SNAPSHOT

Belite Bio zooms in on FDA approval as Stargardt drug hits late-stage milestone

Belite Bio’s drug tinlarebant showed strong results in a Phase 3 trial for Stargardt disease, cutting lesion growth by ~36% and setting up a 2026 approval push.

Why it matters: Tinlarebant could become the first approved treatment for Stargardt disease, a rare genetic eye disorder affecting 50,000 Americans with no current therapies.

Backstory: Stargardt disease disrupts vitamin A processing in the retina, causing toxic buildup and progressive vision loss. Tinlarebant, an oral drug, aims to block this process. Originally studied at Columbia University, it was licensed by Belite in 2018 and fast-tracked through development.

Big picture: Belite’s rise, from a $36M IPO in 2022 to a ~$5B valuation today, shows how rare disease breakthroughs can transform biotech fortunes. Tinlarebant is also being tested for geographic atrophy, potentially widening its impact.

Zoom in: The 2-year trial met its main goal of reducing retinal lesion growth by 36% over the placebo despite visual acuity remaining mostly unchanged. Side effects were mostly mild (night vision issues, color deficiency), with only four dropouts.

What’s next: Belite will meet regulators in early 2026 to pursue global approval. China’s drug agency is already reviewing an application based on interim results. Analysts predict that if all goes according to plan, peak sales could hit $1.4B–$3.6B.

Yes, but: Belite may face competition from Alkeus Pharmaceuticals, which is developing a similar therapy, and other companies such as Ocugen, AAVantgarde, and Splice Bio, which are developing gene therapies. Experts say gene therapy may dominate long term, but Belite’s drug could see rapid early uptake due to the current treatment void.

SNIPPETS

What’s happening in biotech today?

💰Big bucks: Protego Biopharma has raised $130 million in a Series B funding round to advance its lead drug candidate, PROT-001, for amyloid light-chain (AL) amyloidosis into late-stage clinical trials. Unlike current treatments that aim to eliminate misfolded proteins or the plasma cells producing them, PROT-001 works by stabilizing the light-chain proteins before they misfold and accumulate, potentially addressing disease progression at its source. Backed by major investors including Novartis Venture Fund and Forbion, Protego's approach may extend to other protein misfolding diseases. Early clinical data is expected in 2026.

🧬 Gene writing: Regeneron has invested $150 million in Tessera Therapeutics through an upfront payment and equity stake to co-develop TSRA-196, a gene-writing therapy targeting alpha-1 antitrypsin deficiency (AATD), a rare genetic disease with no approved treatments. The partnership includes a potential $125 million in milestone payments, with Tessera leading initial clinical trials and Regeneron overseeing later-stage development and commercialization. TSRA-196 has shown promising preclinical results with precise liver targeting and no off-target effects. Tessera’s RNA-based gene-writing platform, combined with non-viral delivery, aims to correct genetic mutations at their source, offering a potential one-time treatment for AATD and similar monogenic diseases.

🤝VC tag-team: Angelini Ventures and the European Investment Bank (EIB) have launched a €150 million co-investment initiative to support European biotechnology, medical technology, and digital health start-ups over the next six years. This marks the EIB’s first collaboration with a corporate venture capital firm in the health sector under its TechEU programme, which aims to boost Europe’s innovation capacity with €70 billion in investments between 2025 and 2027. The partnership will span seven to ten funding rounds and begin with an investment in French ADC specialist Adcytherix.

🛍️ Cyber Monday deal: Akebia Therapeutics has acquired Q32 Bio’s complement inhibitor ADX-097 for $12 million, $7 million upfront and $5 million in guaranteed payments, as the focal point of its emerging rare kidney disease pipeline. The deal, which includes up to $580 million in potential development, regulatory, and commercial milestones, follows Q32’s decision to halt a phase 2 renal trial and seek strategic alternatives for its tissue-targeted complement inhibitor platform. Akebia plans to initiate a phase 2 basket trial of ADX-097 in multiple rare kidney conditions next year, alongside a separate phase 2 study of praliciguat. The sale extends Q32’s cash runway into late 2027.

🧫 Liver lab: Morphocell Technologies has closed a $50 million Series A funding round, including a recent $10 million extension led by Investissement Québec and Italy’s CDP Venture Capital. The Canadian biotech now has over three years of operating runway to advance its lead engineered-tissue therapy, ReLiver, toward first-in-human trials. With 44 employees across Montréal, Boston, and Toronto, the company is targeting the growing liver disease market, valued at $20.3 billion in 2024 and projected to surpass $40 billion by the early 2030s. Morphocell aims to address the persistent need for alternatives to liver transplantation amid ongoing organ shortages.

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