- Eli Lilly And Co announced updated results from the pivotal Phase 3 monarchE trial of adjuvant Verzenio (abemaciclib) in combination with standard endocrine therapy (ET) for HR-positive, HER2-negative high-risk early breast cancer.
- The new data showed Verzenio’s benefits held up across patient subgroups after longer follow-up.
- In 2020, The monarchE trial hit statistical significance after a median of 15.5 months follow-up. Verzenio’s addition to post-surgery endocrine therapy significantly cut the risk of invasive cancer recurrence or death by 25%, regardless of Ki-67 status.
- Now, investigators have followed patients for a median of 42 months, coming to the second preplanned interim analysis of patient survival. Nearly all patients had already come off the two-year treatment period.
- In the overall population, the Verzenio group had a 33.6% reduced risk of developing invasive disease recurrence or death over the control arm, which got endocrine therapy alone.
- Adjuvant Verzenio also reduced the risk of developing the metastatic disease by 34.1%.
- AVROBIO Inc announced new interim data from AVR-RD-02, an investigational gene therapy for Gaucher disease.
- The data showed stabilization or reversal of multiple clinically relevant measures in five patients with Gaucher disease after receiving a single dose of AVR-RD-02.
- An 11-year-old patient was dosed. Fifteen months post gene therapy, the patient has normalized peripheral glucocerebrosidase (GCase) enzyme activity and plasma chitotriosidase, a marker of activated macrophages, and remains off enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).
- The patient's albumin levels increased by 33% eight months post-gene therapy, reflecting improvements in lymphadenopathy and enteropathy.
- Additionally, the patient did not develop any new lesions post-gene therapy.
- All four adult GD1 patients in the Guard1 trial saw sustained engraftment with vector copy numbers (VCN) between 0.54 - 0.86 per diploid genome 14 weeks to two years post-gene therapy. Reconstitution of GCase enzyme activity in plasma and peripheral blood leukocytes within the normal range.
- Notably, three of the four dosed patients demonstrate a reduced liver and spleen volume below their ERT baseline.
- AVROBIO plans to initiate a global, registrational Phase 2/3 trial in Gaucher disease type 3 (GD3) in 2H of 2023.
- Esperion Therapeutics Inc's CLEAR Outcomes trial met its primary endpoint, demonstrating statistically significant risk reduction in MACE-4 in patients treated with 180 mg/day NEXLETOL (bempedoic acid) compared to placebo.
- The data demonstrate statistically significant and clinically meaningful results.
- The study included over 14,000 patients at over 1,200 sites in 32 countries.
- Bempedoic acid becomes the first ATP citrate lyase inhibitor and oral non-statin to meet the major adverse cardiovascular events (MACE-4) primary endpoint.
- NEXLETOL is indicated as an adjunct to diet and maximally tolerated statin therapy for heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease who require additional lowering of LDL-C.
- The company reported CLEAR Outcomes trial data sooner than expected in January 2023.
- In Q3, NEXLETOL generated sales of $14 million, up 28% Y/Y.
- The company looks forward to sharing this data with the medical community and submitting it to appropriate regulatory authorities as quickly as possible in 2023.
