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Highlights & Summary
Intro
September showcases an abundance of partnership announcements and clinical trial news throughout Next-Generation Therapeutics.
Happy Reading!
Cell Therapy
- Carvykti Reduces Death Risk by 45% in Multiple Myeloma, Setting New Milestone for Cell Therapy | Clinical Trial
- Bristol Myers Squibb and 2seventy bio Halt Abecma Phase 3 Trial Due to Enrollment Challenges | Clinical Trial
- Kite Pharma Sells Stake in Fosun Kite, Grants Full Control of CAR-T Therapies in China | Partnership
Gene Therapy
- Novartis Licenses Capsid from Voyager for $15M Upfront | Partnership
- BridgeBio Halts Development of AAV5 Asset | Clinical Trial
- Tern Therapeutics Soars from Stealth with REGENXBIO-Licensed Assets | Company Creation
Oligo Therapy
- Vico Therapeutics Announces Positive Early Clinical Trials Data for Huntington’s Disease Therapeutic | Clinical Trial
Antibody-Drug Conjugates
- Daiichi Sankyo and Merck’s ADC Demonstrates Progression-Free Survival Benefit in EGFRm NSCLC | Clinical Trial
- Daiichi Sankyo and AstraZeneca’s ADC Fails to Show Overall Survival Benefit in HR+ Breast Cancer | Clinical Trial
- Araris Biotech Expands its IP Portfolio with Acquisition of Innate Pharma’s Transglutaminase Patents | M&A
- Pinotbio Secures Milestone Payment from Celltrion’s Execution of ADC Platform Technology Option | Partnership
Cell Therapy
Kyverna CEO Exists After IPO And Is Replaced by Ex-Kite Leaders | Personnel
Kyverna Therapeutics has reshaped its leadership following CEO Peter Maag's resignation, just seven months after its high-profile IPO. Warner Biddle, former global head of Gilead's Kite Pharma, has taken over as CEO. This leadership change comes as Kyverna’s cell therapy, KYV-101, moves into later-stage development amid challenges in the autoimmune cell therapy space, which has seen oversaturation and declining investor interest. Despite these hurdles, analysts like Leerink Partners’ Thomas Smith view Kyverna’s pipeline as promising. The company had $346.2 million in cash at the end of June.
Carvykti Reduces Death Risk by 45% in Multiple Myeloma, Setting New Milestone for Cell Therapy | Clinical Trial
Johnson & Johnson and Legend Biotech's CAR-T cell therapy, Carvykti, significantly reduced the risk of death by 45% compared to standard care in multiple myeloma patients, marking a milestone in cell therapy for this cancer. After nearly three years, 76.4% of Carvykti patients were alive, versus 63.8% on standard care. These findings, presented at the International Myeloma Society meeting, highlight Carvykti's potential to improve survival, especially when used earlier in treatment. J&J plans to use the data for regulatory updates and commercial efforts.
Bristol Myers Squibb and 2seventy bio Halt Abecma Phase 3 Trial Due to Enrollment Challenges | Clinical Trial
Bristol Myers Squibb and 2seventy bio have halted their Phase 3 KarMMa-9 trial for Abecma as a first-line maintenance treatment in multiple myeloma due to enrollment challenges. The trial, which sought to recruit 618 patients, only reached 10% of its target after a year. The improving efficacy of first-line myeloma treatments has reduced the pool of eligible patients for the trial. This discontinuation further weakens Abecma's competitive position against Johnson & Johnson and Legend Biotech’s rival CAR-T therapy Carvykti, which has seen greater success in both clinical trials and commercial performance.
Kite Pharma Sells Stake in Fosun Kite, Grants Full Control of CAR-T Therapies in China | Partnership
Gilead's Kite Pharma is selling its 50% stake in Fosun Kite Biotechnology to its joint venture partner, Fosun Pharma, for $27 million. This move gives Fosun full control over Yescarta, the first CAR-T therapy approved in China, and Tecartus, which is in clinical trials. Despite the high price of Yescarta, patient access has been facilitated through commercial insurance and outcomes-based payment programs. After the sale, Kite will continue to supply viral vectors and receive royalties from sales in China. The deal follows a trend of foreign pharmaceutical companies divesting local operations to Chinese firms.
Gene Therapy
Novartis Licenses Capsid from Voyager for $15M Upfront | Partnership
Novartis has exercised its option with Voyager Therapeutics, paying $15 million for a novel capsid to use in a rare neurological disease gene therapy program. This move is part of a broader collaboration established in March 2022, where Novartis initially paid $54 million and added another $25 million in 2023 to license two targets. With this latest option, Voyager could receive up to $305 million in milestone payments and tiered royalties.
4DMT Releases Additional Data on 4D-150, Hosts Wet AMD Development Day | Clinical Trial
4D Molecular Therapeutics shared positive interim data from its Phase 1/2 PRISM clinical trial of 4D-150 for wet age-related macular degeneration (AMD), reporting significant reduction in treatment burden and stable vision in patients. In the trial, 4D-150 reduced the need for supplemental injections by up to 98% in recently diagnosed patients. The company is planning to kickoff a Phase 3 trial in early 2025. Other groups with AAV in development leveraging anti-VEGF strategies include REGENXBIO / AbbVie and Adverum.
Neurocrine Biosciences Nominates AAV Candidate with Voyager | Partnership
Voyager Therapeutics and Neurocrine Biosciences have selected a new gene therapy development candidate for an undisclosed neurological disease, leveraging a capsid from Voyager’s TRACER™ platform. This marks the third candidate under their collaboration, which also includes programs for Friedreich’s ataxia and GBA1 Parkinson’s disease. The selection triggered a $3 million milestone payment to Voyager. This partnership, part of a 2023 agreement, covers research and development of AAV gene therapies for neurological conditions.
BridgeBio Halts Development of AAV5 Asset | Clinical Trial
BridgeBio is scaling back its investment in gene therapy after disappointing results from a phase 1/2 trial of BBP-631, its candidate AAV5 for congenital adrenal hyperplasia (CAH). Although the gene therapy showed evidence of transgene activity, it did not meet the company's criteria for continued development. CEO Neil Kumar noted the therapy did not achieve the necessary cortisol levels to surpass current treatment options. The company is paring back its R&D budget and looking for a partner for the asset while focusing its efforts on its therapy for Canavan disease.
Tern Therapeutics Soars from Stealth with REGENXBIO-Licensed Assets | Company Creation
Tern Therapeutics has emerged from stealth with $15 million in funding to revive two gene therapies for Batten disease that were deprioritized by REGENXBIO. The startup, founded by three former REGENXBIO executives, will advance these therapies targeting CLN2, a late-infantile form of Batten disease. Tern’s lead program, TTX-381, is an AAV gene therapy delivered to the retina to address vision loss associated with Batten disease and is currently being tested in a clinical study in London. The second therapy, which delivers a functional copy of the TPP1 gene to the central nervous system, aims to halt neurological degeneration and was previously tested in a single child in Brazil.
Oligo
Korro Bio Announces Novo Nordisk Collaboration for Cardiometabolic Disease Targets | Partnership
Korro Bio has partnered with Novo Nordisk to develop two RNA-based therapeutic candidates targeting cardiometabolic diseases using Korro’s OPERA™ RNA editing platform. The collaboration could result in milestone payments of up to $530 million for Korro, in addition to royalties. Novo Nordisk will leverage its cardiometabolic expertise, while Korro’s technology aims to enable specific RNA edits without altering DNA. This approach could open new possibilities for treating previously undruggable disease targets. The partnership advances Korro’s RNA editing capabilities without impacting its internal pipeline.
Vico Therapeutics Announces Positive Early Clinical Trials Data for Huntington’s Disease Therapeutic | Clinical Trial
Vico Therapeutics' Phase 1/2a trial of VO659, an antisense oligonucleotide therapy for Huntington’s disease (HD), has shown promising interim results in a first clinical trial. In patients with early-stage HD or mild to moderate spinocerebellar ataxia (SCA1 or SCA3), VO659 treatment enabled a 28% reduction in mutant huntingtin protein (mHTT) in cerebrospinal fluid by day 85 post-treatment. This suggests a positive impact on a key biomarker associated with HD progression. Additionally, there was no significant change in neurofilament light (Nf-L) protein levels, indicating no signs of neurodegeneration. Additionally, the therapy was well-tolerated at a 40 mg dose with no major safety concerns, and its long half-life suggests the possibility of infrequent dosing (once or twice a year).
Explorna and Primrose Bio Announce Strategic Partnership to Advance mRNA Medicines | Partnership
Primrose Bio and ExPLoRNA Therapeutics have formed a strategic partnership aimed at advancing mRNA medicines. Primrose Bio holds expertise in engineering high-performance RNA polymerases, while ExPLoRNA is known for its cutting-edge mRNA cap analogs, which are essential for efficient protein production. Together, the two companies aim to co-develop and market innovative RNA polymerases and capping compounds to improve mRNA quality, enhance manufacturing efficiency, and boost therapeutic efficacy. This collaboration is designed to provide mRNA drug developers with better options for creating therapeutics and vaccines with improved safety profiles.
Arrowhead Pharmaceuticals’ Plozasiran Receives FDA Breakthrough Therapy Designation | Regulatory
Arrowhead Pharmaceuticals’ Plozasiran received an FDA Breakthrough Therapy designation. Plozasiran reduced triglycerides in patients with familial chylomicronemia syndrome (FCS), a rare genetic disorder. The therapy demonstrated an 80% reduction in triglyceride levels in the Phase 3 PALISADE trial. The treatment also showed an 83% decrease in the risk of acute pancreatitis, a major complication of FCS. Plozasiran has received both Orphan Drug and Fast Track designations to date.
Arcturus Therapeutics has received Investigational New Drug (IND) clearance for ARCT-032, an inhaled mRNA therapy designed to treat cystic fibrosis. Arcturus will begin a Phase 2 clinical trial to evaluate the drug’s safety, tolerability, and efficacy. ARCT-032 uses the company's proprietary LUNAR® technology, a lipid-based delivery platform, to deliver mRNA to the lungs. The therapy is aimed at restoring CFTR protein function in individuals who cannot benefit from cystic fibrosis existing treatments. This drug has already received multiple designations, including Orphan Drug and Rare Pediatric Disease status.
ADCs
Daiichi Sankyo and Merck’s ADC Demonstrates Progression-Free Survival Benefit in EGFRm NSCLC | Clinical Trial
The HERTHENA-Lung02 phase 3 trial showed that patritumab deruxtecan significantly improved progression-free survival (PFS) in patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC) compared to standard chemotherapy. The treatment is a HER3-directed antibody drug conjugate developed by Daiichi Sankyo and Merck. Overall survival data is still immature, and two more people died after developing interstitial lung disease, a known problem for Daiichi's ADCs. Despite these challenges, this advancement addresses a significant opportunity in the treatment of EGFR-mutated NSCLC with prior tyrosine kinase inhibitor treatment.
Daiichi Sankyo and AstraZeneca’s ADC Fails to Show Overall Survival Benefit in HR+ Breast Cancer | Clinical Trial
AstraZeneca and Daiichi Sankyo's TROP2-directed antibody-drug conjugate, datopotamab deruxtecan (Dato-DXd), has failed to improve overall survival (OS) in a phase 3 trial for hormone receptor-positive, HER2-low or negative breast cancer, despite meeting the progression-free survival (PFS) endpoint. The study compared Dato-DXd to standard chemotherapy but did not show a statistically significant OS benefit. The companies suggested that the approval of other ADCs during the trial may have impacted the results. Gilead's Trodelvy has previously shown a significant OS improvement in a similar setting. AstraZeneca plans to continue regulatory discussions based on the PFS results, with an FDA decision on Dato-DXd expected in early 2025. The ADC is also under review for non-small cell lung cancer, where it similarly did not improve OS.
Araris Biotech Expands its IP Portfolio with Acquisition of Innate Pharma’s Transglutaminase Patents | M&A
Araris Biotech AG has announced an agreement with Innate Pharma to acquire its portfolio of patents related to transglutaminase conjugation technology for antibody-drug conjugates (ADCs). The patents cover the use of bacterial transglutaminase for conjugating various linker-payloads to antibodies. Their proprietary technology, AraLinQ™, enables efficient ADC development with improved efficacy and tolerability, allowing the creation of multi-payload ADCs from standard antibodies without requiring engineering. This acquisition strengthens Araris's position in developing next-generation site-specific ADCs.
Innate Pharma Announces FDA Clearance of IND for Nectin-4 Targeted ADC | Regulatory
Innate Pharma has announced that the FDA has cleared its investigational new drug (IND) application for IPH4502, a novel antibody drug conjugate (ADC) targeting Nectin-4, aimed at treating solid tumors. A Phase 1 clinical study will begin soon, consisting of a dose escalation and optimization phase to evaluate IPH4502's safety, tolerability, and preliminary efficacy in advanced cancers expressing Nectin-4, such as urothelial, non-small cell lung, breast, ovarian, gastric, and colorectal cancers.
Pinotbio Secures Milestone Payment from Celltrion’s Execution of ADC Platform Technology Option | Partnership
Pinotbio, a Korean biotech firm specializing in antibody-drug conjugates (ADCs), announced it will receive a milestone payment from its partner, Celltrion, after Celltrion opted to advance the development of two drug candidates using Pinotbio’s proprietary ADC platform, PINOT-ADC. This payment, the first under their October 2022 agreement, follows an initial upfront payment of 1 billion won ($749,175) from Celltrion, which secured rights to use PINOT-ADC for up to 15 cancer targets. If all options are exercised, total payments could reach about $1.2 billion. PINOT-ADC aims to address safety and resistance issues seen in existing ADC therapies, utilizing a novel camptothecin derivative for enhanced efficacy.
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