ADC Landscape in Approved Indications
Joe Daccache, Cassidy Humphreys and Phillip Leclair displayed the current Antibody-Drug Conjugates landscape for 7 indications where ADCs have been approved. Take a look!
ADC Landscape in Ovarian, Lymphoma, Leukemia, Breast, Bladder and Gastric Cancer
Highlights & Summary
Intro
June was a busy month for the next-generation therapeutics field, full of new financing deals, manufacturing updates, and regulatory announcements. Other key developments include a number of clinical trial and pipeline updates.
Happy reading!
Cell Therapy
- Bristol Myers Squibb Received FDA Blessing to Open New Cell Therapy Manufacturing Facility in Devens, Massachusetts | Manufacturing
- JNJ’s Phase 3 CARTITUDE-4 study showed Carvykti reduced the risk of disease progression or death by 74 percent in multiple myeloma | Clinical Trial
Gene Therapy
- uniQure Releases Promising Interim Results in Huntington’s Trial | Clinical Trial
- AAVantgarde Bio Raises > $60M Series A | Financing
Oligo Therapy
- Verve Establishes Collaboration with Lilly to Advance Treatments for Atherosclerotic Cardiovascular Disease | Partnership
- Vernal Biosciences Secures $20M for mRNA Manufacturing | Manufacturing
- Roche Terminates Trials of Rugonersen in Angelman Syndrome | Clinical Trial
Antibody-Drug Conjugates
- BiVictriX Nominates First Clinical Candidate | Pipeline
- Lantern Pharma Leverages AI Platform to Develop ADCs in Collaboration with Bielefeld University | Partnership
Cell Therapy
Many existing investors participated in the financing round that is primarily aimed at progressing Acepodia’s existing pipeline of cell therapies, namely ACE1831 and ACE2016. ACE1831 is an anti-CD20 armed allogeneic gamma delta 2 T-cell therapy currently in a Phase 1. ACE 2016 is an anti-EGFR armed allogeneic gamma delta 2 T-cell therapy targeting EGFR-expressing solid tumors. Acepodia has raised $259 million to date in venture capital financing, including a $109 million Series C round completed in December 2021.
Bristol Myers Squibb Received FDA Blessing to Open New Cell Therapy Manufacturing Facility in Devens, Massachusetts | Manufacturing
With the FDA approval of the facility, BMS is adding more capability and capacity to its cell therapy manufacturing footprint. BMS is making significant bets in the cell therapy space and the Devens site is a critical component to ensure the long-term supply of the company’s cell therapy portfolio. The new 244,000 square foot cell therapy manufacturing facility represents the second significant expansion of BMS’ 89-acre Devens site, which has been developing, producing, and testing clinical and commercial medicines for over a decade.
JNJ’s Phase 3 CARTITUDE-4 study showed Carvykti reduced the risk of disease progression or death by 74 percent in multiple myeloma | Clinical Trial
The study, done in patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy, showed significant improvement over two standard of care (SOC) regimens. Additionally, no new safety signals were observed over SOC therapies. Last month JNJ submitted a Type II variation application to EMA based on the CARTITUDE-4 study results seeking approval of CARVYKTI®.
CERo Therapeutics Announces Merger with a SPAC to Create Public Company | Financing
CERo Therapeutics is merging with Phoenix Biotech Acquisition Corp, a SPAC, that will help CERo raise funds for developing its T-cell therapeutics that employ phagocytic mechanisms. Specifically, anticipated funds will go towards advancing CERo’s lead product candidate, CER-1236 to early clinical data in hematological oncology. The transaction reflects a pre-money equity value of $50 million for CERo, pro forma equity capitalization of $145 million.
ProKidney Announces Purchase of Manufacturing Facility in Greensboro, NC | Manufacturing
ProKidney, a late clinical-stage cell therapy company focused on preserving kidney function in patients with chronic kidney disease (CKD), agreed to purchase a 210,000 square foot facility in Greensboro, N.C., for approximately $25.5 million in cash. The facility will support the company’s future manufacturing needs for its key asset, REACT (REnal Autologous Cell Therapy), currently in Phase 3 for diabetic CKD.
Gene Therapy
Sarepta Receives FDA Approval of ELEVIDYS for Muscular Dystrophy | Regulatory
Sarepta received approval for ELEVIDYS for treatment of patients ages 4-5 with Duchenne Muscular Dystrophy. Sarepta plans to price the drug at $3.2M, making it one of the most expensive medicines in the world. In approving the drug, the FDA’s Peter Marks overruled concerns that reviewers had about the level of efficacy the drug provides, citing the incredible unmet need. The field will now watch closely Pfizer’s readout for their competing candidate.
uniQure Releases Promising Interim Results in Huntington’s Trial | Clinical Trial
uniQure’s interim results for AMT-130 was generally well-tolerated and generally showed dose-dependent results in the low and high-dose cohorts that were tested compared to placebo. These interim results also showed improvements in functional capacity and disease progression, which the company touted. The company plans to discuss further development with regulators in the coming year.
Review - Rational and Strategies for Developing Better AAV | Pipeline
Despite a handful of AAV-based therapeutics being approved by regulators, stakeholders appreciate that better delivery modalities are needed. First-generation AAV have a host of issues around inducing immune responses, limited carrying capacity, and limited opportunity for re-dosing. A new review from Prof. Srivastava at the University of Florida outlines approaches being taken to develop these next-generation carriers.
AAVantgarde Bio Raises > $60M Series A | Financing
Based in Milan, Italy, AAVantgarde Bio has come out of stealth with significant cash on hand. The clinical-stage company has two platforms that they believe will enable the delivery of large genes. The company is focusing initially on diseases of the eye and will expand to other tissues in the future.
Beacon Therapeutics Launches with Repurposed Assets and $120M | Financing
Beacon has officially unveiled a venture round lead by Syncona. The company’s lead asset comes from AGTC, who Syncona acquired last year. The company also in-licensed another program from the University of Oxford for cone-rod dystrophy. Syncona is quite active in the gene therapy space, with additional investments in Freeline Therapeutics, SwanBio Therapeutics, and Purespring Therapeutics.
Oligo
Verve Establishes Collaboration with Lilly to Advance Treatments for Atherosclerotic Cardiovascular Disease | Partnership
Verve received $60M as an upfront payment and investment to advance their in vivo gene editing targeting lipoprotein (A). Lp(A) is a risk factor for several vascular-related cardiovascular diseases including stroke and aortic stenosis. The capital Verve is expected to receive from Lilly will help extend the cash runway into 2026.
Moderna Files for FDA Authorization for an Updated Covid Vaccine | Regulatory
Moderna has recently announced a completed submission to the FDA for an updated COVID-19 vaccine containing spike proteins for the XBB.1.5 sublineage. Doses will be ready to ship in the fall, pending authorization.
Vernal Biosciences Secures $20M for mRNA Manufacturing | Manufacturing
Vernal Biosciences is a CDMO that specializes in mRNA and plasmid mRNA and has secured additional capital to build a 20,000 square-foot site in Vermont. This recent funding is an extension of their Series A which totaled $21M in 2022.
Roche Terminates Trials of Rugonersen in Angelman Syndrome | Clinical Trial
Roche recently announced that they would no longer initiate any new trials with Rugonersen (RO7248824) in Angelman Syndrome following the current Phase 1 study results. No safety concerns were reported, and there were some encouraging effects on a key biomarker in AS. As the results were encouraging, Roche will offer an open-label extension for patients.
Charles River and Curigin Collaborate on RNAi Gene Therapy | Partnership
Charles River Laboratories, and Curigin, a Korean biotechnology company announced a collaboration to develop oncolytic ribonucleic acid interference (RNAi) gene therapies. This collaboration will leverage Curigin’s pipeline and Charles River’s manufacturing expertise as a CDMO.
ADCs
BiVictriX Nominates First Clinical Candidate | Pipeline
The company has announced the selection of BVX001 as a clinical candidate for its lead program. BVX001 is a bispecific ADC that targets CD7 and CD33. Recent data has shown significant tumor regressions of up to 93% in a murine model of Acute Myeloid Leukemia (AML), supporting the advancement of BVX001 towards clinical development. According to DeciBio's ADC tracker, TheraTrack, BiVictriX has three bispecific ADC assets in development for both solid and hematologic cancers.
Tagworks Therapeutics Raises $65M to Advance Its Click-to-Release Therapeutics | Financing
Tagworks secured $65 million in Series A financing led by Ysios Capital and Gilde Healthcare, with participation from Novartis Venture Fund, New Enterprise Associates, and Lightstone Ventures. The funding will be used to advance the lead program, TGW101, a click-cleavable ADC targeting TAG72, and expand their pipeline currently consisting of 5 ADCs. The company's platform enables controlled drug release for ADCs, immunomodulators, and radiopharmaceuticals, potentially enhancing efficacy and safety in the treatment of solid tumors.
Sutro Biopharma and Blackstone Announce Financing Collaboration | Partnership
Sutro Biopharma and Blackstone have entered into a collaboration where Sutro will receive $140 million upfront, with potential milestone payments of up to $250 million, in exchange for a 4% royalty in future sales of Vaxcyte's products. This deal provides Sutro with non-dilutive capital to advance its technologies and two promising assets, STRO-002 and STRO-003.
Mersna Announces Partial Clinical Hold on Two studies for Its Lead Asset | Regulatory
The company announced that the FDA has placed a partial clinical hold on 2 clinical trials for its lead ADC asset, UpRi, for the treatment of ovarian cancer. This hold was prompted by five patient fatalities linked to bleeding episodes, revealing potential safety concerns. Mersana is hoping to compete with Immunogen's successful ADC, Elahere, which earlier this month has demonstrated reduction in risk of disease progression and death in FRa-positive Ovarian Cancer.
Lantern Pharma Leverages AI Platform to Develop ADCs in Collaboration with Bielefeld University | Partnership
Lantern Pharma will leverage its RADR® AI platform in collaboration with Professor Norbert Sewald at the Bielefeld University to develop innovative and highly targeted ADCs. The partnership will first focus on synthesizing and evaluating ADCs linked to cryptophycins, with potential outcomes expected before the end of 2023. Lantern also receives an exclusive option to license intellectual property from the collaboration.
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