Amgen, MSD, and AbbVie Highlight Key Small Molecule Drug Candidates in Recent Filings
Ipsen
Biopharmaceutical Manufacturer
Genfit
Biopharmaceutical Company
Elafibranor
CAS: 923978-27-2
Indications: Primary Biliary Cholangitis,Non-alcoholic steatohepatitis, renal diseases
Original Research Company:IpsenAndGenfit
2019Year4Month,FDAGrantelafibranor Breakthrough Drug Designation，This GrantWas based on a randomized, double-blind, placebo-controlledIIData from a Phase III clinical study, which was conducted in adult patients with primary biliary cholangitis who had an inadequate response to ursodeoxycholic acid.Data show,elafibranorSignificantly reduced alkaline phosphatase (ALP) level, showed significant therapeutic effects compared to placebo in the primary endpoint, and also achieved the composite endpoint for drug registration. In addition,ALPSignificantly reduce outside of,2Other Group PatientsPrimary Biliary CirrhosisBiomarkers, includingγ-Glutamyl transferase (GGT), lipid markers (total cholesterol, low-density lipoprotein, and triglycerides), and anti-inflammatory markers (IgM、CRP, haptoglobin and fibrinogen) also showed improvement.
2019Year7Month25On [date], the European Commission granted FranceGenfitTreatment of Primary Biliary CholangitisDrugelafibranorOrphan Title。
2023Year6Month30Day,IpsenAndGenfitAnnouncement, under researchelafibranorIn critical3Reached the primary composite endpoint in the clinical trial.IpsenPlan to collaborate with the United StatesFDASubmit regulatory applications after discussions with the European Medicines Agency. The trial results show,elafibranor（80 mg) Group in51%of patients achieved cholestasis response, while the value for the placebo group was4%（p<0.0001). Compared with the placebo group,elafibranorThe group reached the first secondary endpoint,52WeektimeALPNormalization also showed statistically significant improvement. A trend toward pruritus improvement was observed, but it did not reach statistical significance.elafibranorWell tolerated, with a safety profile consistent with findings reported in previous studies.
On June 10, 2024, Ipsen announced that the U.S. Food and Drug Administration (FDA) had granted accelerated approval to Iqirvo® (elafibranor) 80 mg tablets for the treatment of primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA) for adults with an inadequate response to UDCA, or as monotherapy for patients unable to tolerate UDCA. The approval was based on Phase III trial data published in the New England Journal of Medicine. Compared to placebo plus UDCA treatment (n=53), and compared to Iqirvo plus UDCA treatment (n=108), the ELATIVE trial demonstrated a more than 13-fold increase in the proportion of patients achieving the composite primary endpoint of biochemical response (51% vs. 4%, a treatment difference of 47%). ALP is a biochemical marker used as a surrogate endpoint in PBC trials. Secondary endpoints showed normalization of ALP levels only in patients treated with Iqirvo (15% in the Iqirvo plus UDCA group (n=108) vs. 0% in the placebo plus UDCA group (n=53)). The majority of patients (95%) received study treatment (Iqirvo or placebo) in combination with UDCA.
Paltusotine
Indications:Acromegaly
Original Research Company:Crinetics Pharmaceuticals
2023Year9Month11Day,Crinetics PharmaceuticalsAnnouncementSST2AgonistPaltusotinePhase III Clinical Trial for the Treatment of AcromegalyPATHFNDR-1Achieved the primary endpoint and all secondary endpoints.
On December 9, 2024, the U.S. Food and Drug Administration (FDA) announced that the U.S. Food and Drug Administration (FDA) had acceptedpaltustineNew Drug Application (NDA) for the treatment and long-term maintenance of acromegaly in adults. If approved, paltustine will become the first and only once-daily oral selective somatostatin receptor type 2 non-peptide agonist.。
Danicopan
On April 1, 2024, danicopan (Voydeya) was approved by the U.S. Food and Drug Administration (FDA) as an add-on treatment to ravulizumab-cwvz or eculizumab for the treatment of extravascular hemolysis (EVH) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
Tolebrutinib
Indications:Multiple Sclerosis with Myasthenia Gravis
Original Research Company:Sanofi
2022Year6Month30Day,FDASuspended SanofiTolebrutinibIn the development of multiple sclerosis and myasthenia gravis3Phase Clinical Trial,The reason is the occurrence of multiple cases of drug-induced liver injury.
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Bemnofosbuvir
Omecamtiv mecarbil
Indications: Chronic Heart Failure
Original Research Company:Amgen, Inc.
2022Year2Month,CytokineticsToFDASubmittedOmecamtiv MecarbilThe listing application,PDUFADate:11Month30Day.
2022Year12Month13Day,InFDADrug Review Meeting,The Expert Committee with8:3The voting result suggests,CytokineticsA drug researched for over a decadeOmecamtivmecarbil,The clinical benefit does not outweigh the risks.
On February 28, 2023, Cytokinetics announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for omecamtiv mecarbil. The FDA stated that the effectiveness of GALACTIC-HF in reducing heart failure events and cardiovascular death in adult patients with chronic heart failure and reduced ejection fraction was not sufficiently compelling, as evidenced by at least two adequate and well-controlled clinical studies. The FDA indicated that results from another clinical trial of omecamtiv mecarbil are needed to establish substantial evidence of its efficacy in treating HFrEF, with a favorable benefit-risk profile.
Veliparib
Indications:Ovarian cancer, metastatic non-squamous non-small cell lung cancer, triple-negative breast cancer
Original Research Company:AbbVie
2016Year11Month,AbbVie AnnouncementFDAGrantVeliparibOrphan Drug Designation for Non-Small Cell Lung Cancer.
Ipatasertib
Bezuclastinib
Indications: Gastrointestinal Stromal Tumor，Mastocytosisetc.
Original Research Company:Cogent Biosciences，Plexxikon, Inc.
2022Year6Month10Day,Cogent BiosciencesAnnouncement of ongoingAPEX 2Positive preliminary data obtained from the Phase clinical trial, which evaluated the selectivityKIT D816VInhibitorbezuclastinibFor advanced systemic mastocytosis (AdvSM) The role of the patient.
On July 7, 2025, Cogent Biosciences announced that the registration-guided results from Part 2 of the SUMMIT clinical trial of bezuclastinib in patients with Non-Advanced Systemic Mastocytosis (NonAdvSM) showed clinically meaningful and highly statistically significant improvements in the primary and all key secondary endpoints, including patient-reported symptoms and objective measurements of mast cell burden.87.4% of patients treated with Bezuclastinib had at least a 50% reduction in serum tryptase, compared to 0% in the placebo-treated group.). Based on these data, Cogent is expected to submit its first New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) by the end of 2025.
Milademetan
Indications: Soft Tissue Sarcoma
Original Research Company:Rain Oncology
2023Year5Month22Day,Rain OncologyAnnouncementMDM2InhibitorMilademetan（RAIN-32) Treatment of Dedifferentiated Liposarcoma (DDLPS) ofIIIPeriodMANTRAThe study did not achieve a significant improvement in progression-free survival compared to standard treatment.PFS) The primary endpoint.
Bemcentinib
