Portfolio Update: The floor plan takes shape for the Trump-RFK Biopharma agenda
Good news first. Actuate Therapeutics, Inc. (NASDAQ:ACTU) said on May 6 its drug to treat
pancreatic ductal adenocarcinoma (mPDAC), elraglusib, demonstrated statistically significant improvement in median overall survival in a phase II trial. The topline results compared the most recent measurable survival benefit in the elraglusib/GnP combination arm to results disclosed in December 2024.
Actuate plans to present the full results on May 31, 2025, at the annual meeting of the American Society of Clinical Oncology (ASCO). Actuate designed elraglusib to enter cancer cells and block the function of glycogen synthase kinase-3 (GSK-3). GSK-3β is highly expressed in pancreatic cancer and appears to cause tumor cells to resist conventional cancer therapy.
When we issued a report on Actuate’s trial in September 2024 the company’s stock traded at $8.15 per share.
Actuate enters public market with front-line treatment for metastatic pancreatic cancer | $ACTU
Physicians unfortunately diagnose pancreatic ductal adenocarcinoma patients only when they express metastatic disease (mPDAC). As a result, advancing new mPDAC treatments remains a high priority for drug developers and mPDAC families. The 5-year survival rate in mPDAC is less than 10%.
Acutate shares are currently trading at about $10
Moderna, Inc. (NASDAQ:MRNA) did not experience the same result as Actuate, both this week and during the first months of 2025. The skepticism by Robert F. Kennedy, Jr., appointed by President Trump as Health & Human Services Secretary, toward vaccines was well known at the time of his confirmation. The stocks of vaccine manufacturers then fell sharply during early 2025 as investors foresaw headwinds for the expansion of existing vaccines and the introduction of new vaccines.
Those concerns were amplified this week with the appointment of Vinay Prasad as the new director of the Center for Biologics Evaluation and Research (CBER). Prasad, recently a professor of epidemiology and biostatistics at the University of California at San Francisco (UCSF), was a critic of social distancing recommendations during the Covid19 period, selected vaccine mandates, and also urged caution in directing Covid19 vaccines in children.
The Prasad appointment does not bode well for Moderna, which likely plans to release an updated Covid19 vaccine in the fall. The CBER could adjust the requirements for an updated vaccine, which would result in additional cost and a potential delay for Moderna and other vaccine manufacturers.
Moderna’s financial guidance early in 2025 did not meet investors’ expectations, adding to the company’s stock slide. On May 6, Moderna and Vertex Pharmaceuticals (NASDAQ:VRTX) paused a phase I/II trial testing the companies’ mRNA-3692/VX-522 drug as a treatment for a CFTR genotype not responsive to CFTR modulator therapy. Success of the drug, for which Moderna provided mRNA technology, would have lessened Moderna’s dependence on vaccine revenue. The trial pause, however, signals the drug will not advance on schedule. Though the pause is considered temporary, Vertex did not provide any guidance on when it would resume.
We issued a report on the phase I/II cystic fibrosis trial in January 2025, when Moderna’s stock traded at $47.53 per share.
Company Seeks to Expand mRNA’s Reach in Cystic Fibrosis: A Path Toward New Treatment Possibilities
Today's report is on a biotechnology firm’s messenger RNA platform that, while originally developed for an innovative immunization, has also been adapted by a partner company to address cystic fibrosis. This approach seeks to deliver a key protein directly to the lungs, targeting individuals who currently lack effective treatment options. Over the years, the partner has introduced multiple therapies for this genetic disease, substantially benefiting a large portion of those affected. Despite initial hurdles using the messenger RNA delivery system, the new therapy showed enough promise to advance into an early clinical study, where there is measured optimism for positive results. Cystic fibrosis is an inherited condition that progressively impacts lung function and can also affect the pancreas and other organs.
Moderna shares are currently trading near $24.
Accordingly, we are discontinuing coverage and closing its positions in ACTU and MRNA.
Hi Bill,
Would love your perspective on eXoZymes Inc.
https://www.slack-capital.com/p/exozymes-research-report