Eventful JPM24: Key Pharma and Biotech Highlights
ALSO: Towards Novel ALS Therapy; 10 Major Biotech Funding Rounds of 2023; Life Sciences & Net Zero: Charting a Sustainable Path
Hi! I am Andrii Buvailo, and this is my weekly newsletter, ‘Where Tech Meets Bio,’ where I talk about technologies, breakthroughs, and great companies moving the biopharma industry forward.
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Now, Let’s get to this week’s topics!
One of the key events in the pharma and biotech industries, the J.P. Morgan Healthcare Conference in San Francisco (JPM24), has concluded with cautious optimism for the year ahead. Despite ongoing challenges, there are early signs of a potential turnaround, with expectations of a biotech funding recovery in the coming year.
The cell and gene therapy sector remains strong, with predictions of up to 17 approvals by regulatory agencies in the U.S. and EU.
Artificial intelligence (AI) was a dominant topic, with Big Tech companies like Alphabet making significant deals in AI-based drug discovery. NVIDIA’s generative AI grip on biomedical field and healthcare gets stronger.
Additionally, AI, neuroscience, and GLP-1 drugs are expected to gain momentum in 2024. Policy and regulatory issues, including drug price negotiations and patent rights, are also on the industry's radar. While challenges persist, the biopharma sector is energized to tackle them in the year ahead.
JPM24: Weekly pharma & biotech highlights
The announcements below are a continuation of a really eventful start to the year, which I outlined in my previous newsletter. Here, let’s continue with the highlights of JPM Week:
🔬 Amgen partners with NVIDIA to utilize DGX SuperPOD for generative AI models, aiming to accelerate drug discovery through analysis of one of the world's largest human datasets at deCODE genetics in Iceland.
💰 Illumina Ventures launches a refreshed startup accelerator program, "Illumina Ventures Labs," providing genomics startups with up to 18 months of incubation, mentorship, and access to DNA analysis technology in the UK and California.
🔬 Recursion Pharmaceuticals unveils LOWE, a Large Language Model-Orchestrated Workflow Engine, in partnership with NVIDIA, at the J.P. Morgan Healthcare Conference, demonstrating a natural language interface for drug discovery and automated laboratory experimentation.
💰 Boston Scientific announces a $3.7 billion acquisition of medtech company Axonics, venturing into advanced technology for bladder function improvement and bolstering its portfolio in medical devices for urological conditions.
🔬 WuXi Biologics partners with BioNTech to discover investigational monoclonal antibodies for next-generation therapeutic products, leveraging its antibody discovery technology platforms. WuXi Biologics will receive a $20 million upfront payment and additional payments based on research, development, regulatory, and commercial milestones, along with tiered royalties.
💰 Johnson & Johnson announces a $2 billion cash acquisition of Ambrx Biopharma, a pioneer in antibody-drug conjugates (ADCs) for cancer treatment, joining other major pharmaceutical companies in the ADC field, including Pfizer, AbbVie, and Merck, as part of J&J's strategy to address future revenue challenges.
🔬 Flagship Pioneering has announced an "enabling technologies" initiative during the J.P. Morgan Healthcare Conference, with a focus on developing tools and technologies for biotech and life sciences companies, supported by partnerships with Thermo Fisher Scientific and Samsung; the collaboration with Samsung involves providing tech tools and investment for Flagship's portfolio companies, spanning AI, translational medicine, and clinical trials infrastructure.
🔬 ConcertAI and Caris Life Sciences have formed a strategic agreement with AbbVie to accelerate drug discovery, clinical development, and precision medicine efforts in oncology, utilizing their extensive clinical and genomic databases, as well as AI and machine learning insights, to guide therapy development and optimize clinical trials and patient enrollment.
🔬 Caris Life Sciences and Flatiron Health have joined forces to create a multimodal data offering that supports and accelerates biopharmaceutical drug development and patient care in oncology by combining Caris' extensive genomic, transcriptomic, and imaging database with Flatiron's longitudinal patient data and clinical outcomes, aiming to provide cancer researchers with robust real-world data at scale
🔬 Deepcell has partnered with NVIDIA to accelerate the use of generative artificial intelligence in single cell research, aiming to advance understanding of cell morphology and enhance the use of AI-powered cellular analysis in various life sciences applications, including cancer, stem cell, and cell therapy research.
A Proof-of-Concept Study Towards Novel ALS Therapy
I first read about Verge Genomics in this Forbes article back in 2018, and I remember being somewhat skeptical. The business was centered on using AI to tackle one of the most difficult areas of drug discovery—neurodegenerative diseases. ALS. A "graveyard" for drug hunters...
But here we are in 2024. And the company, it seems, is doing fine!
They have just announced a Phase 1b proof-of-concept study for VRG50635, a drug developed for the treatment of ALS, also known as Lou Gehrig’s disease. The study aims to evaluate the safety and tolerability of escalating doses of VRG50635, a small-molecule inhibitor of PIKfyve, a therapeutic target discovered in diseased human tissues, using the company's AI-powered platform, CONVERGE,® which includes a comprehensive database of multi-omic patient data.
ALS is a progressive neurodegenerative disease that affects motor neuron cells in the brain and spinal cord, leading to paralysis of voluntary muscles. This disease typically affects individuals between 40 and 70 years of age, and an estimated 100,000 people worldwide may have ALS at any given time. The disease is fatal, and current treatments only offer modest benefits to the majority of patients, who often succumb within 2 to 5 years after diagnosis.
VRG50635 is notable for being one of the first drugs to enter clinical trials that was entirely discovered and developed using an AI-enabled platform, including the target.
Innovative Clinical Trial Design
The proof-of-concept study for VRG50635 is distinct from traditional ALS clinical trials in its approach to measuring patient progress.
Instead of relying on physician rating scales in a clinical setting, which often provide limited data, this study utilizes digital devices to collect a vast array of objective, quantitative measurements directly from patients in their homes.
These measurements include mobility, breathing, and sleeping patterns. This innovative method enables a more precise and personalized assessment of disease progression and treatment-related changes.
VRG50635 has shown efficacy in preclinical studies and has the potential to be a best-in-class therapy for ALS. It is a potent, orally bioavailable PIKfyve inhibitor optimized specifically for the treatment of central nervous system disorders like ALS.
Earlier in 2023, BiopharmaTrend reported promising results from the Phase 1 clinical trial of VRG50635.
The trial, which tested the drug's safety and tolerability in 80 healthy adult volunteers, reported no serious adverse events and confirmed the drug's safety. The pharmacokinetics of VRG50635 indicated dose-proportional increases, supporting its potential as a once-daily oral medication.
Notably, Verge Genomics managed to progress the drug candidate to this stage within just 4 years, including the target discovery and drug design, which speaks in favor of their AI platform capabilities.
Time will tell how it all goes. But I sincerely wish Verge Genomics good luck with solving this riddle!
10 Major Biotech Funding Rounds of 2023
These 10 companies raised a total of more than USD2.3 billion in 2023, to advance a wide range of technologies—RNA therapeutics and platforms, mAbs, gene editing, AI in drug discovery, and cell therapies.