Daily Snap - 6. August 2025

Author

Joachim Eeckhout
August 06, 2025

Good morning! Sumitomo Pharma has applied to Japanese regulators for approval of a stem cell-based treatment for advanced Parkinson's disease, following a successful clinical trial. The therapy involves transplanting induced pluripotent stem (iPS) cells directly into patients' brains. Developed in collaboration with Kyoto University, the trial showed the procedure was safe, with no major adverse effects over two years and symptom improvement in four of the seven participants. A parallel clinical trial is underway in the U.S., and if approved, the treatment could mark a hopeful step forward for millions living with Parkinson's worldwide.

Enjoy today’s read!

—Joachim E.

SNIPPETS

What’s happening in biotech today?

🎯 mRNA focus: BioNTech is laying off 90 employees across its Maryland and Massachusetts sites as part of a strategic pipeline realignment focused on therapies with pan-tumor potential, including personalized mRNA cancer immunotherapies and the bispecific antibody BNT327, which is being co-developed with Bristol Myers Squibb. These layoffs follow a previous reduction of 63 roles linked to the discontinuation of an early-stage CAR-T program and a broader reassessment of U.S. operations. Meanwhile, BioNTech continues its $1.2 billion acquisition of CureVac and will see Chief Strategy Officer Ryan Richardson depart in September.

💥 Highs and lows: BioMarin has reported promising early results for BMN 333, a potential next-generation treatment for achondroplasia that could succeed its current therapy, Voxzogo, which is projected to generate up to $935 million in 2025. A phase 2/3 trial of BMN 333 will start in early 2026 and target a 2030 market launch. The drug may offer a best-in-class profile, potentially surpassing Voxzogo and competing therapies. On a more negative note, BioMarin also announced the end of development for BMN 390, a preclinical phenylketonuria (PKU) therapy once seen as a potential successor to its already approved drug Palynziq, after it failed to meet immunogenicity targets necessary for clinical advancement. Despite this setback, the company remains committed to PKU treatment, with other projects ongoing and a planned FDA submission to expand Palynziq’s use to adolescents.

🏃‍♂️ Obesity retreat: Pfizer has discontinued its final GLP-1 obesity candidate, PF-06954522, marking the third such program cut and further weakening its internal obesity strategy amid poor data and strong competition. Unlike prior GLP-1 candidates abandoned due to liver safety concerns, this decision stemmed from underwhelming phase 1 data and market considerations. Pfizer now relies on a single phase 2 GIPR antagonist and is exploring external opportunities to rebuild its position in the competitive obesity market. The company also ended development of its C. difficile vaccine PF-06425090 but is advancing a next-generation version with improved immune response. Additionally, Pfizer dropped two phase 1 assets targeting heart failure and solid tumors.

🤞Phase 3 hope: HMNC Brain Health's phase 2b trial of its depression candidate BH-200 (nelivaptan) failed to meet its primary endpoint in V1b-high patients, the target group identified through a proprietary genetic biomarker test. However, the German biotech observed stronger antidepressant responses in patients with lower peripheral vasopressin activity, prompting a shift in its precision psychiatry approach. Despite the setback, HMNC plans to discuss a phase 3 program with regulators as it continues efforts to develop BH-200 as a first-in-class, precision-guided antidepressant.

💸 Cash surge: Shattuck Labs' stock surged 40% following the announcement of a private placement financing expected to raise up to $103 million, led by OrbiMed and supported by new and existing investors. The proceeds will fund clinical development of SL-325, a potentially first-in-class DR3 blocking antibody targeting Crohn’s disease, ulcerative colitis, and other autoimmune conditions. Subject to IND clearance, enrollment for a Phase 1 trial of SL-325 is set to begin this quarter, with funding projected to support operations into 2029.

SPEED READ

More news

  • President Donald Trump has escalated his push for domestic pharmaceutical manufacturing by proposing a phased tariff on imported drugs that would eventually reach 250%, up from an earlier proposed ceiling of 200%. In a CNBC interview, Trump stated the tariff would start at a lower rate and increase over 12 to 18 months, with an official announcement expected soon.

  • Beam Therapeutics delayed its key AATD trial data to early 2026, citing promising but undisclosed results and shifting to a two-dose gene-editing treatment strategy.

  • Beijing-based METiS Technologies raised $55.6 million in Series D funding to advance its AI-powered nanodelivery platforms, expand partnerships, and accelerate innovation in cell and gene therapy.

  • Facing dwindling cash reserves and shifting priorities, Imunon has shelved its COVID-19 vaccine program and is seeking a development partner while focusing on cancer trials.

  • MetaVia and Syntekabio have partnered to enhance DA-1241's therapeutic potential after promising Phase IIa results showed safety and efficacy in treating MASH-related metabolic dysfunction.

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