.png)
Highlights & Summary
Intro
November was an exciting month for the next-generation therapeutics field, full of new partnerships, regulatory and financing updates.Happy reading!
Cell Therapy
- Kite and Arcellx Announce Expansion in Strategic Partnership In a Deal Valued CloseTo $285M Overall | Partnership
- Atara Biotherapeutics' Phase II Readout in MS Dissapoints, Sending the Stock Down Nearly 50% | Clinical Trial
Gene Therapy
- Sarepta Announces Shock ELEVIDYS Primary Endpoint Miss | Clinical Trial
- REGENXBIO Announces Additional Data on Clinical-Stage Eye Trial | Clinical Trial
- Astellas Publishes Additional Detail Around Deaths in AT132 Trial | Clinical Trial
Oligo Therapy
- Moderna mRNA patent revoked by EPO after Pfizer Suit | Regulatory
- Daiichi Sankyo Secures Agreement to Provide 1.4 Million Doses of mRNA COVID Vaccine in Japan | Partnership
Antibody-Drug Conjugates
- MBrace therapeutics Raises $85 Million Series B | Financing
- Tubulis Partners its CD30-Targeting ADC with OncoTeq | Partnership
Cell Therapy
The FDA is investigating the safety of CAR-T therapies after 20 reports of patients developing blood cancers post-treatment since 2017. The agency assures that the benefits of these therapies still outweigh the risks. All six approved CAR-T products are under review. The announcement surprised key researchers and impacted the stocks of CAR-T developers, particularly smaller biotechs. Despite concerns, the industry, including companies like Gilead and BMS, stands by the safety of their treatments. Concurrently, research into CAR-T's potential in treating autoimmune diseases like lupus continues, with several studies underway.
Kite and Arcellx Announce Expansion in Strategic Partnership In a Deal Valued CloseTo $285M Overall | Partnership
Gilead's Kite has expanded its partnership with Arcellx, investing an additional $200 million for equity and $85 million in cash. This follows the FDA lifting a clinical hold on their joint CART-ddBCMA candidate for multiple myeloma. The enhanced collaboration now also covers lymphoma treatments. Kite's investment, priced at a significant premium, boosts Arcellx’s financial runway into 2027 and gives Gilead about a 13% stake in Arcellx. The companies anticipate sharing new trial data at the upcoming ASH conference in December.
BMS and 2seventy bio announced an upcoming FDA Oncologic Drugs Advisory Committee review for the supplemental BLA of Abecma for earlier treatment stages of triple-class exposed relapsed or refractory multiple myeloma. The meeting date is unconfirmed, and the FDA will not meet the original decision deadline of December 16, 2023. The review will focus on overall survival data from the KarMMa-3 study, which showed significant improvement in progression-free survival. The companies are also awaiting regulatory decisions in Japan, Europe, and Switzerland for Abecma based on these results.
CARGO IPOs In What is LIkely One of The Lasts IPOs of 2023 | Financing
CARGO Therapeutics launched its public offering at $15.00 per share, offering 18,750,000 shares and with gross proceeds of about $281.3 million. The biotech company also granted underwriters an option to buy up to 2,812,500 additional shares. CARGO's common stock will started trading on the Nasdaq under the ticker "CRGX" on November 10, 2023. J.P. Morgan, Jefferies, TD Cowen, and Truist Securities managed the offering. This is likely one of the last public offerings for 2023, resulting in a relatively quiet funding year
Atara Biotherapeutics' Phase II Readout in MS Dissapoints, Sending the Stock Down Nearly 50% | Clinical Trial
Atara Biotherapeutics' cell therapy, ATA188, failed to meet its primary endpoint in a Phase II study for progressive multiple sclerosis, leading to a significant drop in the company's shares. Atara will now reduce investment in this program and focus more on its allogeneic CAR-T pipeline and partnership with Pierre Fabre, extending its cash runway beyond Q3 2025. The company's CEO expressed disappointment in the results and plans to halt the study. Atara is also preparing for upcoming milestones, including presenting pre-clinical data for ATA3431, and progressing with its T cell immunotherapy Ebvallo, approved in the EU and heading for a US filing in Q2 2024.
Gene Therapy
Sarepta Announces Shock ELEVIDYS Primary Endpoint Miss | Clinical Trial
Sarepta Therapeutics released topline results from its Phase 3 study of ELEVIDYS. In a surprise, The study did not meet its primary endpoint, although patients showed improvement compared to the placebo group, hitting the trial’s secondary endpoints around time to rise and a 10-meter walk test. The company plans to submit an efficacy supplement to the BLA, but these results do cast a shadow on the drug’s prospects. Rival Pfizer now sees a potential opening for its candidate, currently in Phase 3 testing. The company has suggested they may reveal interim data before year-end.
Freeline Ultimately Decides to Sell to Investor Syncona | M&A
Freeline Therapeutics has ultimately decided selling itself to current investor, Syncona Ltd, is the best course of action. The all-cash transaction values each share at $6.50. This deal, subject to the approval of Freeline's minority shareholders, will close in Q1 2024. The primary asset in Freeline’s pipeline is FLT201 for the treatment of Gaucher disease. Data released last month from an ongoing Phase 1 /2 trial was encouraging. Freeline’s predicament underscores the difficulty of smaller, pre-commercial biotechs riding the waves of the public markets.
REGENXBIO Announces Additional Data on Clinical-Stage Eye Trial | Clinical Trial
REGENXBIO reported positive one-year data from the ongoing Phase II ALTITUDE® trial of ABBV-RGX-314 for treating diabetic retinopathy. The trial demonstrated ABBV-RGX-314, administered through suprachoroidal delivery, to be well-tolerated and to have clinically meaningful improvements for patients with non-proliferative retinopathy. The candidate is being jointly developed with Abbvie dating back to the pair’s September 2021 partnership.
Ascidian Adds to their Series A Haul for RNA Editing Platform | Financing
Ascidian Therapeutics raised $40 million in Series A extension funding from Apple Tree Partners (ATP), bringing its total Series A investment to $90 million. The funds will support the advancement of Ascidian's lead program, an RNA exon editor targeting Stargardt disease and other ABCA4 retinopathies, as well as additional pipeline programs. The firm uses AAV to deliver the machinery locally. The lead program is on the cusp of beginning human trials in Stargardt disease.
Astellas Publishes Additional Detail Around Deaths in AT132 Trial | Clinical Trial
Astellas has detailed the circumstances surrounding four deaths in its AT132 gene therapy clinical trial for X-linked myotubular myopathy. The study, published in The Lancet Neurology, reveals liver toxicity as the cause of death, distinct from other gene therapies. The study remains on clinical hold, with all four deceased patients experiencing cholestatic liver failure. While the deaths were not linked to immune responses, immunosuppressive regimens showed no benefits. Astellas continues investigations to address safety issues.
Oligo
Arcturus Therapeutics' ARCT-032 Granted FDA Orphan Drug Designation | Regulatory
Arcturus Therapeutics has received Orphan Drug Designation from the U.S. FDA for ARCT-032, targeting cystic fibrosis (CF). The designation offers incentives such as potential market exclusivity for seven years and tax credits for qualified clinical trials. The first CF patient in the Phase 1b study of ARCT-032 has completed two administrations successfully, and the company is set to share interim Phase 1b data in the first half of 2024. ARCT-032 utilizes Arcturus' LUNAR® lipid-mediated aerosolized platform to deliver messenger RNA to the lungs.
Moderna mRNA patent revoked by EPO after Pfizer Suit | Regulatory
Moderna's lawsuit against Pfizer and BioNTech, filed in August 2022, has resulted in the invalidation of one of its COVID-19 patents by the European Patent Office (EPO). The suit alleged infringement by Pfizer and BioNTech's Comirnaty vaccine, utilizing Moderna's patented mRNA technology. The EPO ruled in favor of Pfizer and BioNTech, stating that Moderna's patents were overly broad. Moderna plans to challenge the decision, and while success is plausible, legal proceedings may be prolonged and costly. The outcome could impact vaccine sales revenue for Pfizer, which has faced challenges in this regard
Nutcracker Therapeutics Showcases Antitumor Activity of Two Preclinical mRNA Assets | Pipeline
Nutcracker Therapeutics, a biotech company focusing on RNA therapies, presented preclinical data at the Society for Immunotherapy of Cancer 38th Annual Meeting. The data highlighted two therapeutic candidates: NTX-471 and NTX-250, the lead mRNA candidate for HPV-driven tumors. The company's proprietary platform facilitated the rapid design and production of multiple RNA molecules. Additionally, data on NTX-250 demonstrated its efficacy in eradicating HPV16-transformed tumors, showcasing the potential of Nutcracker's RNA therapies in cancer treatment.
Daiichi Sankyo Secures Agreement to Provide 1.4 Million Doses of mRNA COVID Vaccine in Japan | Partnership
Daiichi Sankyo is awaiting local approval for its omicron-adapted mRNA COVID-19 vaccine and, in the meantime, has secured a supply deal with Japan’s Ministry of Health, Labor and Welfare (MHLW). The agreement entails providing 1.4 million doses of the vaccine during the 2023 fiscal year. Although the financial terms remain undisclosed, Daiichi is gearing up its production facilities to meet the supply commitment.
ADCs
AbbVie to Acquire ImmunoGen | M&A
AbbVie's strategic acquisition of ImmunoGen for ~$10 billion positions the company to accelerate its impact in the solid tumor space, with a particular emphasis on ovarian cancer. This move underscores AbbVie's commitment to diversifying its oncology pipeline and strengthening its capabilities in ADCs. Concurrently, AbbVie's Phase 2 LUMINOSITY trial for Teliso-V in NSCLC revealed promising results, demonstrating a compelling ORR and meaningful clinical outcomes. This marks a significant advancement in AbbVie's pursuit of a leadership position in the ADC market
MBrace therapeutics Raises $85 Million Series B | Financing
MBrace raised $85 million in a Series B financing round, led by TPG, bringing the total funds raised to over $110 million. This funding will accelerate the clinical development of MBrace's EphA5-targeting ADCs, with a focus on the lead candidate, MBRC-101, for the treatment of specific solid cancers. MBrace aims to initiate Phase 1 studies with MBRC-101 by the end of 2023
BMS and Avidity Announce Expansion of Cardiovascular Collaboration | Partnership
Avidity Biosciences has expanded its collaboration with BMS, focusing on up to five specific cardiovascular targets and utilizing Avidity's proprietary AOC platform technology. The partnership is valued at ~$2.3 billion. This collaboration underscores Avidity's commitment to advancing RNA therapeutics in cardiology by combining its AOCs with BMS’s expertise in cardiovascular drug discovery. Avidity continues to pursue internal research in rare cardiac indications while broadening the application of its AOC platform. According to DeciBio’s TheraTrack, the company has ~7 ongoing internal AOC programs
BMS Acquires Orum’s CD30-Targeting ADC | M&A
ORM-6151 is a first-in-class anti-CD33 antibody-enabled GSPT1 degrader, and has received FDA clearance for Phase 1 to treat AML or MDS. The acquisition involves an upfront payment of $100 million, with potential milestone payments totaling $180 million. Orum's unique Dual-Precision Targeted Protein Degradation approach, utilizing antibody drug conjugates, aims to enhance therapeutic efficacy in cancer cells. According to DeciBio’s TheraTrack Orum currently has more than 8 active programs
Tubulis Partners its CD30-Targeting ADC with OncoTeq | Partnership
Oncoteq AG has secured exclusive global development rights for the potential best-in-class ADC, TEQ-102, from Tubulis. This CD30-targeting ADC aims to treat T-cell and Hodgkin’s lymphomas, offering improved drug delivery to cancer cells. TEQ-102's design allows for higher, more effective doses, aligning with Oncoteq's approach of building a robust pipeline through in-licensing deals. According to TheraTrack Tubulis currently has ~5 active internal ADC programs
.png)
.png)
