Highlights & Summary
Intro
August was a busy month for the next-generation therapeutics field, full of new partnership announcements and pipeline updates. Other key developments include a number of clinical trial and regulatory updates.
Cell Therapy
- BMS and BeiGene End Their China Licensing Deal | Partnership
- Kyverna Therapeutics Raises $145 Million in its Series B, Bringing New Investors on Board | Financing
- Celularity Announces Collaboration With Regeneron | Partnership
Gene Therapy
- Taysha Reports Early Positive Results From TSHA-102 Study | Clinical Trial
Oligo Therapy
- Amgen Presents Phase 2 Data on Olpasiron | Clinical Trial
- Amber Bio Raises $26M in Seed Funding | Financing
- Biden Admin Awards $24M for mRNA Research | Financing
Antibody-Drug Conjugates
- Vincerx Pacing Investment in VIP924 | Pipeline
- Avidity Biosciences Receives Orphan Drug Designation for AOC 1044 for The Treatment of DMD | Regulatory
Cell Therapy
FDA Lifts Partial Hold on Gilead’s and Arcellx’ CART-ddBCMA for Multiple Myeloma | Regulatory
The phase 2 trial had been put on a partial hold in June of this year after reports of a patient death emerged. Arcellx claims that the death was due to limited bridging therapies available (treatments given in between the T cells being removed from a patient’s body and the CAR-T being infused), which has now been remedied by the FDA signing off on several bridging therapies. This is good news for both Arcellx and Gilead’s Kite pharma who invested $225M in upfront cash and $100M in Arcellx for the the rights to co-develop and co-commercialize the therapy.
BMS and BeiGene End Their China Licensing Deal | Partnership
BMS and BeiGene decided to end a deal, signed in 2017, that allowed BeiGene to sell BMS’ Abraxane in China. In 2020, after manufacturing issues emerged with BMS' contractor Fresenius Kabi’s facility in Phoenix, Chinese authorities decided to put Abraxane on an import and sale ban. This prompted legal action from BeiGene against BMS and put a notable strain on the relationship, which led both sides to settle by ending the entire legacy Celgene deal, effective at the end of 2023.
Kyverna Therapeutics Raises $145 Million in its Series B, Bringing New Investors on Board | Financing
Kyverna is one of the first drug developers to repurpose CAR-Ts from oncology to treat autoimmune disease. This approach is clearly resonating with investors as Kyverna’s Series B got oversubscribed, raising $145M well-ahead of the targeted $60M. In addition to that, the pool of investors in Kyverna has increased with Bain Capital Life Sciences and GordonMD Global Investments joining existing investors.
Celularity Announces Collaboration With Regeneron | Partnership
Celularity announced a new collaboration with Regeneron, where under the deal Celularity will provide research support to Regeneron’s CAR-T program for solid tumors. The research collaboration will take place at Celularity’s facility in Florham Park, N.J. Financial terms were not disclosed.
BMS to Evaluate Cellares’ Automated Manufacturing of CAR-T Cell Therapy Platform | Partnership
Cellares is dedicated to cell therapy manufacturing with one of its key offerings being an automated platform, the Cell Shuttle. BMS has shown interest in this approach, joining Cellares’ Technology Adoption Partnership (TAP) program. Through this agreement BMS will enter into a proof-of-concept transfer process for the manufacture of one of its CAR-T cell therapies.
Gene Therapy
Regeneron Acquires Decibel Therapeutics | M&A
Regeneron has agreed to acquire its longtime partner Decibel to further expand its work in hearing loss and balance-related issues. DB-OTO, the most advanced asset, is a dual-vector treatment presently in a Phase 1/2 trial in patients with hearing loss related to mutations in the OTOF gene. Regeneron is acquiring the company for $4.00 / share with a CVR of $3.50 based on DB-OTO hitting certain milestones.
Taysha Reports Early Positive Results From TSHA-102 Study | Clinical Trial
Taysha announced early positive results from their ongoing study of TSHA-102 ex-U.S. as well as clearance by FDA of their IND for pediatric patients with Rett syndrome. Initial patients dosed showed no serious adverse events and efficacy improvements at four weeks post treatment. Leveraging AAV9, the therapy is administered intrathecally. Patients showed no seizure activity 5-weeks out from dosing.
Voyager Adds Program Targeting Amyloid Beta Plaques | Pipeline
Voyager announced a new gene therapy program targeting amyloid beta plaques. This adds to the company’s ongoing programs in the Alzheimer’s and dementia space. The company has previously been working on an antisense program targeting tau protein, as well as an antibody-based program also targeting tau. Leveraging their TRACER capsids, the gene therapy would deliver a vectorized antibody targeting amyloid beta.
Team Develops AAV to Transduce Kidney for Nephrotic Syndrome | Pipeline
An academic team from the University of Bristol published a study in Science Translational Medicine on a novel capsid for transducing kidney cells. The team identified the AAV-LK03 serotype as efficiently transducing kidney cells and used this to treat an animal model of genetic nephrotic syndrome due to mutations in the NPHS2 gene.
Quest Receives FDA Breakthrough for CDx for Sarepta’s ELEVIDYS | Partnership
Quest Diagnostics announced that their ELISA test for antibodies against AAVrh74 capsid received a breakthrough device designation from the FDA. Designed as a companion diagnostic for Sarepta’s DMD therapy, the test will help screen those patients who may be amenable for the therapy. This is part of a larger agreement that will see Quest potentially develop additional diagnostic tests for a range of Sarepta’s programs and products.
Oligo
Amgen Presents Phase 2 Data on Olpasiron | Clinical Trial
Amgen announced data from the Phase 2 Ocean(a)-Dose study of olpasiran, an siRNA therapeutic at the European Society of Cardiology (ESC). Olpasiran showed promising results and reduced Lp(a) levels over the course of a year. Elevated Lp(a) levels are associated with an increased risk of cardiovascular disease. Olpasiran also reduced OxPL-ApoB, a pro-atherogenic oxidized phospholipid, which is strongly associated with atherosclerosis.
Amber Bio Raises $26M in Seed Funding | Financing
Amber Bio, a novel RNA editing startup, recently announced $26M in seed funding. The round was co-led by Playground Global and Andreessen Horowitz Bio + Health. Participants include Elil Lily and Hummingbird Ventures, among others. Amber Bio was founded by Broad Institute and UC Berkeley researchers with an effort to treat genetic diseases with high allelic diversity.
Biden Admin Awards $24M for mRNA Research | Financing
The Biden Administration and White House awarded a team of biomedical engineers at Georgia Tech and Emory $24M to support mRNA research for cancer and other chronic diseases. The project is called Curing the Uncurable via RNA-Encoded Immunogene Tuning (CUREIT) and focuses on developing mRNA therapeutics. This funding comes from the Advanced Research Projects Agency for Health, a federal agency that is working to support novel health research.
Ionis Enters Collaboration with Novartis to Develop Cardiovascular Disease Therapies | Partnership
Ionis and Novartis further invest in their collaborative partnership for pelacarsen, a next generation program that targets Lp(a). Ionis will receive $60M upfront from Novartis, and Novartis will take on development and manufacturing for the commercialization of this therapy which is currently being assessed in a Phase 3 cardiovascular clinical study.
Agios and Alnylam Announced Agreement for siRNA Therapeutic | Partnership
Agios and Alnylam Pharmaceuticals have entered a global license agreement. Agios will acquire rights to commercialize TMPRSS6, a siRNA therapeutic as a potential treatment for patients with polycythemia vera. Early data has demonstrated that TMPRSS6 has low off-target activity and a 90% knockdown of TMPRSS6 mRNA over 3 months in non-human studies. Agios will make a payment of $17.5M to Alnylam for the global license.
ADCs
Abbvie Stops Development of 2 Preclinical ADCs | Pipeline
AbbVie removed two early-stage ADCs from its pipeline, ABBV-011 and ABBV-647. Both assets were in Phase I trials for the treatment of lung cancer. ABBV-011 targeted SEZ6 protein and ABBV-647 targeted the PTK7 protein. This decision contrasts with the industry's growing interest in ADC therapeutics and the pipeline change diverges from trends such as Pfizer's significant investment in the ADC space.
Bio-Thera Solutions Announces Positive Phase 1 Clinical Data for BAT8006 | Clinical Trial
The company unveiled positive Phase 1 clinical outcomes for its anti-FRα, BAT8006, showcasing a 31% overall objective response rate (ORR) and an 86% disease control rate (DCR) among patients with diverse advanced solid tumors. The study observed manageable safety and plans now involve a Phase 1b study and expanding BAT8006's clinical program worldwide. Earlier this year ImmunoGen received approval for ELAHERE in ovarian cancer and there are currently around 13 anti-FRα ADCs in development worldwide.
DualityBio Expands Global Strategic Partnership with BioNTech | Partnership
DualityBio has announced an extended partnership with BioNTech to accelerate the development of a third ADC called DB-1305 for the treatment of solid tumors. DB-1305 is currently in a Phase 1/2 clinical trial for solid tumors and is based on DualityBio's proprietary Duality Immune Toxin Antibody Conjugates (DITAC) platform. The agreement includes upfront payments, milestone payments, and royalties for DualityBio.
Vincerx Pacing Investment in VIP924 | Pipeline
The company has deprioritized VIP924, a CXCR5-KSPi ADC targeting B-cell malignancies, while focusing on lead programs. Vincerx aims to optimize resources for its next-generation ADC technology, including VIP943 (Phase 1 in Q4 2023) and VIP236 (Phase 1 ongoing), which target leukemias and solid tumors, respectively. This strategic shift extends the company’s cash runway into late 2024.
Avidity Biosciences Receives Orphan Drug Designation for AOC 1044, for The Treatment of DMD | Regulatory
Avidity Biosciences' AOC 1044 gains FDA Orphan Drug status for Duchenne muscular dystrophy (DMD) treatment in patients with exon 44 skipping mutations. Treatment enhances functional dystrophin production and is being assessed in the Phase 1/2 EXPLORE44 trial. Avidity plans to share trial results in late 2023. DeciBio’s TheraTrack estimates that there are 5 ADCs being developed for the treatment of DMD.
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