Everolimus

FDA Approves Belzutifan as First Oral Treatment for Advanced Pheochromocytoma and Paraganglioma

8 days ago

• The FDA has approved belzutifan (Welireg) for adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma, based on promising phase 2 trial results. • As a selective HIF-2α inhibitor, belzutifan demonstrated a 26% overall response rate with a median duration of response of 20.4 months, offering a novel mechanism of action compared to historically used treatments. • This approval is particularly significant as previous treatment options were limited, with the only previously approved agent no longer commercially available and alternative therapies associated with concerning toxicity profiles.

Biocon Announces $150 Million Capital Expenditure Plan Amid Strong Q4 Performance

12 days ago

• Biocon plans to invest $150 million in capital expenditure over the next two years, with $100 million allocated to biologics expansion in Malaysia and $50 million to generics. • The biopharmaceutical company reported a 153% surge in Q4 net profit to Rs 344 crore, driven by strong growth in generics and steady progress in biosimilars. • Biocon is expanding its product portfolio with recent launches of lenalidomide and dasatinib in the US, liraglutide in the UK, and plans to launch bevacizumab and insulin aspart in the US market.

Sunshine Biopharma Launches Generic Everolimus in Canada's $2.5B Oncology Market

a month ago

• Sunshine Biopharma's Canadian subsidiary, Nora Pharma Inc., has introduced generic Everolimus to the Canadian market, targeting multiple cancer indications including advanced renal cell carcinoma and breast cancer. • The global Everolimus market, valued at $2.5 billion in 2023, is projected to reach $4.8 billion by 2032 with a CAGR of 7.1%, while the Canadian segment grows at 6.4% annually. • Sunshine Biopharma continues expanding its portfolio of 70 generic drugs with 15 additional launches planned for 2025, including NIOPEG®, a biosimilar of NEULASTA® for reducing infection in cancer patients.

Fruquintinib-Sintilimab Combination Meets Primary Endpoint in Phase II/III RCC Trial

2 months ago

• The FRUSICA-2 Phase II/III study evaluating fruquintinib plus sintilimab as second-line treatment for advanced renal cell carcinoma has met its primary endpoint of progression-free survival. • The combination therapy demonstrated improvements in secondary endpoints including objective response rate and duration of response, offering new hope for patients who have progressed on previous therapies. • HUTCHMED and Innovent Biologics plan to submit detailed findings to regulatory authorities and progress toward NDA filings in the coming months.

FDA Breakthrough Therapy Status Emerges as Game-Changer for Oncology Drug Development

3 months ago

• FDA's Breakthrough Therapy designation offers accelerated approval pathways for promising oncology drugs, potentially reducing development timelines to just 3 years and extending patent life. • The new designation has shown particular promise in oncology, with the majority of the first 10 breakthrough approvals being cancer drugs, demonstrating significant market advantages over traditional orphan drug status. • Companies like Pharmacyclics and Genmab have seen immediate positive market response, with stock prices jumping 10-13% following breakthrough designation announcements for their cancer drugs.

ITM-11 Demonstrates Superior Progression-Free Survival Compared to Everolimus in GEP-NETs

4 months ago

• ITM-11, a targeted radiopharmaceutical, significantly improved progression-free survival (PFS) in patients with Grade 1/2 gastroenteropancreatic neuroendocrine tumors (GEP-NETs). • The Phase 3 COMPETE trial marks the first time a radiopharmaceutical has shown superior PFS compared to everolimus in this patient population. • ITM plans to present the COMPETE data at an upcoming medical conference and discuss a potential New Drug Application (NDA) submission with the FDA in 2025. • The trial results support ITM-11's potential as a more effective treatment option for GEP-NETs, addressing a significant unmet need.

Cabozantinib Shows Promise in Advanced GI Neuroendocrine Tumors

4 months ago

• Cabozantinib (Cabometyx) significantly improved progression-free survival (PFS) in patients with advanced gastrointestinal neuroendocrine tumors (GI-NETs) after prior therapy. • The CABINET trial subgroup analysis revealed a median PFS of 8.5 months with cabozantinib versus 5.6 months with placebo in GI-NET patients. • Benefits were observed across various clinical factors, including tumor grade and prior treatment, except in non-midgut GI primary tumors. • The safety profile of cabozantinib was consistent with previous data, with manageable adverse events reported.

Afinitor Plus Somatuline Significantly Prolongs Survival in GEP-NETs

4 months ago

• Combination therapy with Afinitor and Somatuline demonstrates a significant improvement in progression-free survival (PFS) compared to Afinitor alone in patients with unresectable or recurrent GEP-NETs. • The phase 3 STARTER-NET trial reported a median PFS of 29.7 months in the Afinitor plus Somatuline group versus 11.5 months in the Afinitor monotherapy group. • The combination therapy showed an acceptable safety profile, with manageable side effects, positioning it as a potential new first-line treatment for GEP-NETs. • Subgroup analysis indicates that patients with a Ki-67 score above 10%, indicating more aggressive tumors, may particularly benefit from the Afinitor and Somatuline combination.

Everolimus Plus Lanreotide Significantly Extends PFS in GEP-NETs

4 months ago

• The combination of everolimus and lanreotide significantly prolonged progression-free survival (PFS) compared to everolimus alone in GEP-NETs. • Median PFS reached 29.7 months in the combination arm versus 11.5 months in the everolimus monotherapy arm, demonstrating a substantial improvement. • The objective response rate was significantly higher in the combination group (23.0%) compared to the everolimus group (8.3%). • The combination therapy showed a manageable safety profile, suggesting its potential as a new first-line treatment option.

Lerociclib Plus Fulvestrant Shows Promise in HR+/HER2- Advanced Breast Cancer

4 months ago

• The LEONARDA-1 phase 3 trial demonstrated that lerociclib plus fulvestrant significantly improved progression-free survival (PFS) compared to placebo plus fulvestrant in patients with HR+/HER2- advanced breast cancer. • The combination of lerociclib and fulvestrant showed a manageable safety profile, with hematological toxicities being the most common adverse events, effectively managed with dose adjustments. • Lerociclib plus fulvestrant exhibited efficacy across various patient subgroups, including those with primary endocrine therapy resistance and liver metastasis, addressing a critical unmet need. • The study supports lerociclib plus fulvestrant as a potential treatment option for patients with HR+/HER2- advanced breast cancer who have progressed on prior endocrine therapy.

Fruquintinib Plus Sintilimab Shows Promise in Advanced Renal Cell Carcinoma

4 months ago

• A Phase Ib/II trial of fruquintinib plus sintilimab demonstrates promising efficacy in both treatment-naive and previously treated advanced clear cell renal cell carcinoma (ccRCC) patients. • In treatment-naive patients, the combination therapy achieved a confirmed objective response rate (ORR) of 68.2% with an 18-month progression-free survival rate of 59.4%. • Previously treated patients experienced a 60.0% confirmed ORR and a median progression-free survival of 15.9 months with the fruquintinib and sintilimab regimen. • The combination was generally well-tolerated, with manageable adverse events, supporting further investigation in the ongoing Phase III FRUSICA-02 study.

Belzutifan Plus Cabozantinib Shows Promise as First-Line Treatment for Advanced Clear Cell Renal Cell Carcinoma

5 months ago

• The combination of belzutifan and cabozantinib demonstrates promising antitumor activity in treatment-naive patients with clear cell renal cell carcinoma (ccRCC). • In the LITESPARK-003 trial, the objective response rate was 70%, with a disease control rate of 98% at a median follow-up of 24.3 months. • The combination therapy showed manageable toxicity, with the most common grade 3-4 treatment-related adverse events being hypertension, anemia, and fatigue. • These findings support further randomized trials to evaluate belzutifan in combination with other tyrosine kinase inhibitor-based regimens for ccRCC.

Olema Oncology Secures $250M Funding and Novartis Partnership for Phase 3 Breast Cancer Trial

6 months ago

• Olema Oncology has secured a $250 million private placement to advance its clinical programs, including the Phase 3 OPERA-02 trial. • A collaboration with Novartis will provide ribociclib for the OPERA-02 trial, evaluating palazestrant in combination for ER+/HER2- metastatic breast cancer. • The OPERA-02 trial is expected to begin in mid-2025, with approximately 1,000 patients, and Olema retains global commercial rights to palazestrant. • Olema's funding supports multiple clinical milestones, including OPERA-01 Phase 3 data in 2026 and OP-3136 Phase 1/2 data, strengthening its development pipeline.

Real-World Data Suggests Elacestrant Offers Similar or Improved Outcomes in Advanced Breast Cancer

6 months ago

• A real-world analysis shows elacestrant demonstrates similar or slightly higher time to treatment discontinuation compared to the EMERALD trial. • The median real-world time to next treatment with elacestrant was 6.43 months, and time to treatment discontinuation was 4.6 months. • Patients with PIK3CA pathway alterations experienced worse outcomes with elacestrant, underscoring the need for precision medicine. • Most patients received elacestrant beyond the second line of treatment, with outcomes remaining consistent across different lines of therapy.

Novel Anti-Estrogen Therapies Expand Options for Advanced ER+ Breast Cancer

8 months ago

• Novel anti-estrogen therapies are poised to expand treatment options for patients with estrogen receptor (ER)-positive advanced breast cancer after CDK4/6 inhibitor therapy. • The phase 3 INAVO120 trial showed that inavolisib, combined with palbociclib and fulvestrant, significantly improved progression-free survival in patients with PIK3CA-mutated advanced breast cancer. • Updated INAVO120 findings presented at ASCO 2024 demonstrated a longer time to next-line treatment with the inavolisib combination compared to palbociclib and fulvestrant alone. • Researchers are focusing on identifying specific subgroups of patients who may benefit most from certain therapies or combinations, bringing a more personalized approach.

Optimizing Kidney Cancer Treatment: Keytruda, Inlyta, and Novel Approaches for Non-Clear Cell RCC

8 months ago

• Considerations for discontinuing Keytruda (pembrolizumab) and Inlyta (axitinib) after two years involve balancing recurrence risk and side effects, necessitating individualized patient-physician discussions. • For non-clear cell chromophobe renal cell carcinoma, IO-TKI combinations like Lenvima (lenvatinib) plus Keytruda or Cabometyx (cabozantinib) plus Opdivo (nivolumab) show promise, with treatment choice depending on approval status. • Managing fatigue and chronic kidney disease in RCC patients requires investigating reversible causes and employing supportive measures, with nephrologist involvement for significant renal impairment.

U.S. Tuberous Sclerosis Drug Market Projected to Reach $1.25 Billion by 2030, Growing at 23.1% CAGR

2 years ago

• The U.S. tuberous sclerosis drug market is expected to grow from $237.2 million in 2022 to $1.25 billion by 2030, driven by FDA approvals of new treatments and increasing disease prevalence. • FDA-approved treatments include Afinitor (everolimus) for patients aged 2 and above, approved in 2018, and Epidiolex (cannabidiol) for patients aged 1 and above, approved in 2020. • mTOR inhibitors are gaining significant traction in treatment protocols, showing effectiveness for internal tumors and skin lesions, with FDA approval for treating lung complications like Lymphangioleiomyomatosis (LAM) in 2015.

Novartis Agrees to $678M Settlement Over Doctor Kickback Scheme in US

5 years ago

• Novartis will pay $678 million to resolve allegations of using speaker programs and lavish entertainment to incentivize doctors to prescribe their cardiovascular and diabetes medications. • The company faces an additional $51 million settlement for illegally covering Medicare copayments for patients prescribed Gilenya and Afinitor. • As part of the settlement, Novartis commits to new corporate integrity measures through 2025, including limited use of paid external physicians and a shift toward digital engagement.

Palbociclib Plus Letrozole Approved for First-Line ER+, HER2- Metastatic Breast Cancer

10 years ago

• Palbociclib, a CDK4/6 inhibitor, received FDA accelerated approval in combination with letrozole for postmenopausal women with ER+, HER2- metastatic breast cancer. • The approval was based on the PALOMA-1 trial, which demonstrated improved progression-free survival compared to letrozole alone in previously untreated patients. • Common adverse events associated with the combination therapy include neutropenia, leukopenia, and fatigue, requiring careful monitoring and dose adjustments. • Clinicians should consider toxicities, quality-of-life issues, and cost when making treatment decisions, as alternative regimens with different toxicity profiles exist.

Drug Development Updates