It was a busy week for the biotech sector, with quite a few important regulatory and pipeline updates. Among these, pipeline updates from Moderna MRNA and Rocket Pharmaceuticals RCKT grabbed the spotlight.

Recap of the Week’s Most Important Stories:

Updates From Moderna:  Moderna announced key business and clinical updates at its annual R&D Day. Management provided an encouraging update on its reformulated seasonal influenza vaccine candidate, mRNA-1010. Interim analysis of data from the ongoing phase III immunogenicity study (P303) showed that mRNA-1010 elicited higher immune responses across all four A and B strains of influenza than Fluarix.

In a separate phase I/II head-to-head study, mRNA-1010 also elicited a higher antibody response across all four A and B strains over Fluzone HD. Shares most likely gained on this update. Data from the P303 study is expected to support Moderna’s filing for mRNA-1010.

Moderna and its partner Merck initiated a pivotal phase III study on its investigational individualized neoantigen therapy mRNA-4157 in melanoma patients. Management announced that it has completed enrolling participants in a late-stage study on mRNA-1283, a next-generation refrigerator-stable COVID-19 vaccine. Moderna expects to launch 15 new products, including four products in rare diseases, over the next five years. The company expects to bring in an additional $10 billion to $15 billion in annual sales of cancer, rare and latent disease therapies by 2028. Management reiterated its previously announced revenue guidance of $8-$15 billion in product sales from its respiratory franchise by 2027.

Management plans to spend around $25 billion in R&D over the next five years. The company also plans to resize its manufacturing footprint and supply base to increase its gross margin in the long term between 75% and 80%.

Moderna currently carries a Zacks Rank #3 (Hold).

Bicycle Gains on Cancer Study Update:  Shares of Bicycle Therapeutics BCYC gained after the company announced an expedited development plan for pipeline candidate BT8009 after recent discussions with the FDA. Bicycle stated that it has aligned with the FDA on a phase II/III registrational trial, Duravelo-2, that has an innovative design allowing for potential accelerated approval in untreated (first-line) and previously treated (second-line plus) metastatic bladder (urothelial) cancer. Bicycle plans to initiate this registrational trial in the first quarter of 2024. Investors were encouraged by the advancement and shares were up.

The Duravelo-2 study will assess BT8009 in untreated metastatic bladder cancer (Cohort 1) and previously treated metastatic bladder cancer (Cohort 2).  Two doses of BT8009 plus standard Keytruda (pembrolizumab) regimen will be initially evaluated in Cohort 1. BT8009 plus pembrolizumab will be evaluated against chemotherapy after an optimal dose is selected. Two doses of BT8009 as monotherapy will be initially studied in Cohort 2. An additional arm of BT8009 plus standard pembrolizumab regimen will be added after the selection of the optimal dose. Meanwhile, discussions with the FDA about the design of the confirmatory trial for previously treated metastatic bladder cancer are ongoing.

Rocket Pharmaceuticals Soars on Regulatory Update: Shares of late-stage biotechnology, Rocket Pharmaceuticals, surged following the company’s announcement of an alignment with the FDA on the global phase II trial of investigational gene therapy RP-A501 for the treatment of Danon Disease. The single-arm, multi-center mid-stage study will evaluate the efficacy and safety of RP-A501 in 12 patients with Danon Disease, including a pediatric safety run-in (n=2), with a natural history comparator and a dose level of 6.7 x 1013 GC/kg.

The study will assess the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression and reductions in left ventricular mass in order to support accelerated approval.  Key secondary endpoint is a change in troponin. Additional secondary endpoints will include natriuretic peptides, Kansas City Cardiomyopathy Questionnaire, New York Heart Association class, event-free survival to 24 months and treatment emergent safety events. These endpoints could support full approval with longer-term follow-up. A global natural history study will serve as an external comparator and run concurrently with the mid-stage study.

In order to enable the initiation of EU study activities, the filing of the Clinical Trial Application /Investigational Medicinal Product Dossier for RP-A501 is expected in the third quarter.  Rocket has secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease.

Gilead’s Trodelvy Shows Promise in NSCLC: Gilead Sciences, Inc. GILD announced encouraging data from the phase II EVOKE study evaluating its breast cancer drug Trodelvy in combination with Keytruda with or without chemotherapy, regardless of PD-L1 expression, in patients with advanced or metastatic non-small cell lung cancer (NSCLC) without actionable genomic alterations.

Per the status of the cancer or PD-L1 expression, patients were assigned to cohorts. Patients were assigned to Cohorts A or B according to tumor proportion score (TPS) status. While Cohort A enrolled patients with squamous/non-squamous NSCLC with a TPS ≥ 50%, Cohort B enrolled patients with squamous/non-squamous NSCLC with a TPS < 50%. Patients enrolled in Cohorts A or B received the combination of Trodelvy and Keytruda. Patients enrolled in Cohorts A or B received the combination of Trodelvy and Keytruda.

Results show encouraging activity of Trodelvy in combination with Keytruda in first-line metastatic NSCLC across all PD-L1 subgroups and histologies studied. Data showed a confirmed and unconfirmed objective response rate (ORR) of 69% and a disease control rate (DCR) of 86% in Cohort A (n=29). Confirmed and unconfirmed ORR was 44% and DCR was 78% in Cohort B (n=32). Across both cohorts, ORR was 56% and DCR was 82%. The median duration of response (DoR) was not reached at the time of data cut-off and the DoR rate at six months was 88% in both cohorts. The results support further investigation of the combination in the first-line metastatic NSCLC.

Gilead is also evaluating the phase III EVOKE-03 study of Trodelvy in combination with Keytruda versus Keytruda monotherapy for patients with first-line PD-L1-high metastatic NSCLC.

Bristol Myers’ Pulmonary Fibrosis Drug Study Data: Bristol Myers Squibb BMY announced the results from a mid-stage study evaluating novel antifibrotic treatment BMS-986278 in patients with progressive pulmonary fibrosis (PPF).

This phase II study was a global, randomized study wherein parallel cohorts of patients with IPF and PPF were given 30 mg or 60 mg of BMS-986278 or a matched placebo orally twice daily.  There were three phases in the study – a 26-week placebo-controlled treatment period, an optional 26-week active treatment extension period and a four-week post-treatment follow-up period. Patients were permitted to take background antifibrotics in the idiopathic pulmonary fibrosis cohort and background antifibrotics and/or immunosuppressants in the PPF cohort. The primary endpoint was the rate of change in the percent predicted forced vital capacity (ppFVC) from baseline to week 26 in the IPF cohort. A key secondary point was the rate of change in ppFVC from baseline through week 26 in the PPF cohort.

Results showed that a twice-daily 60 mg dose of BMS-986278 resulted in a 69% relative reduction in the rate of decline in ppFVC with 26 weeks of treatment versus the placebo arm. In the 30 mg group, a 42% relative reduction was observed in the while-on-treatment analysis and a 37% relative reduction was observed in the treatment policy analysis. Bristol Myers plans to evaluate the candidate in the global phase III ALOFT trial program.

Performance

The Nasdaq Biotechnology Index has gained 0.09% in the past five trading sessions. Among the biotech giants, GSK has gained 6.83% during the period. Over the past six months, shares of Moderna have plunged 26.58%.

What’s Next in Biotech?

Stay tuned for more pipeline updates.

Bristol Myers Squibb Company (BMY): Free Stock Analysis Report

Gilead Sciences, Inc. (GILD): Free Stock Analysis Report

Moderna, Inc. (MRNA): Free Stock Analysis Report

Rocket Pharmaceuticals, Inc. (RCKT): Free Stock Analysis Report

Bicycle Therapeutics PLC Sponsored ADR (BCYC): Free Stock Analysis Report

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