National Cancer Institute

PureTech Health unveiled positive Phase 2b data on Monday for its idiopathic pulmonary fibrosis drug, which showed it can reduce lung function decline in patients. The drug hit the trial’s primary and secondary endpoints, with deupirfenidone showing superiority to placebo in slowing the rate of lung function decline in people with IPF as measured by forced vital capacity — defined as the amount of air a person can exhale after taking a breath in — at 26 weeks. Patients taking the investigational drug at 825 mg three times a day saw an 80.9% treatment effect. In contrast, patients taking 801 mg of pirfenidone, a standard-of-care for IPF, saw a 54.1% effect. In a secondary endpoint, there was a 99.6% probability that the pooled deupirfenidone arms of the trial were superior to placebo in slowing the rate of lung function decline when using a Bayesian analysis. Executives on a Monday investor call said that the results supported moving into a Phase 3 trial. CEO Bharatt Chowrira added on the call that based on the data, only one more trial may be necessary before the company seeks approval. PureTech’s stock $PRTC on the Nasdaq was up nearly 10% on Monday. Deupirfenidone, a deuterated form of pirfenidone, is an oral treatment that works by inhibiting IL-6, TNF-α and transforming growth factor beta, according to the National Cancer Institute. As for safety, the most common adverse events included gastrointestinal issues like nausea and dyspepsia, and the only “key” gastrointestinal adverse event seen was abdominal pain, with 14.1% of patients on the drug experiencing the issue. “We’ve looked into it,” PureTech’s VP of medical affairs Camilla Graham told investors. “There’s no signal, no cause for any concern, and we’re continuing to dig into all of these numbers.” There were discontinuations, with 187 out of 257 patients completing the trial: 42 out of 65 patients in the deupirfenidone 550 mg arm and 50 out of 64 patients in the 825 mg arm. Of the patients who completed the trial, 170 enrolled in the open-label extension portion of the trial. In response to a question about discontinuations from Endpoints News , a representative for the company said they “have just received these data and our primary focus has been assessing efficacy and the key GI adverse events,” adding that “there is nothing in the data to give us cause for concern.”

FRIDAY, Dec. 13, 2024 -- It's a decision no woman wants to have to make, but new research shows that young breast cancer patients with high-risk genes may be able to prevent their cancer from returning if they have their breasts or ovaries removed. Breast cancer patients aged 40 or younger with BRCA mutations had a 35% lower risk of death and 42% lower risk of cancer recurrence if they underwent a risk-reducing operation to remove their breasts, according to findings presented this week at the San Antonio Breast Cancer Symposium. Patients also had a 42% lower risk of death and 32% lower risk of recurrence following removal of their ovaries. “This global study provides the first evidence that risk-reducing surgeries improve survival outcomes among young BRCA-mutation carriers with a prior history of early-onset breast cancer,” said researcher Dr. Matteo Lambertini , an associate professor of medical oncology at the University of Genova-IRCCS Policlinico San Martino Hospital in Italy. “We believe that our findings are critical for improving the counseling of BRCA-mutation carriers with early-onset breast cancer on cancer-risk management strategies,” Lambertini added in a meeting news release. It’s been long known that women with BRCA1 and BRCA2 gene mutations are at higher risk for breast and ovarian cancer. More than 60% of women who inherit a BRCA mutation will develop breast cancer in their lifetimes, according to the National Cancer Institute (NCI). Likewise, as many as 58% of women with a BRCA1 mutation and 29% of women with a BRCA2 mutation will develop ovarian cancer, the NCI says. One strategy to lower these women’s risk of cancer is to remove their “at-risk” tissue – that is, to remove their breasts or ovaries. This strategy has been shown to help reduce risk in women with BRCA mutations who haven’t had cancer, but its effectiveness hasn’t been tested in young women who’ve already developed breast cancer, Lambertini said. The negative effects also must be considered, particularly when it comes to ovary removal, Lambertini noted. Ovary removal leads to infertility and early menopause. “Considering the unique traits and needs of this younger population, and their high risk for secondary malignancies, it is critical to understand how risk-reducing surgeries affect patient outcomes, so that the risks and benefits of these procedures can be carefully weighed,” Lambertini said. For the new study, researchers analyzed data on nearly 5,300 breast cancer patients with BRCA mutations treated at 109 hospitals across five continents. The women were all diagnosed with breast cancer at age 40 or younger between 2000 and 2020. Among those patients, nearly 3,900 underwent at least one risk-reducing surgery, including around more than 1,800 who had both their breasts and ovaries removed, researchers said. Results showed that women with BRCA1 mutations benefitted more than those with BRCA2 mutations, with their risk of death reduced by 56% compared with 15%. Because these findings were presented at a medical meeting, they should be considered preliminary until published in a peer-reviewed journal. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

DEERFIELD, Ill.--( BUSINESS WIRE )--TerSera Therapeutics LLC announced the presentation of new real-world analyses on the use of goserelin 3.6 mg and goserelin 10.8 mg in premenopausal women with breast cancer. These data were presented in a poster session at the 2024 San Antonio Breast Cancer Symposium, held December 10-13 in San Antonio, TX. 1 A copy of the poster is available here . This was a retrospective, observational, non-inferiority study using structured, de-identified, patient-level data and curated progress notes from U.S. electronic medical record (EMR) data from the ConcertAI Patient360™ Breast Cancer Dataset. The objective of this study was to compare real-world treatment outcomes among breast cancer patients treated with goserelin 3.6 mg and 10.8 mg. The primary outcome was the real-world event-free survival (rwEFS) rate at 12 months to assess the non-inferiority of the goserelin 10.8 mg dose given every 12 weeks vs. the 3.6 mg dose given every 4 weeks. The 12-month rwEFS rates for goserelin 3.6 mg and goserelin 10.8 mg, respectively, were 76.6% vs. 79.2% (treatment difference: 2.7% [95% CI: -1.8%, 7.0%]) which supports the non-inferiority of goserelin 10.8 mg to goserelin 3.6 mg at the prespecified -15% margin based on previously published oncology trials. In evaluating time to discontinuation, this study also found that more patients remained on goserelin 10.8 mg than 3.6 mg at years 1 and 2 post treatment initiation (51.2% vs. 39.6% and 32.4% vs. 23.1%, respectively), supporting previously published data in a separate real-world evidence analysis of breast cancer patients receiving goserelin 10.8 mg. 2 “Ovarian function suppression (OFS) with gonadotropin-releasing hormone agonists (GnRHas) is an essential component of therapy for a large proportion of pre- or perimenopausal women with hormone-sensitive breast cancer,” said Kelly E. McCann, M.D., Ph.D., assistant clinical professor of medicine at the UCLA Health David Geffen School of Medicine. “ZOLADEX 3.6 mg given every 4 weeks is the only FDA-approved GnRHa for women with breast cancer. Our data suggests that the 10.8 mg dose given every 12 weeks may also be an effective option.” U.S. INDICATIONS ZOLADEX 3.6 mg and ZOLADEX 10.8 mg are indicated for: Management of locally confined Stage T2b-T4 (Stage B2-C) carcinoma of the prostate in combination with flutamide. Treatment with ZOLADEX and flutamide should start 8 weeks prior to initiating radiation therapy and continue during radiation therapy. Palliative treatment of advanced carcinoma of the prostate. ZOLADEX 3.6 mg is also indicated for: Management of endometriosis, including pain relief and reduction of endometriotic lesions for the duration of therapy. Experience with ZOLADEX for the management of endometriosis has been limited to women 18 years of age and older treated for 6 months. Use as an endometrial-thinning agent prior to endometrial ablation for dysfunctional uterine bleeding. Palliative treatment of advanced breast cancer in pre- and perimenopausal women. IMPORTANT SAFETY INFORMATION Anaphylactic reactions to ZOLADEX have been reported in the medical literature. ZOLADEX is contraindicated in patients with a known hypersensitivity to GnRH, GnRH agonist analogues, or any of the components in ZOLADEX. ZOLADEX is contraindicated during pregnancy unless used for palliative treatment of advanced breast cancer. ZOLADEX can cause fetal harm when administered to a pregnant woman. If used during pregnancy, the patient should be apprised of the potential hazard to the fetus. There is an increased risk for pregnancy loss due to expected hormonal changes that occur with ZOLADEX treatment. ZOLADEX should not be given to women with undiagnosed abnormal vaginal bleeding. Pregnancy must be excluded for use in benign gynecological conditions. Women should be advised against becoming pregnant while taking ZOLADEX. Effective nonhormonal contraception must be used by all premenopausal women during ZOLADEX therapy and for 12 weeks following discontinuation of therapy. Transient worsening of tumor symptoms, or the occurrence of additional signs and symptoms of breast cancer, may occasionally develop during the first few weeks of treatment. Some patients may experience a temporary increase in bone pain. Monitor patients at risk for complications of tumor flare. Hyperglycemia and an increased risk of developing diabetes or worsening of glycemic control in patients with diabetes have been reported in men receiving GnRH agonists like ZOLADEX. Monitor blood glucose levels and glycosylated hemoglobin (HbA1c) periodically and manage according to current clinical practice. Increased risk of developing myocardial infarction, sudden cardiac death and stroke has been reported in association with use of GnRH agonists like ZOLADEX in men. Patients receiving a GnRH agonist should be monitored for symptoms and signs suggestive of development of cardiovascular disease and be managed according to current clinical practice. Hypercalcemia has been reported in some breast cancer patients with bone metastases after starting treatment with ZOLADEX. If hypercalcemia does occur, appropriate treatment measures should be initiated. Hypersensitivity, antibody formation and acute anaphylactic reactions have been reported with GnRH agonist analogues. ZOLADEX may cause an increase in cervical resistance. Therefore, caution is recommended when dilating the cervix for endometrial ablation. GnRH agonists may prolong the QT/QTc interval. Providers should consider whether the benefits of androgen deprivation therapy outweigh the potential risks in patients with congenital long QT syndrome, congestive heart failure, frequent electrolyte abnormalities, and in patients taking drugs known to prolong the QT interval. Electrolyte abnormalities should be corrected. Consider periodic monitoring of electrocardiograms and electrolytes. Injection site injury and vascular injury including pain, hematoma, hemorrhage and hemorrhagic shock, requiring blood transfusions and surgical intervention, have been reported with ZOLADEX. Extra care should be taken when administering ZOLADEX to patients with low BMI and/or to patients receiving full dose anticoagulation. Depression may occur or worsen in women receiving GnRH agonists. Treatment with ZOLADEX may be associated with a reduction in bone mineral density over the course of treatment. Data suggest a possibility of partial reversibility. In women, current available data suggest that recovery of bone loss occurs on cessation of therapy in the majority of patients. In women, the most frequently reported adverse reactions were related to hypoestrogenism. The adverse reaction profile was similar for women treated for breast cancer, dysfunctional uterine bleeding, and endometriosis. The most commonly reported adverse reactions with ZOLADEX in clinical trials for endometriosis were: hot flashes (96%), vaginitis (75%), headache (75%), decreased libido (61%), emotional lability (60%), depression (54%), sweating (45%), acne (42%), breast atrophy (33%), seborrhea (26%), and peripheral edema (21%). The most commonly reported adverse reactions with ZOLADEX in clinical trials for endometrial thinning were: vasodilation/hot flashes (57%), headache (32%), sweating (16%), and abdominal pain (11%). The most commonly reported adverse reactions with ZOLADEX in breast cancer clinical trials were hot flashes (70%), decreased libido (47.7%), tumor flare (23%), nausea (11%), edema (5%), and malaise/fatigue/lethargy (5%). Injection site reactions were reported in less than 1% of patients. For ZOLADEX 3.6 mg: Hot flashes (62%), sexual dysfunction (21%), decreased erections (18%), lower urinary tract symptoms (13%), lethargy (8%), pain (worsened in the first 30 days) (8%), edema (7%), upper respiratory infection (7%), rash (6%), and sweating (6%). For ZOLADEX 10.8 mg: Hot flashes (64%), pain (general) (14%), gynecomastia (8%), pelvic pain (6%), and bone pain (6%). In the locally advanced carcinoma of the prostate clinical trial, additional adverse event data were collected for the combination therapy with radiation group during both the hormonal treatment and hormonal treatment plus radiation phases of this study. Adverse experiences (incidence >5%) in both phases of this study were hot flashes (46%), diarrhea (40%), nausea (9%), and skin rash (8%). Treatment with ZOLADEX and flutamide did not add substantially to the toxicity of radiation treatment alone. Please see Full Prescribing Information for ZOLADEX 3.6 mg and ZOLADEX 10.8 mg . You are encouraged to report negative side effects of prescription drugs to the FDA. Visit fda.gov/safety/medwatch or call 1-800-FDA-1088. You can also contact TerSera Therapeutics at 1-844-334-4035 or medicalInformation@tersera.com . About HR-positive breast cancer Breast cancer is the second most commonly diagnosed cancer and one of the leading causes of cancer-related deaths worldwide. 3 In the United States, over 310,000 women will be diagnosed with breast cancer this year; 40,000 will be under the age of 50. 4 Approximately 75% of diagnosed cases in women under age 50 are considered to be hormone positive (HR+) breast cancer. Compared to older women, young women generally face more aggressive cancers and lower survival rates. 5-7 Recent studies have shown that breast cancer before age 40 differs biologically from the cancer faced by older women. 6,7 About ZOLADEX® (goserelin implant) ZOLADEX is an injectable luteinizing hormone-releasing hormone agonist (LHRHa) used to treat prostate cancer, breast cancer, and certain benign gynecological disorders. First approved in the U.S. in 1989, ZOLADEX is available as a 3.6 mg implant dosed every 28 days or as a 10.8 mg implant dosed every 12 weeks. Worldwide, ZOLADEX 3.6 mg is approved for use in breast cancer in 125 countries. ZOLADEX 10.8 mg is approved for use in breast cancer in over 60 countries. About TerSera Therapeutics TerSera Therapeutics is a biopharmaceutical company with a focus in oncology and non-opioid pain management. Founded in 2016, TerSera is building new cornerstones of care through its portfolio of unique therapeutics, amplifying their ability to deliver meaningful outcomes for patients. TerSera has been recognized as a 2024 Top Workplace. For additional information, please visit TerSera.com and follow us on LinkedIn . References McCann KE, Osman N, Cannon, J, et al. Real-world outcomes comparing 3-monthly with monthly goserelin implant in premenopausal women with breast cancer. Poster presented at: San Antonio Breast Cancer Symposium (SABCS); December 10-13, 2024; San Antonio, TX. Abstract #SESS-672; Poster ID: P1-08-09. McCann KE, Kaklamani V, Osman N, et al. Treatment patterns of goserelin 3.6 mg once monthly and 10.8 mg every three months in women with breast cancer: a real-world analysis. Poster presented at: American Society of Clinical Oncology (ASCO) Quality Care Symposium; September 27-28, 2024; San Francisco, CA. Abstract #391; Poster Session B Poster F19. Bray F, Laversanne M, Sung H, et al. Global cancer statistics 2022: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin . 2024;74(3):222-263. doi: 10.3322/caac.21834. National Cancer Institute. Surveillance, Epidemiology and End Results Program. Cancer stat facts: female breast cancer subtypes. https://seer.cancer.gov/statfacts/html/breast-subtypes.html . Accessed December 5, 2024. Varella L, Rosenberg S. Breast cancer disparities related to young age at diagnosis. Curr Breast Cancer Rep. 2022;14(3):174-179. doi:10.1007/s12609-022-00459-x. Tesch ME, Partridge AH. Treatment of breast cancer in young adults. Am Soc Clin Oncol Educ Book. 2022;42:795-803. doi:10.1200/EDBK_360970. Guzmán-Arocho YD, Rosenberg SM, Garber JE, et al. Clinicopathological features and BRCA1 and BRCA2 mutation status in a prospective cohort of young women with breast cancer. Br J Cancer . 2022;126(2):302-309. doi:10.1038/s41416-021-01597-2 . ZOLADEX® is a registered trademark of AstraZeneca or its affiliates and is used herein under license. ©2024 TerSera Therapeutics LLC. All rights reserved.