Us Fda Approves Bridgebio's Drug For Rare Heart Condition

This week Novartis NVS upgraded its mid-term sales growth guidance. The European Commission approved Pfizer’s PFE hemophilia drug Hympavzi (marstacimab). AstraZeneca’s AZN Tagrisso was recommended for approval in the EU for expanded use in EGFR-mutated lung cancer. J&J JNJ and Lilly LLY announced positive data from mid/late-stage studies.

Here's a recap of the week’s most important stories.

Novartis Raises Mid-Term Sales Outlook

Novartis raised its mid-term sales growth guidance by increasing its CAGR target for the 2023-2028 period from 5% to 6%. Using 2024 as the base year, Novartis expects its sales to witness a CAGR of 5% during 2024-2029. The company’s guidance increase was backed by higher sales expectations for key drugs like Pluvicto, Kesimpta, Kisqali, Cosentyx and Leqvio coupled with some upcoming launches. According to Novartis, eight of its drugs have peak sales potential between $3 billion and $8 billion. Additionally, it said that there are 30 candidates in its pipeline, which should help continue mid-single-digit sales growth beyond 2029. NVS remains on track to deliver core operating income margin of more than 40% by 2027.

EU Approval for Pfizer’s Hemophilia Gene Therapy Hympavzi

The European Commission granted marketing authorization to Pfizer’s anti-TFPI inhibitor Hympavzi (marstacimab) for the treatment of hemophilia A and B. The drug will be marketed as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in adults and adolescents with hemophilia A or B without inhibitors. Hympavzi’s approval was based on positive data from the phase III BASIS study. It was approved in the United States by the FDA last month.

Hympavzi is Pfizer’s second hemophilia treatment to be approved this year. Beqvez/ Durveqtix (fidanacogene elaparvovec), a one-time gene therapy for hemophilia B, was approved by the FDA in April 2024 while it is still under review in the EU.

J&J’s Icotrokinra Meets Goals in Pivotal Plaque Psoriasis Study

J&J pivotal phase III study evaluating icotrokinra (JNJ-2113) for moderate to severe plaque psoriasis (PsO) met its co-primary endpoints at week 16. The ICONIC-LEAD study’s primary endpoints were Psoriasis Area and Severity Index (PASI) 90 and Investigator’s Global Assessment (IGA) of 0/1 response at week 16. Top-line data from the study showed that at week 16, 64.7% of adult and adolescent patients treated with icotrokinra achieved clear or almost clear skin (IGA 0/1) and 49.6% achieved PASI 90.

The positive responses continued to improve through week 24. At week 24, 74% achieved clear or almost clear skin (IGA 0/1), and 64.9% achieved PASI 90. J&J also said that another phase III study, ICONIC-TOTAL, which evaluated once-daily icotrokinra, met the primary endpoint of IGA of 0/1 at week 16 compared to placebo.

CHMP Nod to AZN’s Tagrisso for Expanded Use in NSCLC

AstraZeneca announced that the Committee for Medicinal Products for Human Use (“CHMP”) of the European Medicines Agency (“EMA”) has recommended approval of Tagrisso (osimertinib) for treating unresectable EGFR-mutated non-small cell lung cancer (NSCLC) following treatment with platinum-based chemoradiation therapy. The CHMP recommended Tagrisso for the treatment of EGFRm patients whose tumors have exon 19 deletions or exon 21 (L858R) substitution mutations. The CHMP’s recommendation was based on data from the phase III LAURA study. The FDA approved Tagrisso for similar use in September.

Currently, Tagrisso is approved in several countries as a monotherapy for the first-line treatment of EFGR-mutated NSCLC, locally advanced or metastatic EGFR T790M mutation-positive NSCLC and adjuvant treatment of early-stage EGFRm NSCLC. Tagrisso is also approved in combination with chemotherapy for frontline EGFR-mutated NSCLC based on data from the FLAURA2 study in the United States and some other countries.

LLY Oral Cholesterol Drug Lowers Lipoprotein Levels in Phase II

Lilly’s oral, once-daily cholesterol-lowering candidate muvalaplin reduced lipoprotein(a) or Lp(a) levels in adults with a high risk of cardiovascular events in a 12-week phase II study.

In the study, muvalaplin led to a significant reduction in elevated Lp(a) levels compared to placebo, meeting the study’s primary endpoint of percent change in Lp(a) from baseline to week 12. Using an intact Lp(a) assay, the highest dose (240 mg) of muvalaplin reduced Lp(a) levels by 85.8%. Muvalaplin also met secondary endpoints for all three doses. Muvalaplin has a novel mechanism of action. It disrupts the interaction between apolipoprotein(a) and apolipoprotein(b), which prevents the formation of Lp(a). Apolipoprotein(a) is a key component of Lp(a), which increases the risk of cardiovascular events.

The NYSE ARCA Pharmaceutical Index declined 1.1% in the past five trading sessions.

Large Cap Pharmaceuticals Industry 5YR % Return

Large Cap Pharmaceuticals Industry 5YR % Return

Here’s how the eight major stocks performed in the previous five trading sessions.

In the last five trading sessions, Lilly declined the most (4.6%), while J&J rose the most (2.4%).

In the past six months, while AbbVie rose the most (8.5%), Novo Nordisk declined the most (24.3%).

(See the last pharma stock roundup here: AZN, BAYRY’s Earnings, ABBV’s Pipeline Setback)

What's Next in the Pharma World?

Watch this space for regular pipeline and regulatory updates next week.

AstraZeneca, Lilly, Merck and J&J have a Zacks Rank #3 (Hold) each, while Pfizer has a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Zacks Investment Research

Replimune Group REPL announced that it has submitted a biologics license application (BLA) to the FDA for its lead pipeline candidate, RP1, in combination with Bristol Myers’ BMY blockbuster immuno-oncology drug Opdivo (nivolumab) to treat adult patients with anti-PD1 failed advanced melanoma. The regulatory filing has been made under the FDA’s Accelerated Approval pathway. In response to the encouraging news, the stock rose 19.4% in the after-market hours on Thursday. The stock is also rising today during the pre-market hours.

RP1 is based on a proprietary strain of herpes simplex virus, engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF, intended to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response.

REPL’s RP1 BLA Submission Supported by Robust Study Data

Replimune’s BLA submission for RPI, in combination with Bristol Myers’ Opdivo, for the advanced melanoma indication is supported by positive top-line results from the primary analysis of the IGNYTE study. The study evaluated the combo drug in adult patients who experienced confirmed disease progression while being treated with at least eight weeks of prior anti-PD1 therapy.

Results showed that the overall response rate (ORR) was 33.6% by modified RECIST 1.1 criteria, the primary endpoint as defined in the protocol. ORR was 32.9% by RECIST 1.1 criteria, an additional analysis requested by the FDA. Responses from the baseline were highly durable, with all responses lasting more than six months and the median duration of response exceeding 35 months.

The combination continues to be well-tolerated, with mainly grade 1-2 constitutional-type side effects. Year to date, shares of Replimune have gained 31.1% against the industry’s 9.7% decline.