Your monthly synopsis of new drugs expected to hit the market

At Prime Therapeutics (Prime), we have positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs likely to be approved by the United States (U.S.) Food and Drug Administration (FDA).

Drug pipeline for September 2024

Sept. 13, 2024: ocrelizumab/hyaluronidase (Ocrevus^®) SC
Genentech (a member of Roche Group) has submitted a subcutaneous (SC) formulation of the CD20-directed cytolytic antibody Ocrevus^® to the FDA. They are seeking approval for the same indications as the original intravenously (IV) administered formulation, which includes relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). A health care professional administers the SC formulation over 10 minutes twice a year, while maintenance dosing of the original formulation is administered via IV infusion twice a year. In the OCARINA II study, patients with RMS or PPMS who received the SC formulation experienced near-complete suppression (97.2%) of clinical relapses and brain lesions, which is consistent with IV Ocrevus^®.¹

Sept. 15, 2024: atezolizumab/hyaluronidase (Tecentriq^®) SC
Genentech also submitted a SC version of their programmed death-ligand 1 (PD-L1) blocking antibody Tecentriq^®. They are pursuing approval for all the same indications that the IV formulation Tecentriq^® holds, which includes, alveolar soft part sarcoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer (NSCLC), small cell lung cancer and urothelial cancer. A health care professional injects the SC formulation over 3–8 minutes and has the potential for administration in the home. The IV formulation is administered over 30–60 minutes every two, three or four weeks, depending on the indication and dose. In a phase 3 trial, similar pharmacokinetics and safety profile were reported with Tecentriq^® SC compared to Tecentriq^® IV in patients with NSCLC.²

Sept. 18, 2024: tradipitant
Vanda and Eli Lilly are awaiting FDA approval of tradipitant for the treatment of gastroparesis (GP). Tradipitant is a neurokinin receptor 1 (NK-1R) antagonist. If approved, it will be the first agent in this class to treat GP symptoms and the first new mechanism for GP to be approved in over 40 years. In clinical trials, twice-daily oral administration of tradipitant led to a significant improvement in average nausea severity compared to placebo in patients who did not use rescue medications (e.g., ondansetron, prochlorperazine, promethazine).³ However, no significant difference in average nausea severity was reported in patients who were also given rescue medications.

For more information, see the tradipitant Deep Dive in the July 2024 edition of Prime Therapeutics’ Quarterly Drug Pipeline.

Sept. 21, 2024: arimoclomol
Zevra Therapeutics submitted arimoclomol for the treatment of Niemann-Pick disease type C (NPC), a rare, inherited, lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes. There is no cure for NPC, but miglustat has been prescribed for off-label use in the U.S. in select patients. Arimoclomol has been evaluated for NPC based on its action in preserving cellular function and preventing cell death in cells experiencing lysosomal stress. It was evaluated in a 12-month clinical trial (NCT02612129) in patients with NPC who were 2–18 years of age and able to walk independently or with assistance. The study showed that the addition of arimoclomol to routine clinical care resulted in a 65% relative reduction in annual disease progression as measured by the 5-Domain (5D) NPC Clinical Severity Scale (5D-NPCCSS) score (difference versus placebo, -1.4 point; p=0.046), which includes parameters for ambulation, cognition, fine motor skills, speech and swallowing. In the trial, arimoclomol was administered by mouth or by feeding tube three times a day. The FDA granted arimoclomol Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations. On Aug. 2, 2024, the Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted (11-5) in favor of arimoclomol for NPC. If approved, arimoclomol could be one of two new agents to treat NPC.

For more information, see the arimoclomol Deep Dive in the July 2024 edition of Prime Therapeutics’ Quarterly Drug Pipeline.

Sept. 24, 2024: N-acetyl-L-leucine (IB1000)
IntraBio is awaiting approval for N-acetyl-L-leucine (NALL) for the treatment of NPC. NALL is a modified, acetylated derivative of the natural, essential amino acid leucine that normalizes energy production, improves lysosomal function and cellular signaling, and reduces neuronal inflammation. NALL administered orally two to three times per day was evaluated in a 12-week double-blind, cross-over clinical trial (NCT05163288) in patients 4 years and older with mild to severe symptoms of NPC. NALL demonstrated a significant improvement compared to placebo in the Scale for the Assessment and Rating of Ataxia (SARA) score (least square mean difference, -1.28 points; p<0.001). The FDA granted NALL Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and a Priority Review. If approved, NALL could be one of the first agents indicated for NPC.

For more information, see the N-acetyl-L-leucine Deep Dive in the July 2024 edition of the Prime Therapeutics’ Quarterly Drug Pipeline.

Sept. 26, 2024: xanomeline/trospium (KarXT)
KarXT is a combination product by Bristol Myers Squibb that contains the antipsychotic agent xanomeline and the muscarinic acetylcholine receptor agonist trospium and was submitted to the FDA for the treatment of schizophrenia. KarXT distinguishes itself from first- and second-generation antipsychotic agents by providing selective M1/M4 muscarinic receptor agonism through xanomeline without directly impacting dopamine D2 receptors and mitigating muscarinic side effects using trospium. In the double-blind EMERGENT-2 and EMERGENT-3 trials, KarXT administered orally twice daily led to a statistically significant and clinically meaningful reduction in the primary endpoint of Positive and Negative Syndrome Scale (PANSS) total score compared to placebo (difference in mean change compared to placebo, -9.6 points and -8.4 points, respectively; p<0.0001 for both) at five weeks.^{4, 5} If approved, KarXT will provide the first new mechanism of action to treat schizophrenia in several decades.

For more information, see the xanomeline-trospium Deep Dive in the April 2024 edition of the Prime Therapeutics’ Quarterly Drug Pipeline.