Next-Generation Therapeutics Newsletter | March Roundup
Highlights & Summary
Intro
March was a month full of new partnerships and financing deals. Other key announcements include a number of new clinical trial updates and regulatory announcements.
Happy reading!
Cell Therapy
Gene Therapy
Oligo Therapy
Antibody-Drug Conjugates
Cell Therapy
CARGO Therapeutics Raises $200 Million in Series A for its Pipeline of CAR T-Cell Therapies | Financing
CARGO has managed to successfully close an oversubscribed funding round after its CD22 CAR-T demonstrated durable complete responses in > 50% of patients with large B-cell lymphoma (LBCL) that is relapsed/refractory (R/R) to CD19 CAR-T. The proceeds from the financing will be used to advance CARGO’s autologous CD22 CAR T-cell therapy candidate, CRG-022, through a pivotal multicenter Phase 2 trial in patients with LBCL.
Seagen entered a non-exclusive global deal with Gracell, a China based biotech, for Gracell to conduct pre-clinical research on Seagen’s cell therapy products and acquire (non-exclusive) rights to five of Seagen’s cell therapies. Details around the deal are sparse and the non-exclusive nature of the agreement is unusual in for this type of deal.
The non-exclusive licensing agreement enables the use of CRISPR/Cas9 to help accelerate the development of Vertex’s hypoimmune cell therapies for T1D. Vertex will pay CRISPR Therapeutics $100 million up-front for non-exclusive rights to CRISPR Therapeutics’ technology and CRISPR Therapeutics will be eligible for up to an additional $230 million once certain milestones are hit and receive royalties on any future products resulting from this agreement.
Sonoma and Regeneron Announce Collaboration to Discover & Launch Treg Cell Therapies for Autoimmune Diseases | Partnership
The two companies will discover, develop and commercialize novel regulatory T cell (Treg) therapies for autoimmune diseases. Under the agreement, Sonoma will receive $75 million in upfront payments, which includes a $30 million equity investment in Sonoma by Regeneron. Sonoma is also eligible to receive a $45 million development milestone payment. Regeneron and Sonoma will jointly research and develop Treg cell therapies for ulcerative colitis, Crohn’s disease and two other undisclosed indications, with a Regeneron option for a fifth indication.
Evotec and Bristol Myers Squibb initiated the collaboration in 2016 to identify disease-modifying treatments for a broad range of neurodegenerative diseases. This partnership leverages several of Evotec’s modality-agnostic precision medicine platforms. The partnership has already been successful in generating programmes - EVT8683, was in-licensed by Bristol Myers Squibb in September 2021. Evotec receives a $ 50 m upfront payment, undisclosed license and performance milestone payments and tiered royalties with the total deal being valued at potentially $4 bn.
Gene Therapy
Sarepta’s SRP9001 to Stand Before FDA Adcom Afterall | Regulatory
The FDA has surprisingly decided to hold an advisory committee meeting to discuss Sarepta’s product for Duchenne Muscular Dystrophy. The agency had previously decided not to hold such a meeting. It is unclear what drove the decision, but the company does not believe it is because of any data or evidence provided. Sarepta is in a tight race to reach the market with Pfizer, who also has an asset in Phase 3 development.?
Sangamo Loses Two Partners in Novartis and Biogen | Partnership
Sangamo, who previously signed deals with Novartis and Biogen both in 2020, had to announce both groups are walking away. Novartis explained it was due to their broader pipeline strategic review. Both partners’ interests were within CNS, and now those programs revert in full back to Sangamo. The company now must pin its AAV hopes on its Phase 3 asset for Hemophilia A, which is partnered with Pfizer.?
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领英推荐
GenSight’s LUMEVOQ Shows Sustained Improvements in Visual Acuity | Clinical Trial
GenSight reported positive efficacy and safety data out to 3 years for LUMEVOQ for the treatment of Leber Hereditary Optic Neuropathy. It has been a long road for GenSight, who previously saw an earlier trial miss its endpoint due to a contralateral treatment effect in which the therapy delivered to one eye can also migrate and improve acuity in the non-injected “control” eye. The company spent considerable effort investigating and ultimately demonstrating this as the cause for prior endpoint misses.
Novartis Ops-In to Two CNS-Focused Programs with Voyager | Partnership
After signing an agreement last year to evaluate capsids, Novartis is opting into two of three targets from the original agreement. This will see Voyager receive a $25M payment with an additional $600M in potential future milestones. These targets fall within the CNS and the new capsids are a result of Voyager’s TRACER platform.
ROCTAVIAN PDUFA Pushed Back to June | Regulatory
The FDA has relegated BioMarin to further waiting for a decision on their product for Hemophilia A. This comes after the company submitted analysis of 3-year data from the ongoing GENEr8-1 study. The agency deemed this a Major Amendment, resulting in a pushed-out decision date.?
Oligo?
QurAlis Closes Series B with $88M | Financing
QurAlis Corporation, focusing on therapeutic development for neurodegenerative diseases, including amyotrophic lateral sclerosis, announced #88 million in Series B financing. This brings their total funds to $143 million. The proceeds from their Series B will fund further development of their lead therapeutics candidates for ALS, QRL-2-1 and QRL-101.?
Switch Therapeutics has landed $52 million in denture funding. Switch focuses on development of small interfering RNA therapies with a goal to develop cell-specific targeting. The invested syndicate included Insight Partners and UCB Ventures.
Eplontersen demonstrated a positive effect in Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy | Clinical Trial
Positive results from the NEURO-TTRransform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy showed that eplontersen met the endpoints through 66 weeks. AstraZeneca and Ionis will be seeking regulatory approval for eplotersen. Eplotersen was granted Orphan Drug Designation previously for transthyretin amyloidosis.?
Alnylam Pharmaceuticals and Medison Pharma Expand Partnership to Commercialize RNAi Therapeutic Development | Partnership
The strategic expansion of Alnylam Pharmaceuticals and Medison Pharma will now enable patients in Central and Eastern Europe to access Alnylams’s RNAi therapies. Medison has a commercial platform. Moving forward, additional efforts will be made to further iexpand reimbursements in these areas.?
The company reported new preclinical data for VERVE-201 for the treatment of Homozygous Familial Hypercholesterolemia and is planning to initiate the first clinical trial in 2024. The asset is an mRNA-based therapy targeting the ANGPTL3 gene, using Verve’s LNP technology. According to DeciBio’s TheraTrack, there are currently ~5?unique oligo assets in development for the treatment of hypercholesterolemia.?
ADCs?
Pfizer projects to lose ~$17B due to patent expirations by 2030 and is working to add significant revenue from BD moves. The company has come to an agreement with Seagen for its first acquisition this year. Seagen is an ADC leader with 3 approved ADC assets, and according to DeciBio's TheraTrack, it has ~11 unique ADCs currently in development
Enhertu Met Pre-specified Target for ORR in Advanced Solid Tumors | Clinical Trial
Are we close to the first histology-agnostic approval from an ADC and is Enhertu going to make history again?
AstraZeneca and Daiichi announced positive results from the ongoing phase 2 trial for T-DXd in patients with multiple HER2-expressing advanced solid tumors. The asset met the pre-specified target for ORR and demonstrated durable responses across all patients. Data will be presented at an upcoming medical meeting.
The company informed the FDA that it is voluntarily pausing the trial for the drug, following a grade 5, fatal serious adverse event. The situation is under review and the company will evaluate next steps for development following the conclusion of the investigation. The company has 5 unique unique ADCs in development.
Currently recipients of exa-cel for the treatment of sickle cell disease and beta-thalassemia undergo an aggressive and toxic myeloablative conditioning regimen. Vertex sees promise in using ADCs as a more gentle and targeted conditioning regimen and is partnering with ImmunoGen to discover novel assets for this purpose. Immunogen is eligible to receive up to ~$340M in total payments.
Ambrx Re-evaluates It’s stance on the Development of ARX788 | Clinical Trial
In October 2022 the company nixed the internal development of ARX788 but is now reviving the program and will position the asset as a third-line option for HER2-positive breast cancer patients. 25% of patients who are on Enhertu progress within the first year, and Ambrx believes it can capture that market due to the asset's unique mechanism of action and the existing unmet needs in this patient population.