Clinical Catch-Up: Pfizer's Paxlovid Gaff, a Win for Medicago-GSK's Vaccine
It was a very busy week for clinical trial news and updates. Pfizer's Paxlovid racks up a rare trial miss, Medicago and GSK publish positive data for their plant-based particle vaccine and Rhythm Pharmaceuticals touts strong potential in Bardet-Biedl Syndrome. Here's a look at that and more in this week's clinical catch-up.
Pfizer reported topline results from the Phase II/III Epic-PEP trial of its antiviral regimen Paxlovid (nirmatrelvir and ritonavir tablets) for post-exposure prophylactic use. The company observed risk reductions of 32% and 37% in adults who received Paxlovid for five and 10 days, respectively, to prevent infection. However, these figures were not statistically significant. The trial failed to hit the primary endpoint of reducing the risk of confirmed and symptomatic COVID-19 in adults who had been exposed via a household contact.
Medicago and GlaxoSmithKline published promising data from their study of CoVLP in symptomatic COVID-19 caused by five variants. It is a plant-based particle vaccine, and it demonstrated 69.5% efficacy against the variants and 78.8% against moderate-to-severe disease.
Sen-Jam Pharmaceutical initiated enrollment in a Phase II trial in Nepal for SJP-002C for COVID-19. The oral therapeutic is a combination therapy using an approved drug with another that mitigates the side effects of the other. No details on the therapeutic itself were provided.
Rhythm Pharmaceuticals presented new data from the Phase III trial of setmelanotide in patients with Bardet-Biedl Syndrome. It also announced cumulative safety data from across the setmelanotide clinical development program. The data supported the drug’s potential for safe and effective precision therapy.
Setmelanotide was approved by the FDA in November 2020 under the brand name Imcivree for chronic weight management in adult and pediatric patients 6 years and older with obesity due to POMC, PCSK1 or LEPR deficiency.
Rezolute announced positive results from its Phase IIb RIZE study of RZ358 in congenital hyperinsulinism. The study demonstrated a reduction of ~75% in hypoglycemia events by blood glucometer (BGM) as well as time in hypoglycemia by continuous glucose monitoring (CGM). RZ358 is a human monoclonal antibody that binds to a unique allosteric site on the liver, fat, and muscle insulin receptors.
Tarsus Pharmaceuticals announced that its SATURN-2 Phase III trial of TP-03 for demodex blepharitis hit the primary endpoint and all secondary endpoints. Demodex blepharitis is an eyelid disease. TP03 (lotilaner ophthalmic solution, 0.25%) is designed to target and eradicate mite infestation that is the cause of Demodex blepharitis.
UCB published the data from its Phase III trial of Fintepla when added to a patient’s current anti-epileptic treatment for seizures associated with Lennox-Gastaut Syndrome (LGS). The drug was effective in reducing the frequency of drop seizures. The FDA recently approved the drug for seizures associated with LGS.
Vertex Pharmaceuticals reported the FDA placed a clinical hold on its Phase I/II trial of VX-880, a pancreatic islet cell replacement therapy for patients diagnosed with type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia. The FDA is claiming there is insufficient information to support dose escalation.
Medicenna announced new clinical data from the Phase I/II Ability trial of MDNA11. The drug is a long-acting IL-2 super-agonist being developed to treat advanced, relapsed or refractory solid tumors.
Aptorum Group finalized data from its Phase I trial of SACT-1 for neuroblastoma and other cancer types. The drug is a repurposed small molecule and is designed to be used in combination with standard-of-care chemotherapy.
Imago Biosciences completed enrollment for the Phase II trial of bomedemstat for essential thrombocythemia. Bomedemstat is an oral lysine-specific demethylase 1 (LSDA) inhibitor.
BioXcel Therapeutics dosed the first patient in the Phase III TRANQUILITY II trial of BXCL501 for acute treatment of agitation in patients with Alzheimer’s disease. BXCL501 is the company’s proprietary, orally dissolving thin film formulation of dexmedetomidine.
Denali Therapeutics initiated dosing in a Phase II trial of SAR443820 in amyotrophic lateral sclerosis (ALS). The trial is sponsored by Sanofi. SAR443820 is an oral, brain-penetrant small molecule RIPK1 inhibitor.
Hoth Therapeutics announced preliminary findings from a Phase Ib trial of BioLexa Lotion in mild to moderate atopic dermatitis. The company reported results from three efficacy secondary endpoints: Eczema Area and Severity Index (EASI), the SCORing Atopic Dermatitis scale (SCLRAD), and an Investigator Global Assessment scale (IG).
XORTX Therapeutics initiated dosing in the XRX-OXY-101 bridging PK study of XRx-008 for autosomal dominant polycystic kidney disease. XRx-008 is a novel proprietary formulation of oxypurinol.
Bausch + Lomb presented data from the second pivotal Phase III trial of Nov03 (perfluorohexyloctane) for first-in-class treatment for dry eye disease (DED) associated with Meibomian gland dysfunction (MGD). The data hit both efficacy endpoints. Nov03 is a water-free, single-component preservative-free eye drop.
Compass Therapeutics reported positive interim Phase II data of CTX-009 with paclitaxel for biliary tract cancer, which demonstrated a 42% overall response rate and clinical benefit of 92%. The drug is a bispecific antibody that blocks the Delta-like ligand 4/Notch (DLL4) and VEGF-A signaling pathways.
Connect Biopharma announced its Phase II study of CBP-307 for ulcerative colitis did not meet statistical significance. The drug is a once-daily, oral, selective sphingosine 1-phosphate (S1P) receptor modulator.
Suzhou Kintor Pharma dosed the first patient in the U.S. in its Phase II study of ALK-1 antibody and Bristol Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab) for advanced hepatocellular carcinoma (HCC). ALK-1 antibody is a potential first-in-class, fully human IgG2 neutralizing monoclonal antibody that inhibits BMP9, TGFbeta through ALK-1 receptor-mediated signal transduction and tumor angiogenesis.
Ilya Pharma announced the FDA cleared its IND for ILP100-Topical for a Phase II trial. The drug is a first-in-class immunotherapy designed to treat surgical wounds in patients with prediabetes, diabetes and obesity.
Melt Pharmaceuticals announced topline results from its Phase I PK trial of Melt-210. The drug is a fixed dose of 3 mg of midazolam in a proprietary rapidly dissolving sublingual tablet for procedural sedation.
NeuroPace announced the first patient was treated in the Response clinical trial of the RNS System in adolescent patients with drug-resistant focal epilepsy. RNS System is an FDA-approved brain-responsive neuromodulation system.
Amphera announced positive Reactive Phase II safety and efficacy data from the first cohort of 10 patients receiving its MesoPher dendritic cell therapy for pancreatic cancer. The data showed an average OS after surgery of about 36 months. No disease recurrence or tumor progression was observed in 7 of 10 patients.
Genenta Science escalated to the next planned dose in its Phase I/IIa trial of Temferon for glioblastoma multiforme patients with an unmethylated MGMT gene promoter. Temferon is based on ex-vivo gene transfer into autologous hematopoietic stem/progenitor cells to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages.
BioCryst announced that the FDA had placed a partial clinical hold on three of its clinical trials evaluating BCX9930 after finding early onset and moderate or severe elevations in serum creatinine after receiving 500 mg twice a day for several weeks. The company cannot enroll new patients, although patients already enrolled who appear to be receiving clinical benefits from the treatment and have no other options can continue to be dosed.
The drug is an oral Factor D inhibitor to treat complement-mediated diseases. Redeem-1 is evaluating the drug alone in PNH patients with inadequate response to a C5 inhibitor. Redeem-2 is studying it alone in PNH patients not currently receiving complement inhibitor therapy. Renew is evaluating the drug in patients with either C3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN) or primary membranous nephropathy (PMN).
Karuna Therapeutics completed enrollment in the Phase III Emergent-2 trial of KarXT for schizophrenia. KarXT (xanomeline-trospium) is an oral investigational antipsychotic with a novel mechanism of action mediated via muscarinic cholinergic receptors.
AstraZeneca announced positive high-level results for the Phase III Deliver study of Farxiga (dapagliflozin) for heart failure with mildly reduced or preserved EF, defined as a left ventricular ejection fraction greater than 40%. Farxiga is approved for indications associated with type 2 diabetes, heart failure (HF) with reduced ejection fraction and chronic kidney disease. The study demonstrated a statistically significant and clinically meaningful decrease in cardiovascular death or worsening health failure.
The company also released data from the Phase III Champion-NMOSD trial of Ultomiris in patients with AQP4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD). The data, which used the Soliris Prevent trial as an external placebo control, demonstrated a statistically and clinically significant reduction in the number of relapses.
Argenyx announced positive data from the Phase III Advance trial of Vyvgart for primary immune thrombocytopenia (ITP). Vyvgart (efgartigimod alfa-fcab) demonstrated a 21.8% sustained platelet count.
Palisade Bio and Newsoara Biopharma received clearance to initiate a Phase III trial of LB1148 in China for accelerated return of bowel function in adult patients undergoing bowel/abdominal surgery. LB1148 is an oral, broad-spectrum serine protease inhibitor.
Moleculin Biotech received the go-ahead in Poland to proceed with a Phase I/II trial of Annamycin in combination with Cytarabine for acute myeloid leukemia patients who are refractory to or relapsed after induction therapy. Annamycin is a next-generation anthracycline.
Belite Bio presented interim results from a Phase Ib/II trial of LBS-008 in adolescent Stargardt disease (STGD1). BTGD1 is a childhood-onset retinal disease. The drug works by reducing and maintaining the levels of serum retinol binding protein 4 (RBP4).
Aulos Bioscience dosed the first patient in its Phase I/II trial of AU-007 for solid tumors. The drug is a human monoclonal antibody computationally designed by Biolojic Design.
Alzamend Neuro dosed the first patient in its Phase I trial of AL001 for dementia related to Alzheimer’s. AL001 is a novel lithium-delivery system.
Novartis announced positive results from its Phase III Monaleesa-3 trial of Kisqali and fulvestrant in metastatic breast cancer. The combination of Kisqali (ribociclib) and chemotherapy fulvestrant added almost 16 months of survival benefit compared to fulvestrant alone.
Evofem Biosciences announced new data from the Phase III Ampower trial of Phexxi (lactic acid, citric acid, and potassium bitartrate). The women in the trial had fewer urinary tract infections compared to the general population.
Scilex, a Sorrento company, presented Phase II data of Semdexa. The drug is a non-opioid novel injectable corticosteroid gel formulation for the treatment of lumbar radicular pain. The trial hit the primary endpoint of a highly statistically significant reduction in average daily leg pain.