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FDA outlines plans to phase out required animal testing in drugs

The FDA announced a new roadmap to reduce and potentially replace required animal testing for monoclonal antibodies and other drugs. The FDA stated that companies that use non-animal testing like AI-based computational methods and organ-on-a-chip systems may receive a streamlined review. FDA Commissioner Martin Makary said in the press release, “This initiative marks a paradigm shift in drug evaluation and holds promise to accelerate cures and meaningful treatments for Americans while reducing animal use.” Some biotech scientists and public health researchers welcomed the changes, including scientist and attorney Paul Locke of Johns Hopkins University, who told CNN that it was a “really important step.” Yet, others pointed to the current lack of evidence for non-animal methods. Sergiu Pasca, one of the leading pioneers in the brain organoid field, told Axios, "We just don't have enough information about how most of the [brain's] circuits are functioning, how some of the genes are expressed, where they're expressed, and how they're expressed dynamically in diseases." In a statement released by the National Association for Biomedical Research, President Matthew Bailey said, “No AI model or simulation has yet demonstrated the ability to fully replicate all the unknowns about many full biological systems. That’s why humane animal research remains indispensable.” – Allison Whitten

Two Phase 1 trials show promise for stem cell therapy in Parkinson’s disease

This week, two Phase 1 clinical trials published in Nature showed that transplanting stem cell-derived dopamine-producing neurons into the brain could be a promising approach to treating Parkinson’s disease (PD):

1. In the first trial, scientists at Kyoto University Hospital implanted dopaminergic progenitor cells derived from allogeneic induced pluripotent stem cells into both sides of the brain in seven PD patients (1). The procedure caused no serious side effects, though it led to 73 mild-to-moderate events. The transplanted cells produced dopamine without forming tumors in all participants. Notably, five of six patients showed reduced motor symptoms while on their standard PD medication, and four improved even after stopping it, indicating that the grafted cells were functioning as dopamine-producing neurons.
2. In the second trial, researchers at Memorial Sloan Kettering Cancer Center treated 12 people with an off-the-shelf dopaminergic neuron progenitor cell product (bemdaneprocel) derived from a human embryonic stem cell line (2). One year after the procedure, they reported no serious side effects caused by the therapy. Patients who received a high dose showed a 50 percent reduction in PD symptom scores at 18 months compared to their baseline levels. Using brain scans, the researchers also confirmed that the transplanted cells survived and increased dopamine production in all successfully grafted participants. The research team would like to further assess the therapy’s efficacy in a larger cohort of PD patients. – Dika Ojiakor

Pig liver trial gets the go ahead

The FDA cleared biotech company eGenesis and medtech company OrganOx to begin a clinical trial of their gene-edited pig liver and blood filtration system for patients with acute-on-chronic liver failure (ACLF). The researchers will enroll up to 20 people with ACLF who don’t qualify for a liver transplant in the Phase 1 trial. Participants will be connected to a gene-edited pig liver that sits outside the body, and the OrganOx system will pump their blood through the pig liver for 72 hours. The researchers hope that this outside-the-body blood filtration system will give patients’ livers a chance to recover and potentially heal or that it will give future patients more time to receive a liver transplant. – Stephanie DeMarco

Progress and setbacks for oral GLP-1 drugs

On Thursday, Eli Lilly announced positive results with their oral GLP-1 drug, orforglipron, based on their ACHIEVE-1 Phase 3 trial — the first oral GLP-1 drug to make it that far. In a press release, they stated that in the trial of 559 adults with type 2 diabetes, their once-daily pill resulted in the lowering of A1C values by an average of 1.3 percent to 1.6 percent across doses (from a baseline of 8 percent), an average weight loss of 16 pounds at the highest dose, and an overall safety and tolerability profile similar to already approved injectable GLP-1 drugs. The ACHIEVE-1 trial is the first of seven Phase 3 trials from Eli Lilly testing orforglipron in people with type 2 diabetes and obesity. 

Earlier in the week, Pfizer announced that they will discontinue development of their oral GLP-1 drug, danuglipron, as “a single asymptomatic participant in one of the dose-optimization studies experienced potential drug-induced liver injury which resolved after discontinuation of danuglipron.” They were testing a once-daily version in Phase 1 trials and said that they now plan to continue their development of another oral drug targeting the gastric inhibitory polypeptide receptor (GIPR) instead. – Allison Whitten

Executive order aims to remove the “pill penalty” on drug pricing law

On Tuesday, President Trump signed an executive order with the aim to lower drug prices in the United States. In the order, he directed HHS Secretary, Robert F. Kennedy Jr. to work with Congress to remove the “pill penalty” in the 2022 Inflation Reduction Act (IRA). This refers to the part of the law that exempts small molecule drugs from Medicare price negotiations for only nine years while biologics are exempted for 13 years. The healthcare consultancy firm Vital Transformation reported in a preprint on medRxiv that investment in small molecule drug development decreased by 70 percent after the IRA passed (3). While removing the “pill penalty” will require Congressional action, drug industry analysts are hopeful, with Biospace reporting that many are “cautiously optimistic” and expect to see renewed investment in small molecule drug development. – Stephanie DeMarco

A new blood test could predict skin cancer recurrence

A new gene-based blood test for melanoma may help predict the cancer's recurrence significantly earlier than currently available methods. Stage III melanoma is an aggressive form of skin cancer in which tumor cells spread from the skin to nearby lymph nodes. After the affected lymph nodes are surgically removed, recurrence of the cancer can be hard to detect using common imaging methods. In a recent Phase 3 trial, researchers at NYU Langone Health found that approximately 80 percent of stage III melanoma patients who had detectable levels of circulating tumor DNA (ctDNA) — fragments of DNA shed from dying cancer cells — in their blood before they started tumor suppression treatment went on to experience recurrence (4). The cancer returned more than four times faster in this group compared to those with no detectable biomarker levels, and the higher their levels of ctDNA, the faster the recurrence. The new blood test may soon help doctors better identify which patients are likely to benefit from specific treatments and could become a standard tool in managing melanoma. – Dika Ojiakor

References

1. Sawamoto, N. et al. Phase I/II trial of iPS-cell-derived dopaminergic cells for Parkinson’s disease. Nature (2025).
2. Tabar, V. et al. Phase I trial of hES cell-derived dopaminergic neurons for Parkinson’s disease. Nature (2025).
3. Schulthess, D.G. et al. The Inflation Reduction Act’s Impact upon Early-stage Venture Capital Investments. Preprint at medRxiv. (2025).
4. Syeda, M.M. et al. Clinical validation of droplet digital PCR assays in detecting BRAF^V600-mutant circulating tumour DNA as a prognostic biomarker in patients with resected stage III melanoma receiving adjuvant therapy (COMBI-AD): a biomarker analysis from a double-blind, randomised phase 3 trial.  Lancet Oncol (2025).