Highlights & Summary
Intro
A number of key developments occurred during the last month of 2022, including new partnerships forming across the cell, oligo, and antibody-drug conjugate spaces, approval of the first autologous T-cell immunotherapy (tabelecleucel), and exciting clinical trial updates.
Happy reading!
Cell Therapy
- Atara Biotherapeutics’ tabelecleucel Becomes the First Allogenic T-Cell Immunotherapy to Receive Marketing Approval | Regulatory
- AstraZeneca to Acquire Neogene Therapeutics for $320M, Signaling Further Ambition in Cell Therapy | M&A
- Catalent Opens a 60,000 Square Feet Cell Therapy Manufacturing Facility at its European Center of Excellence for Cell Therapies in Belgium | Manufacturing
Gene Therapy
- Passage Bio Announces Positive Interim Data from their Phase I/II Trial in GM1 Gangliosidosis | Clinical Trial
Oligo Therapy
- Alnylam Submits Supplemental New Drug Application for ONPATTRO | Clinical Trial
- Moderna and Merck Announce an Investigational mRNA Cancer Vaccine in Combination with Keytruda in Phase 2B Met Primary Efficacy| Clinical Trial
Antibody-Drug Conjugates
- Avidity Biosciences Announces Positive AOC 1001 Phase 1/2 Data | Clinical Trial
- Dantari Debuts with $47M Series A | Company Creation
- Gilead Announces New Data from the Phase 3 TROPiCS-02 Study Evaluating Trodelvy® in Metastatic Breast Cancer | Clinical Trial
- Merck and Kelun-Biotech Announce a ~9.4B License and Collaboration Agreement for 7 New ADCs | Partnership
Cell Therapy
Atara Biotherapeutics’ tabelecleucel Becomes the First Allogenic T-Cell Immunotherapy to Receive Marketing Approval | Regulatory
The European Commission (EC) has granted marketing authorization for Ebvallo™ (tabelecleucel) as a second line monotherapy for the treatment of adult and pediatric relapsed or refractory EBV+ PTLD, a rare hematologic disease in the post-transplant setting. Under a previous agreement with Atara, Pierre Fabre will lead all commercialization and distribution activities in Europe.
AstraZeneca to Acquire Neogene Therapeutics for $320M, Signaling Further Ambition in Cell Therapy | M&A
AstraZeneca’s acquisition will allow it to leverage Neogene’s portfolio and expertise in development and manufacturing of next-generation T-cell receptor therapies (TCR-Ts) for solid tumors. This acquisition will include an initial payment of $200m on deal closing, and a further up to $120m in both contingent, milestones-based and non-contingent consideration.
Catalent Opens a 60,000 Square Feet Cell Therapy Manufacturing Facility at its European Center of Excellence for Cell Therapies in Belgium | Manufacturing
Catalent has opened one of the world’s largest cell therapy manufacturing facilities housing multi-product, segregated suites designed to support autologous and allogeneic cell therapy manufacturing through to late-stage clinical and commercial-scale supply.
The first Blackstone payment of $35m comes after an interim analysis of pivotal FELIX Phase 2 clinical trial of obe-cel in relapsed/refractory (r/r) adult ALL, showing the study has met its primary endpoint. The second $35m milestone is a result of completion of planned activities demonstrating the performance and qualification of Autolus’ obe-cel’s manufacturing process.
The collaboration will be to co-develop and co-commercialize Arcellx’s lead Phase 2 product candidate, CART-ddBCMA, an autologous T cell therapy utilizing Arcellx’s novel D-Domain binder. Arcellx will receive an upfront cash payment of $225 million and $100 million equity investment as well as other potential contingent payments. The companies will share costs and will jointly commercialize the product and split U.S. profits 50/50. Outside the US, Kite will commercialize the product and Arcellx will receive royalties on sales.
Gene Therapy
Sean Nolan takes Over as CEO of Taysha Gene Therapies | Personnel
RA Session II founded the company in 2019 is stepping aside as Sean Nolan takes the helm. Session, who will remain on the board, helped execute one of the fastest seed to IPOs in biotech history. More recently, in parallel with the broader downturn, the company honed its focus on key pipeline assets while deprioritizing others. The company will now try to expedite progress on their lead assets and strengthen their partnership with Astellas, who made an equity investment in October.
Passage Bio Announces Positive Interim Data from their Phase I/II Trial in GM1 Gangliosidosis | Clinical Trial
Passage Bio announced their candidate for treating GM1 gangliosidosis showed a clean safety profile at both the low and high dose. The trial demonstrated a dose-dependent response in levels of GM1 gangliosides in cerebrospinal fluid, with one patient notching a 75% reduction from baseline at the 6-month mark.
Verve Announces Letter from FDA Outlining Requirements for Lifting IND Hold | Clinical Trial
The FDA, which placed a hold on Verve’s IND for VERVE-101, has now told the company what is needed to address the issue. The therapy is designed to permanently turn off the PCSK9 gene in liver cells to reduce LDL levels. The FDA said they would like to see data on potency across human and non-human cell types as well as the risk of editing germline cells. Despite this delay, the company continues to enroll patients in a trial of the therapy in the UK and New Zealand.
Solid BIosciences Closes on Acquisition of AavantiBio and $75M Private Placement | M&A
Solid Biosciences announced the close of its acquisition of AavantiBio, which it first announced in September. The company will now focus on neuromuscular and cardiac-related programs. In parallel, the company closed on a $75M investment lead by Perceptive Advisors, LLC, RA Capital, and Bain Capital Life Sciences. This should allow the company to advance its pipeline to the clinic.
Oligo
Alnylam Submits Supplemental New Drug Application for ONPATTRO | Clinical Trial
Alnylam Pharmaceuticals submits an sNDA for ONPATTRO (patisiran). This is an RNAi therapeutic for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, based on the results from the APOLLO-B Phase 3 study.
Moderna and Merck Announce an Investigational mRNA Cancer Vaccine in Combination with Keytruda in Phase 2B Met Primary Efficacy| Clinical Trial
Moderna’s mRNA-4157/V940, in combination with KEYTRUDA, demonstrated a statistically significant improvement for recurrence-free survival versus KEYTRUDA alone. There is a plan for a phase 3 study in melanoma next year.
Wave Life Sciences and GSK Collaborate on Oligo Therapies | Partnership
Wave Life Sciences receives $170M USD and brings together Wave’s PRISM oligonucleotide platform with GSK’s perspective on therapy development. One of the main focuses of the collaboration will include Wave’s RNA editing program that targets alpha-1 antitrypsin deficiency (AATD).
CSL Announces Licensing Agreement with Arcturus Therapeutics | Partnership
CSL finalized their collaboration with Arcturus Therapeutics. This agreement permits CSL access to Acrturus’ sa-mRNA platform technology for vaccines. Arcturus will receive $200M USD and will be focusing on the next generation of mRNA vaccines.
ADCs
Avidity Biosciences Announces Positive AOC 1001 Phase 1/2 Data | Clinical Trial
AOC 1001 is being investigated to treat the root cause of Myotonic Dystrophy Type 1 and preliminary data demonstrated that AOC 1001 is the first-ever successful targeted delivery of RNA into muscle, opening the door to previously unreachable tissues and targets. According to DeciBio’s TheraTrack, Dyne Therapeutics is also investigating the efficacy of its DYNE-101 ADC for the treatment of DM1.
Dantari Debuts with $47M Series A | Company Creation
Dantari is advancing its novel and differentiated platforms for the development of Targeted High-capacity Drug Conjugates (T-HDC). In comparison to today’s ADCs, this innovative technology enables the conjugation and delivery of a high DAR. The company is currently developing 2 ADCs, DAN-311 and DAN-405, with a DAR of ~60 with at least one expected to enter the clinic in 2023.
Biosion Announces Research Collaboration with ImmunoGen to Create Novel ADCs | Partnership
An exploratory research collaboration between Biosion and ImmunoGen was announced for the development of novel ADCs. Biosion will identify and generate highly selective, highly endocytic antibodies and Immunogen will provide its proprietary linker-payload technology.
Freenome and ADC Therapeutics Find Promising Results in Biomarker Research | Partnership
As part of a large collaboration, Freenome and ADC Therapeutics have been applying the platform to investigate signatures of responses to Lonca (anti-CD19 ADC) in DLBCL. Early data presented at ASH showed that, by looking at cfDNA, Freenom's technology was able to identify biomarkers associated with responses to Lonca, as well as mechanisms of resistance in non-responders.
Gilead Announces New Data from the Phase 3 TROPiCS-02 Study Evaluating Trodelvy® in Metastatic Breast Cancer | Clinical Trial
Post-hoc analysis from the phase 3 study in HR+/HER2- metastatic patients, revealed improved outcomes compared to chemotherapy. The PDUFA target date is set for February 2023. The competition is heating up for this patient population with Daiichi Sankyo also announcing results from the TROPION-PanTumor01 trial. According to DeciBio’s Theratrack, there are currently 6 Trop-2 targeting ADCs in development, for the treatment of breast cancer.
Merck and Kelun-Biotech Announce a $9.4B License and Collaboration Agreement for 7 New ADCs | Partnership
This third agreement between the two companies amounts to a staggering $9.4B deal for 7 investigational preclinical antibody-drug conjugates. Additionally, Merck did not only secure the right to research, develop, manufacture and commercial the assets, but also snagged a hold on future ADCs with exclusive options to obtain additional licenses.
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