Recent News

• Celcuity’s breast cancer combo hits primary endpoints, teeing up FDA filing
  Fierce Biotech – July 28, 2025
  A phase 3 trial of Celcuity’s breast cancer combination has hit its primary endpoints, blasting past the biotech’s bar for success to tee up a filing for FDA approval in the fourth quarter. The study tested doublet and triplet combinations of Celcuity’s gedatolisib, a PI3K and mTOR inhibitor, as second-line treatments for PIK3CA wild-type breast cancer. The doublet combined gedatolisib with fulvestrant, the breast cancer drug AstraZeneca sells as Faslodex. The triplet added palbociclib, which Pfizer sells as Ibrance, to the mix. A control arm received fulvestrant as a monotherapy. Celcuity said the triplet reduced the risk of disease progression or death by 76% compared to fulvestrant alone. The figure for the doublet was 67%. Progression-free survival was 9.3 months on the triplet and 7.4 months on the doublet, compared to two months in the control arm.
• Researcher uses light to target and kill cancer cells
  Medical Xpress – July 28, 2025
  A Northeastern University researcher has identified a way to target two of the deadliest cancer types, melanoma and triple negative breast cancer, with chemotherapy drugs but without the harms associated with chemotherapy. Both cancers are typically resistant to chemotherapy, says Fleury Augustin Nsole Biteghe, a lecturer in biotechnology, chemistry and chemical biology. But by attaching a light-sensitive drug to a protein called MTf—which appears abundantly in both cancers—and bathing the drug-infused protein in near-infrared light, cancer cells die. Using antibodies to target cancer proteins is typically performed by using multiple drugs at once, Nsole Biteghe says. But this approach stimulates the immune system so much that it can end up attacking healthy body tissues, he says. “The antibody is like a key and we know what the lock is,” he says.
• Stem cell transplant without toxic preparation successfully treats genetic disease
  Stanford Medicine – July 22, 2025
  An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a phase 1 clinical trial has shown. While the researchers tested the protocol on patients with Fanconi anemia, a genetic disease that makes standard stem cell transplant extremely risky, they expect it may also work for patients with other genetic diseases that require stem cell transplants. “We were able to treat these really fragile patients with a new, innovative regimen that allowed us to reduce the toxicity of the stem cell transplant protocol,” said the study’s co-senior author, Agnieszka Czechowicz, MD, PhD, assistant professor of pediatrics. “Specifically, we could eliminate the use of radiation and genotoxic chemotherapy called busulfan, with exceptional outcomes.”
• AstraZeneca blood pressure drug succeeds in late-stage trial
  BioPharma Dive – July 14, 2025
  An experimental drug being developed by AstraZeneca significantly reduced blood pressure versus placebo in a Phase 3 study of people with either uncontrolled or treatment-resistant hypertension, the pharmaceutical firm said. The reduction in mean seated systolic blood pressure associated with AstraZeneca’s drug was clinically meaningful, the company added. Called baxdrostat, the drug also met all of the study’s secondary endpoints and was “generally well tolerated.” The drug, which works by blocking an enzyme that synthesizes the hormone aldosterone, had mixed results in Phase 2 testing. But AstraZeneca saw enough promise to bet low on the drug succeeding in late-stage testing, particularly as it held potential for use in combination with the company’s diabetes and kidney disease drug Farxiga.
• Pfizer, Astellas boost case for Xtandi in early-stage prostate cancer with combo survival win
  Fierce Pharma – July 10, 2025
  Pfizer and Astellas are underlining the benefits of Xtandi as part of a combination therapy for certain patients with non-metastatic hormone-sensitive prostate cancer (nmHSPC) with an overall survival win. The new data come from the companies’ phase 3 Embark study, which in 2023 led to the FDA approval of Xtandi in patients with nmHSPC with biochemical recurrence (BCR) at high risk of metastasis. In the trial, Xtandi was studied as a single agent and in combination with leuprolide against placebo and leuprolide. For the trial’s primary endpoint, the Xtandi combo delivered a statistically significant improvement in metastasis-free survival compared with placebo and leuprolide. At the time, the trial’s overall survival data were mature. Now, Xtandi plus leuprolide has proved it can, in fact, extend the lives of these patients, demonstrating a “statistically significant and clinically meaningful improvement” in overall survival, Pfizer announced.
• New hope for patients with head and neck cancer
  UC Davis Health News – July 10, 2025
  Head and neck cancers are among the most difficult to treat. They can be aggressive and are located near some of the body’s most vital areas: nose, mouth, throat. Chemotherapy and radiation have been the primary weapons against tumors that cannot be surgically removed, but the results in advanced cancers have been poor. Immunotherapies have great potential to improve this landscape. These drugs recruit immune T cells to attack tumors — harnessing the body’s preexisting defense mechanisms to defeat cancer. Unfortunately, this approach is also a work in progress. But now, researchers at UC Davis Comprehensive Cancer Center have initiated an innovative clinical trial to provide better results against head and neck cancers. It combines the immunotherapy drug pembrolizumab, also known as KEYTRUDA, and a commonly prescribed blood pressure medication losartan, sold as COSAAR, with a new therapeutic radiation.
• Moderna COVID vaccine gets full approval for children
  BioPharma Dive – July 10, 2025
  COVID vaccines have faced increased scrutiny this year under Kennedy’s HHS leadership. Messenger RNA-based shots are especially in the spotlight, but even Novavax’s Nuvaxovid, a vaccine made with older technology, encountered delays. FDA commissioner Marty Makary and his deputy Vinay Prasad, who leads the FDA office overseeing vaccines, have put out a new, stricter vaccine framework that requires drugmakers gather additional data to win approval for booster shots in healthy adults and children. This new criteria could slow down development. Outside of the FDA, Kennedy last month abruptly fired all 17 members of an advisory panel to the CDC, replacing them with his own hand-selected advisers, who don’t all have the typical background and experience in epidemiology and vaccine science. This panel, known as ACIP for short, recommends who should receive FDA-approved vaccines. Its guidelines influence insurance coverage.
• Eli Lilly’s Kisunla wins FDA nod for new dosing with fewer side effects in Alzheimer’s
  Fierce Pharma – July 9, 2025
  The benefit-risk balance is tipping more in favor of Eli Lilly’s Kisunla in early Alzheimer’s disease as the FDA approves a new dosing regimen of the drug with an improved safety profile. The new recommended regimen for Kisunla features a more gradual titration and a significantly lower rate of brain swelling known as amyloid-related imaging abnormalities with edema and effusion (ARIA-E) when compared to the original regimen. ARIA is a class-wide side effect that has impeded broader adoption of anti-amyloid antibody drugs, including Kisunla, in early Alzheimer’s disease. “Anything we can do to advance the science in that space should have an impact on the benefit-risk discussions that clinicians are having with patients,” Brandy Matthews, M.D., Lilly’s VP of global & U.S. medical affairs for Alzheimer’s disease, said in an interview with Fierce Pharma.
• Novartis’ malaria drug for babies wins world-first approval
  Fierce Pharma – July 8, 2025
  Novartis and a Swiss nonprofit have made history, scoring the world’s first approval for a medicine to treat babies who are infected with malaria. Switzerland’s health regulator has signed off on Coartem Baby for infants who weigh between 2 and 5 kilograms (4.4 and 11 pounds) and have contracted the deadly mosquito-borne disease. Coartem Baby was endorsed by Swissmedic under a prearranged Marketing Authorization for Global Health Products (MAGHP) procedure, which will facilitate rapid approvals in eight African nations, Novartis said in a July 8 press release.  The drugmaker plans to introduce the treatment on a “largely not-for-profit basis to increase access in areas where malaria is endemic,” Novartis added. “For more than three decades, we have stayed the course in the fight against malaria, working relentlessly to deliver scientific breakthroughs where they are needed most,” Vas Narasimhan, Novartis’ CEO, said in the release.