D. Michele Nikoloff

Have you seen the latest issue of the Journal of the National Comprehensive Cancer Network® (NCCN®)? There is a must-read article on research conducted by some of our brilliant team at Evidera!

14

AAV is the leader of gene therapy. But what are it's pros and cons? Let's break it down. Pros: - Current leader in vector-based gene therapy - Payload doesn't integrate into patient DNA (normally) - FDA-approved therapies on the market - Can target specific tissues for precise delivery Cons: - High cost of manufacturing - Small payload size - Good quality at scale is a challenge - Single dose administration due to high immunogenicity (for now) - Liver toxicity concerns (for now) I'm excited to see how recent advances in capsid design will fix these drawbacks. Anything else you'd add? Drop it in the comments. For content that breaks down the science of CGT: Check out #cgt_simplified “Created with BioRender.com” #genetherapy #aav #biotechnology

152

25 Comments

<!-- Content start --> South San Francisco, CA — July 3, 2024 — Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the Phase II/III SKYSCRAPER-06 study, evaluating tiragolumab plus Tecentriq® (atezolizumab) and chemotherapy versus pembrolizumab and chemotherapy as an initial (first-line) treatment for people with previously untreated, locally advanced unresectable or metastatic non-squamous non-small cell lung cancer (nSq NSCLC), did not meet its primary endpoints of progression-free survival (PFS) at its primary analysis with a hazard ratio (HR) of 1.27 [95% CI: 1.02,1.57] and overall survival (OS) at its first interim analysis with a HR of 1.33 [95% CI: 1.02, 1.73], which was immature. The combination of tiragolumab plus Tecentriq and chemotherapy showed reduced efficacy in both PFS and OS compared to the comparator arm in the intent-to-treat population, which includes Phase II and Phase III cohorts. The overall safety profile remains consistent with the safety profile previously observed for the combination of tiragolumab plus Tecentriq and chemotherapy, and no new or unexpected findings were identified. Based on these results, patients and investigators will be unblinded and we intend to halt the study. A communication will be sent to the investigators and results will be shared with health authorities and subsequently presented at an upcoming medical meeting. “These results are disappointing as it was our hope that this combination might yield improved outcomes for people living with metastatic non-squamous lung cancer,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We are thankful to all of the patients and healthcare professionals involved in the study, and we will leverage the learnings to inform our scientific understanding of the anti-TIGIT pathway and new avenues in cancer research.” Ongoing Phase III studies are investigating treatment settings and indications distinct from SKYSCRAPER-06. Based on today’s results, we will evaluate any relevant changes needed to the ongoing tiragolumab program. About SKYSCRAPER-06 study SKYSCRAPER-06 is a global Phase II/III, randomized, placebo-controlled and double-blinded study evaluating tiragolumab plus Tecentriq® (atezolizumab) and chemotherapy as an initial (first-line) treatment versus pembrolizumab and chemotherapy in 542 people with non-squamous non-small cell lung cancer. Primary endpoints are overall survival (OS) and progression-free survival (PFS). About tiragolumab Tiragolumab is an investigational novel immune checkpoint inhibitor with an intact Fc region. Tiragolumab selectively binds to TIGIT, a novel inhibitory immune checkpoint which suppresses the immune response to cancer. Based on preclinical research, tiragolumab is thought to work as an immune amplifier with other cancer immunotherapies such as Tecentriq® (atezolizumab). The TIGIT pathway is distinct but

1

A new class of incredibly predictive biomarkers is being advanced to the clinic by Kinomica as phosphoproteomics comes of age. A tour de force of multi-drug response demonstrations at this week's ASCO and ASMS conferences. Is this the future of #diagnostics? #startups #innovation #healthcare #venturecapital

14

Moving Medicine Forward Great news and an endorsement of excellent leadership with an implementation strategy in the growing and complex landscape of CDMO. Abzena specializes in difficult-to-express (DTE) proteins, monoclonal antibodies and related products from early discovery to clinical manufacture and commercial launch. We have deep technical expertise across multiple therapeutic modalities from traditional mabs to bifunctional and bispecifics, from ADCs to AOCs and a lot more. Reach out for more if you want to learn more about how Abzena can help to move your medicine forward. Stay healthy and well Abzena

13

Exciting times ahead in gene therapy! 🧬✨ Regeneron has just announced a colossal partnership with Mammoth Biosciences, spotlighting a promising future for CRISPR-based in vivo gene editing treatments. This dynamic duo is bringing together Regeneron's state-of-the-art viral vectors and Mammoth's cutting-edge compact nucleases to forge treatments with precision targeting. The deal includes a whopping $100 million upfront with an additional $370 million for milestones, plus royalties. That's a serious investment in next-gen healthcare. Dr. Christos Kyratsous from Regeneron cites years of groundwork laying the foundation for this collaboration, set to align the delivery systems with specific diseases for optimal outcomes. With equity involved and the option for Mammoth to co-fund and profit-share, the deal embodies a true symbiotic spirit. As the second major gene editing stride for Regeneron, after its ongoing work with Intellia Therapeutics, we're witnessing an exhilarating era where the potential for breakthrough therapies is skyrocketing. This is game-changing science with patient lives ready to be profoundly impacted! #GeneTherapy #CRISPR #Innovation #Healthcare

27

1 Comment

UGenome would love to meet you in San Diego from June 1-5! #ugenome, #ugenomeai, #pharmacogenomics, #pharmacogenetics, #bioinformatics, #genetics, #dna, #research, #genomics, #medicine, #drugdiscovery, #datascience, #biostatistics, #pharma, #genomicmedicine, #sequencing, #dataanalysis, #precisionmedicine

3

potential to target immunotherapies to affected tissues has substantial implications for benefit:risk calculations in ID, and in particular TB:

1

🚀 Biotech Q3 Scorecard: 19 Stock-Moving Events to Watch! 📊 Exciting times ahead in the biotech world! Here are key developments to keep on your radar: Alnylam Pharmaceuticals vutrisiran vs BridgeBio acoramidis for ATTR-CM: ESC presentation in August Novo Nordisk's monlunabant for obesity: Phase 2 results (watch Skye Bioscience Inc. and Corbus, LLC too!) Viridian Therapeutics, Inc.'s VRDN-001 for thyroid eye disease: Phase 3 readout in September Sage Therapeutics' Sage-324 for essential tremor: Mid-stage study results Apellis Pharmaceuticals' Empaveli for kidney diseases: Phase 3 VALIANT study results early Q3 Eli Lilly and Company's bimagrumab for obesity: Phase 2 results mid-year UCB and Biogen's dapirolizumab for lupus: Phase 3 results Neurocrine Biosciences's NBI-1117568 for schizophrenia: Phase 2 results Elevation Oncology's EO-3021 for gastric/pancreatic cancers: Data update Plus: • AstraZeneca's Imfinzi FDA panel (July 25) • Potential approvals for Alzheimer's drugs: Eisai US/Biogen's Leqembi (EU) • Multiple FDA decisions including Gilead Sciences, Regeneron, Syndax Pharmaceuticals, Roche, and Bristol Myers Squibb 🔬 The biotech sector is buzzing with potential breakthroughs. Which company or development are you most excited about? Share your thoughts below! 👇 #Biotech #PharmaTrends #ClinicalTrials #DrugDevelopment

5

Great news that Gilead’s PURPOSE 1 trial has demonstrated 100% efficacy of Lenacapavir, a twice-yearly injectable, in preventing new HIV infections among cisgender women and adolescent girls. These results are especially significant given adolescent girls and young women are particularly vulnerable to HIV. Gilead expects results in late 2024/early 2025 from the program’s other pivotal trial which is assessing twice-yearly Lenacapavir for PrEP among cisgender men who have sex with men, transgender men, transgender women and gender non-binary individuals who have sex with partners assigned male at birth in Argentina, Brazil, Mexico, Peru, South Africa, Thailand and the United States - groups who are underrepresented in HIV prevention trials and are disproportionately affected by the HIV epidemic. https://lnkd.in/e7s-d6f4

5

1 Comment

Congratulations and good luck to Sanket Shah for launching Otsuka Precision Health, Inc. (OPH). With FDA's new PDURS framework, we are confident a playbook will evolve for molecules and digital medicine to co-exist. It's hard to dismantle molecules from digital medicine. Keeping that in mind, we always conducted our clinical trials to show how JOGO Health + pills provide better outcomes. In one of the clinical trials, we demonstrated that adding JOGO to migraine medications improved MIDAS(Migraine Disability Assessment) score significantly. Another trial demonstrated JOGO's foundational science reduced side effects of cancer medications and medication discontinuation. pill + bytes is where digital medicine is heading.

29

Are Disease-Modifying Treatments on the Horizon for Patients With #ARVC? Gene therapy targeting the underlying desmosomal mutation and approaches to prevent myocardial fibrosis independent of the specific genetic defect by increasing cardiac levels of FGF21 may be on the horizon for patients. https://okt.to/mOF1sy

4

Our joint G2 Intelligence / Today's Clinical Lab webinar on the FDA's final LDTs rule is just over 24 hours away! I recently chatted via email with one of our speakers, regulatory expert Shannon Bennett, to get his initial thoughts on the final rule. Here's what he had to say about what was most surprising about the rule, what labs may find most challenging, and what labs should do right away to prepare: https://lnkd.in/gw3WuQjM #LDTs #FDA #ClinicalLaboratory #MedicalLaboratory

10

🚀 An exemplary demonstration of the effectiveness of utilizing AI-driven multimodal data analytics and modeling to enhance patient outcomes and provide personalized healthcare. This methodology offers advantages across the entire healthcare spectrum: - Patients experience improved diagnostics and personalized medical care. - Healthcare professionals benefit from more precise outputs, aiding in targeted therapy strategies. - The pharmaceutical industry sees reduced clinical development risks through the application of appropriate inclusion/exclusion criteria and optimized market access strategies. ☕ If you're keen on learning more, I'd be delighted to arrange a meeting or catch up at ASCO! #precisionmedicine #targettedtherapy #AIinHealthcare #AIMultimodal

11

When it comes to clinical trials, we know every day saved in development and enrollment timelines is a day closer to bringing new treatments to patients. Labcorp’s diagnostic data empowers your team to make informed decisions that can enhance trial design and recruitment. From identifying the best high-volume sites for your study to accelerating the rate of enrollment, our data provides the answers to drive your health innovation forward. Learn more about Labcorp’s clinical trial enablement services. #ClinicalTrials #ClinicalResearch #ClinicalStudies #ClinicalTrialsDay

40

I'm excited to share that Breyanzi has achieved another significant milestone in the fight against cancer. The US Food and Drug Administration has approved Breyanzi (lisocabtagene maraleucel) for the treatment of relapsed or refractory Mantle Cell Lymphoma. This approval is a testament to the unwavering commitment not only from BMS and other companies developing advanced therapies but also regulatory agencies around the world for advancing innovative therapies that can make a meaningful difference in patients'. Breyanzi (lisocabtagene maraleucel) stands out as the only CAR T cell therapy approved by the FDA for four distinct subtypes of non-Hodgkin lymphoma, expanding the reach of this personalized therapy to a broader range of patients with B-cell malignancies. https://lnkd.in/gq3nRiYv

95

2 Comments

Exciting news in the field of oncology! Bristol Myers Squibb's drug, #Augtyro (repotrectinib), has received FDA accelerated approval for the treatment of solid tumors with NTRK gene fusions. This groundbreaking tyrosine kinase inhibitor has demonstrated remarkable efficacy in clinical trials, significantly shrinking tumors in patients with this specific genetic feature. Augtyro's approval marks a significant milestone in the fight against cancer, offering new hope for patients with NTRK-positive solid tumors. This drug provides an additional treatment option for those who have exhausted other avenues, bringing us closer to a future where cancer is managed effectively. Kudos to the team at Bristol Myers Squibb for their tireless efforts in developing this life-changing medication! Let's continue to push the boundaries of medical innovation and make a meaningful impact in the lives of cancer patients worldwide. https://lnkd.in/eaWPKcwW #oncology #cancerresearch #precisionmedicine #medicalinnovation #immunotherapy #BristolMyersSquibb #innovation

50

1 Comment