Highlights & Summary
Intro
May was a busy month for the next-generation therapeutics field, full of clinical updates, partnerships news and deals. Other key announcements include a number of company creations and regulatory updates.
Happy reading!
Cell Therapy
- Amgen and TScan Therapeutics Announce Collaboration to Identify Novel Targets in Crohn’s Disease | Partnership
- BMS’ Breyanzi Meets Primary Endpoint of Overall Response Rate in TRANSCEND FL and TRANSCEND NHL 001 Studies | Clinical Trial
- NYBC Ventures Launches $50M Fund to Accelerate Innovations in Blood and Cellular Therapies | Company Creation
- BMS Exercised its First Option, yielding $15M for Immatics, as a Part of an Ongoing Multi-target Strategic Collaboration | Partnership
Gene Therapy
- Siren Biotechnology Unveils to Develop AAV for Cancers | Company Creation
- Taysha Begins Clinical Trial for Rett Syndrome Candidate | Clinical Trial
- Rocket Pharmaceuticals Gets IND Clearance for Cardiomyopathy Therapy | Clinical Trial
- Forge and LabCorp Partner on AAV Manufacturing | Manufacturing
Oligo Therapy
- ReNAgade Therapeutics Raises $300M in Series A | Financing
- Rona Therapeutics and KeyMed Biosciences Announce Collaboration to Develop siRNA Therapeutics | Partnership
- Moderna Reports Phase 1 / 2 Interim Data on MRNA-3927 for Propionic Acidemia at ASGCT | Clinical Trial
Antibody-Drug Conjugates
Cell Therapy
Amgen and TScan Therapeutics Announce Collaboration to Identify Novel Targets in Crohn’s Disease | Partnership
This new collaboration is focused on identifying new targets in Crohn’s disease using TScan’s proprietary target discovery platform, TargetScan. TScan will receive an upfront payment of $30 million and can potentially earn over $500 million in milestone and royalty payments. Amgen will have exclusive rights to develop and commercialize therapeutics based on the targets discovered, with an option to expand the collaboration to ulcerative colitis.
BMS’ Breyanzi Meets Primary Endpoint of Overall Response Rate in TRANSCEND FL and TRANSCEND NHL 001 Studies | Clinical Trial
Both trials had positive results, showing that both primary and key secondary endpoints have been met so far. TRANSCEND FL (a phase 2 single arm) and TRANSCEND NHL (a phase 1 single arm) both met the primary ORR endpoint and the secondary CRR (complete response rate) endpoint, showing high CRRs in both relapsed or refractory FL and MCL. These results add to Breyanzi’s track record of showing clinically meaningful benefit in B-cell malignancies.
NYBC Ventures Launches $50M Fund to Accelerate Innovations in Blood and Cellular Therapies | Company Creation
The inaugural fund was launched with a $50 million investment from New York Blood Center, Inc. (NYBC), with a main focus of investing in early-stage life sciences companies that focus on therapeutics, devices, and innovative technologies that accelerate innovation in blood-related fields, and cellular therapies and enabling technologies. This is one of the first venture funds that is focused solely on innovation in blood and cell-based therapies and technologies.
BMS Exercised its First Option, yielding $15M for Immatics, as a Part of an Ongoing Multi-target Strategic Collaboration | Partnership
BMS exercised its first option for the most advanced TCR-T product candidate from the company’s ongoing collaboration with Immatics to develop four TCR-based adoptive cell therapies targeting solid tumors. From this exercise, Immatics will receive an option payment of $15 million. Immatics stands to receive up to $490 million in additional payments if the product hits further milestones, on top of any tiered royalties on potential net sales of the product.
Cellectis Finishes Q1 2023 with $3.6M in Revenue Earned and Several Pipeline Updates | Financing
The company finished its first r/r ALL patient in Europe with its UCART22 asset, manufactured in-house. Cellectis also implemented CLLS52 for the first time in the clinic with Sanofi’s alemtuzumab and stopped enrollment and treatment of patients in the MELANI-01 clinical trial evaluating UCARTCS1 product candidate in r/r MM. The company’s key focus now will be on its pipeline of allogeneic CAR T-cell product candidates UCART22, UCART20x22 and UCART123.
Gene Therapy
Sarepta Receives Positive AdCom Vote for DMD Candidate | Regulatory
In a surprise twist, Sarepta received a positive vote from FDA advisors on SRP-9001, their AAV9 candidate for treating Duchenne Muscular Dystrophy (DMD). Approval would mark the first viral gene therapy for DMD, with Pfizer’s Ph3 asset close behind. Roche previously partnered with Sarepta, grabbing ex-U.S. rights in a deal valued > $1B.
Siren Biotechnology Unveils to Develop AAV for Cancers | Company Creation
Backed by Founders Fund, Lux Capital, and others, Siren Bio came out of stealth at this year’s ASGCT meeting with the goal of developing AAV therapies for cancer. The company aims to develop “universal”, pan-cancer therapies that would make tumors generate immune-stimulating cytokines. The technology originated in the UCSF lab of Dr. Nicole Paulk, who has left the institution to lead Siren.
Taysha Begins Clinical Trial for Rett Syndrome Candidate | Clinical Trial
Taysha announced it has dosed the first patient in its Phase 1/2 study of TSHA-102. This is one of two therapies that Taysha previously strategically focused the company around. Partnered with Astellas, the therapy leverages AAV9 to deliver a miniature version of the MECP2 gene.
Rocket Pharmaceuticals Gets IND Clearance for Cardiomyopathy Therapy | Clinical Trial
Rocket is cleared to start their clinical trial for RP-A601. Prior studies in mice decreased arrhythmias, improved right ventricular morphology and function, and increased survival. This would be Rocket Pharmaceuticals’ second clinical asset to go along with their program to treat Danon Disease.
Forge and LabCorp Partner on AAV Manufacturing | Manufacturing
Forge and LabCorp are teaming up to expedite drug development and manufacturing services for AAV drug developers. LabCorp brings its AAV drug development expertise and will marry that with Forge’s manufacturing capacity and know-how. The two groups hope to create a more end-to-end contract research and manufacturing organization to streamline AAV development.
Oligo
ReNAgade Therapeutics Raises $300M in Series A | Financing
ReNAgade has recently announced $300M in Series A led by MPM BioImpact and F2 Ventures. ReNAgade’s platform aims to address limitations for delivery of RNA-based therapies to a wide range of tissues and cells that were previously challenging to access. In addition, the company has also established a joint venture with Orna Therapeutics Inc., and has an ongoing collaboration with Merck.
Rona Therapeutics and Keymed Biosciences Announce Collaboration to Develop siRNA Therapeutics | Partnership
Rona Therapeutics, Inc. and Keymed Biosciences announced a partnership to develop siRNA therapeutics for Glomerulonephritis. The collaboration will use the RAZOR platform and leverage the clinical and scientific expertise from Keymed to identify siRNA compounds and will start clinical work in 2024.
Moderna Reports Phase 1 / 2 Interim Data on MRNA-3927 for Propionic Acidemia at ASGCT | Clinical Trial
At the 2023 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting, Moderna reported their interim data from the Phase 1 / 2 trial of mRNA-3927. This therapy is being investigated for propionic acidemia, a rare inherited condition where the body cannot efficiently break down fats and protein. Moderna is approaching the dose-expansion phase. To date, there have been no drug-related adverse events or occurrence of dose-limiting toxicities.
Eterna Therapeutics Acquires Platform from Exacis Biotherapeutics | M&A
Eterna Therapeutics has recently announced their acquisition of Exacis Biotherapeutics’ immuno-oncology platform. Exacis’ platform uses mRNA targeted approaches and gene editing to engineer IPSC-derived cells for cancer therapies.
ADCs
Lonza to Acquire Synaffix and Strengthen ADC Offering | M&A
This acquisition strengthens Lonza's bioconjugates offering by integrating Synaffix's proprietary technology platforms, including payload and linker technologies (GlycoConnectTM, HydraSpaceTM and toxSYNTM), and expands their capabilities in developing and commercializing novel ADCs. Synaffix currently partners with 13 different companies on ADC assets
Sony and Astellas Enter into Collaborative Agreement to Discover a Novel ADC Platform | Partnership
This agreement focuses on discovering a novel ADC platform, focusing on Sony's unique polymeric material known as "KIRAVIA™ Backbone." This material offers design flexibility, high stability, and efficient drug delivery to cells. The goal is to optimize the ADC platform by achieving a high Drug-to-Antibody Ratio (DAR), enhancing therapeutic efficacy, and minimizing side effects
ADC Therapeutics Terminates Preclinical Programs | Pipeline
ADC Therapeutics has chosen to discontinue its investment in two preclinical programs, ADCT-212 targeting PSMA and ADCT-701 targeting DLK-1. The company's decision is part of a strategic shift to prioritize programs with more immediate clinical impact and higher potential for generating value. By reallocating resources accordingly, ADC Therapeutics aims to accelerate and expand its advanced programs
Pyxis Oncology to Acquire Apexigen | M&A
By integrating Apexigen's commercially and clinically validated antibody generation platform with Pyxis Oncology's existing ADC toolkit, the company gains a comprehensive system for designing and producing novel ADC candidates with improved potency, stability, and tolerability enabling targeting of difficult-to-treat cancers. This will solidify Pyxis Oncology’s position as a frontrunner in ADC innovation
Convergent Therapeutics Raises $90M to Advance its Radioantibodies Pipeline | Financing
The company has raised a $90 million Series A to support the development of next-generation radioantibodies. Its main asset CONV01-α, targets PSMA and delivers actinium-225 to prostate cancer. The company also plans to to use the funding to explore additional radioantibody targets and potential pipeline acquisitions
FDA Issues a Complete Response LEtter for Byondi’s SYD985 | Regulatory
The FDA has issued a CRL for Byondis' lead asset SYD985, an anti-HER2 ADC for the treatment of metastatic breast cancer. The letter indicates that the FDA has temporarily suspended the decision on the product's approval, requesting additional information that extends beyond the current evaluation period. Byondis has at least 2 additional ADC assets in development, BYON3521 targeting c-Met and BYON4413 targeting CD123
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