Category Reports Biotech

Achondroplasia already has one FDA-approved therapy on the market (BioMarin’s Voxzogo), so TransCon CNP is a “second entrant” story. That means the regulatory decision matters, but so will positioning, differentiation, and adoption post-approval.

Daily Hit on Ocular Therapeutix after the latest spike on renewed takeover chatter and ahead of the 52-week phase 3 SOL-1 readout for AXPAXLI (suprachoroidal axitinib). Cash-heavy balance sheet, a single pivotal asset at the centre of the story, and a market now trying to price both M&A optionality and binary clinical risk.

Deep-dive update on ImmunityBio after the Saudi Food and Drug Authority granted approval to ANKTIVA + BCG for BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, on top of existing approvals in the U.S., U.K. and conditional approval in the EU. Focus on the non-muscle invasive bladder cancer franchise, commercial trajectory, balance sheet and the next catalysts around QUILT-3.032 and QUILT-2.005.

Short update on VK2735 after the Phase 2 VENTURE data were published in the journal Obesity and after CEO Brian Lian’s comments at the J.P. Morgan Healthcare Conference 2026. For the full story, fundamentals and detailed scenarios, see the main VKTX report on Merlintrader.

NRx has licensed a nationwide Real-World Evidence dataset from Osmind covering more than 70,000 patients treated with IV ketamine or intranasal esketamine for major depression with suicidal ideation. The full analysis will be submitted to the FDA in support of an Accelerated Approval application for NRX-100, the preservative-free ketamine IV formulation already under Fast Track Designation for suicidal ideation in depression and bipolar depression.

Travere confirmed that the PDUFA date for the FILSPARI sNDA in FSGS is January 13, 2026. In the latest corporate update (press release) and at the JPM conference, management explained that the FDA recently sent a series of additional information requests “to further characterize the clinical benefit of FILSPARI” and that Travere has now submitted complete responses, which are under review.

In its January 13, 2026 press release, BriaCell announced the durable and sustained complete resolution of a lung metastasis in a patient treated with Bria-OTS™ (off-the-shelf personalized immunotherapy). The release describes a 78-year-old woman with metastatic breast cancer who achieved complete resolution after four doses of Bria-OTS monotherapy, with confirmation out to 11 months.

Focus: FDA approval of Zycubo (copper histidinate, ex CUTX-101) as first treatment for Menkes disease in children – monetisation of milestones, royalties and potential PRV for Fortress/Cyprium.

Today is about one thing: uncertainty around the FDA decision timing/outcome for the FILSPARI FSGS sNDA. The company explicitly highlighted FDA information requests right before the target date.

Tenax Therapeutics (TENX) – Deep Dive / Analisi Approfondita (Jan 2023 – Oggi) Tenax Therapeutics (TENX) – Deep Dive / Analisi Approfondita (Jan 2023 – Oggi) Full deep dive on TENX: financials, pipeline, catalysts, shareholder structure, insider activity, dilution, analyst…

Precision oncology company built around synthetic lethality, with darovasertib in uveal melanoma and a broad pipeline (MAT2A, PRMT5, PARG, ADCs) backed by more than 1.1 billion dollars in cash and a guided runway into 2030.

Yesterday’s report framed ATRA around a PDUFA-window setup, with the assumption that the 2025 CRL was primarily a CMC/GMP story and that the ALLELE trial remained acceptable as the pivotal efficacy dataset. Today that premise is broken: the FDA has issued a second Complete Response Letter (CRL) for tabelecleucel/tab-cel in EBV+ PTLD.