and Daiichi Sankyo Plans for DS-1062 Following Phase III Success | Clinical Trial
AstraZeneca and Daiichi Sankyo's Dato-DXd (DS-1062) demonstrated improved progression-free survival (PFS) in a Phase III trial for advanced breast cancer. While the overall survival (OS) data isn't mature yet, Dato-DXd may compete with Gilead's Trodelvy, which received FDA approval for a similar indication. According to DeciBio’s TheraTrack there are ~30 anti-TROP2 ADCs in development across a variety of solid indications.
Mythic Therapeutics Received Fast Track Designation for MYTX-011 in NSCLC | Regulatory
The company received Fast Track designation for MYTX-011, its cMET-targeting ADC for the treatment of NSCLC. This designation is significant as there are currently no approved cancer therapies for this patient group. MYTX-011, currently in phase 1, utilizes Mythic's FateControl™ technology to enhance ADC uptake within cells. TheraTrack estimates that there are currently ~11 unique anti-cMet ADCs in preclinical and clinical development.
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