October 11, 2019

Impact: High

  • Merck has announced that the China NMPA (National Medical Products Administration) has approved the use of Keytruda (pembrolizumab) as a frontline monotherapy agent in patients with locally advanced or metastatic NSCLC tumors with PD-L1 expression of at least 1% (TPS ≥1%, determined by an NMPA-approved test), without EGFR or ALK genomic mutations.
  • The approval is based on data from Chinese patients in the global Phase 3 KEYNOTE-042 trial (NCT02220894) and a separate China extension study (NCT03850444).
  • Patients with PD-L1 expression ≥1% treated with Keytruda (n=128) vs. platinum-based chemotherapy (n=134) showed mOS = 20.0 months vs. 13.7 months.
  • Results from the China extension study were presented at the IASLC World Conference on Lung Cancer in September 2019 (abstract MA11.02).

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October 1, 2019

Impact: High

  • Merck and Eisai have announced that FDA has granted an accelerated approval to the combination treatment of Keytruda/Lenvima (pembrolizumab/lenvatinib) for patients with advanced endometrial carcinoma that is not MSI-H (microsatellite instability-high) or dMMR (mismatch repair deficient), who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation.
  • This accelerated approval is based on ORR and duration of response data from the Phase 2 KEYNOTE-146 trial (NCT02501096).
    • ORR = 38.3% (CR rate = 10.6%)
    • mDoR (median duration of response) = not reached (1.2+ to 33.1+ months)
    • % with duration of response ≥ 6 months = 69%
  • This accelerated approval was reviewed under FDA’s Real-Time Oncology Review (RTOR) pilot program (submission on June 17, 2019; accelerated approval on September 17, 2019).
  • Additionally, this review was conducted under Project Orbis. Project Orbis allows for a concurrent review process from multiple international regulatory agencies. For this approval, FDA, Australian Therapeutic Goods Administration (TGA) and Health Canada collaboratively reviewed the submission and made a simultaneous decision in all 3 countries.

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October 1, 2019

Impact: High

  • The CHMP adopted a positive opinion recommending a change in the MAA for Remsima to include a new route of administration, new strength, and new pharmaceutical form:
  • Remsima 120 mg solution for subQ injection using prefilled pens or syringes.
  • It is only authorized in the RA indication.

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September 25, 2019

Impact: High

Ardelyx received FDA approval for tenapanor for the treatment of IBS-C in adults. The approval was based on two phase 3 clinical trials, T3MPO 1 and T3MPO2. The brand name for the drug will be Ibsrela. The label contains a black box warning on the risk of serious dehydration in pediatric patients, which is due to a class effect of NHE3 sodium transport inhibitors.

Ardelyx has decided not to commercialize the drug for IBS-C indication themselves and is awaiting a commercial partner who would also be interested in developing the drug for CIC. The company did not disclose details of ongoing partnership conversations during the call following tenapanor’s approval. Read more

September 4, 2019

Impact: High

  • GSK has announced that the pivotal phase 2 DREAMM-2 trial (NCT03525678) of belantamab mafodotin has met the primary endpoint of ORR.
  • 196 relapsed multiple myeloma patients who were refractory to an immunomodulatory drug, a PI and an anti-CD38 antibody were enrolled.
  • Safety and tolerability are reportedly consistent with what was observed in the DREAMM-1 trial.
  • Data will be submitted for presentation at an upcoming scientific meeting and be used to support regulatory filings later this year.

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August 21, 2019

Impact: High

Amgen announced that the U.S. District court of New Jersey ruled in favor of Amgen regarding the validity of two patents protecting exclusivity of Enbrel from biosimilars. 

Sandoz immediately announced that it would appeal the ruling.

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August 13, 2019

Impact: High

Get insight from inThought Research experts on the recent approval of Keytruda Monotherapy for 2L+ Esophageal cancer:

  • Merck has announced that FDA has approved Keytruda (pembrolizumab) as a monotherapy for patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (Combined Positive Score [CPS] ≥ 10), with disease progression after one or more prior lines of systemic therapy.
    • PD-L1 positivity will be determined by an FDA-approved companion diagnostic.
  • The approval is based on data from KEYNOTE-181 and KEYNOTE-180.
  • In KEYNOTE-181, the mOS for PD-L1+ esophageal squamous cell carcinoma (ESCC) patients treated with Keytruda was 10.3 months vs. 6.7 months in the chemotherapy arm (HR=0.64; p not provided).
    • The mPFS was 3.2 months for Keytruda treated patients vs. 2.3 months for the chemotherapy arm (HR=0.66, p not provided).
    • An ORR of 22% (CR rate=5%) was observed in Keytruda treated patients compared with 7% (CR rate=1%) in the chemotherapy arm.

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July 25, 2019

Impact: High

Get insight from inThought Research experts on Novartis results from the phase 3 PARAGON-HF clinical trial evaluating Entresto versus valsartan in HFpEF.

  • Novartis announced topline results from the phase 3 PARAGON-HF study evaluating Entresto versus valsartan in HFpEF.
  • The primary endpoint of reducing cardiovascular death and total heart failure hospitalizations did not reach reach statistical significance.
  • The data will be presented at the ESC Congress in September.
  • Scott Solomon M.D., the trial co-chair, noted that the full trial results may results in clinically meaningful benefits in patients with HFpEF and that the company will discuss next steps with clinical experts and regulators. 

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July 25, 2019

Impact: High

  • Bristol-Myers Squibb has provided updates on Part 1a and Part 2 of the Phase 3 CheckMate -227 study of Opdivo in 1L NSCLC.
  • Part 1a, which evaluated Opdivo and Yervoy vs. chemotherapy, met the co-primary endpoint of overall survival in patients with tumor expression levels of PD-L1 > 1%.
  • A survival benefit was also observed with Opdivo/Yervoy vs. chemotherapy alone in patients with tumors devoid of PD-L1 expression levels.
  • Part 2 of the study comparing Opdivo and chemotherapy to chemotherapy alone failed to meet the primary endpoint of overall survival (OS).
  • The mOS in patients treated with Opdivo/chemotherapy was 18.83 months vs. 15.57 months with chemotherapy alone. OS at 1 year was 67% with the combination vs. 59% with chemotherapy alone.
  • Data from this trial will be shared with regulatory authorities and be presented at a future medical conference.

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July 23, 2019

Impact: High

  • Zolgensma is approved for the treatment of pediatric patients less than 2 years of age with SMA including those who are pre-symptomatic at diagnosis.
  • Zolgensma is designed to address the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion.
  • Data from the Phase 3 STR1VE trial show prolonged event-free survival, increases in motor function and significant milestone achievement in patients with SMA Type 1, consistent with the Phase 1 START trial.
  • In the START trial, patients treated with Zolgensma achieved motor milestones never seen in the natural history of the disease, including sitting, talking and some patients walking, with no waning of effect nearly four years post-dosing.
  • AveXis has an exclusive, worldwide license with Nationwide Children’s Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans.

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