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What’s in Today’s Brief? (February 17th Preview)
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FDA says no to Disc’s bitopertin: voucher program faces its first major test
The FDA has declined to approve Disc Medicine’s bitopertin for erythropoietic protoporphyria (EPP), issuing a complete response that questioned whether the drug’s surrogate biomarker reliably predicts clinical benefit. The agency flagged the modest percent reduction in whole-blood metal-free protoporphyrin IX (PPIX) and requested an adequate, well-controlled study using clinical endpoints. Disc has publicly said the concerns are addressable and is already running the Phase 3 APOLLO study, which will include sunlight-exposure clinical endpoints in addition to PPIX. The rejection is notable because bitopertin had been selected for an accelerated review voucher, underscoring that expedited programs do not guarantee approval. Market reaction was swift: shares fell after the CRL and analysts questioned how the agency weighed biomarker changes versus patient-centered outcomes. The FDA’s letter reinforces scrutiny of surrogate endpoints and signals potential higher evidentiary bars for rare-disease approvals moving forward.
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Compass posts second pivotal win – psilocybin approval push intensifies
Compass Pathways reported a second successful Phase 3 study for its COMP360 psilocybin formulation in treatment‑resistant depression, prompting management to pursue regulatory filings. The company disclosed positive pivotal outcomes across two trials and signaled plans to engage regulators on a path to approval. Investors reacted strongly to the topline results, lifting the stock and sparking discussion about COMP360 becoming the first regulated psilocybin product in major markets. Independent experts noted the effect sizes likely meet approval thresholds but emphasized that detailed safety and durability data will determine commercial prospects. Compass now faces standard next steps: full data releases, agency interactions, and potential submission timing. The trials position the company to lead a nascent psychedelics approval wave should regulators accept the totality of evidence.
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Axpaxli edges out low‑dose Eylea – wet‑AMD durability data draw close scrutiny
Ocular Therapeutix reported Phase 3 results showing its Axpaxli formulation maintained vision longer than a low‑dose regimen of Regeneron’s Eylea, meeting the trial’s primary durability and maintenance endpoints. The trial showed higher proportions of patients maintaining vision at nine months and one year with a single injection of Axpaxli versus the low‑dose comparator. Topline figures released by the company and reported by STAT indicate 74% vs 56% at nine months and 66% vs 44% at one year (Axpaxli vs low‑dose Eylea). Analysts and clinicians noted the superiority versus a low‑dose active control but warned that comparisons to full‑dose standard of care and commercial positioning will determine market impact. Ocular will publish detailed data and engage regulators; the company and investors will assess whether the durability gains translate into a competitive commercial profile in a crowded wet‑AMD market.
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FDA clears Lex’s Velo POC PCR and grants CLIA waiver – multiplex respiratory test cleared
Lex Diagnostics received 510(k) clearance and a CLIA waiver for its Velo point‑of‑care molecular system and an influenza A/B plus COVID‑19 assay. The cartridge‑based system delivers multiplex ultra‑fast PCR results from swabs in roughly six to 10 minutes, positioning it for primary care, urgent care, pharmacies, and decentralized acute settings. Lex expects U.S. commercial activities to begin this year; the company noted QuidelOrtho’s prior investment and potential acquisition terms tied to FDA clearance. Lex’s CEO framed the approval as a step to deliver lab‑quality molecular results at the point of care and accelerate clinical decision making. The clearance adds to a wave of rapid molecular diagnostics approvals and could reshape respiratory testing workflows if rollout scales as projected.
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Watchmaker licenses Caribou’s CRISPR‑Cas9 to solve NGS bottlenecks
Watchmaker Genomics secured a non‑exclusive license to foundational CRISPR‑Cas9 intellectual property from Caribou Biosciences to use the nuclease as a programmable binding tool for next‑generation sequencing (NGS) library normalization. The company plans to apply CRISPR guides to stoichiometrically bind and normalize sequencing‑ready libraries without destructive processing. Watchmaker said the approach addresses normalization bottlenecks that limit throughput and that the method integrates with existing adapters and workflows. The firm also plans to adapt CRISPR for a PCR‑free whole‑genome sequencing method—recently patented by Watchmaker—to reduce DNA input and simplify large‑scale genomics use cases such as newborn screening and population studies. The license signals growing cross‑pollination between gene‑editing platforms and sequencing workflows, with potential operational benefits for high‑throughput genomics providers.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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