Clinical Catch-Up: September 28-October 2

Clinical Trial Concept_Compressed

Biopharma companies wrapped up September and headed into October with plenty of clinical trial news. Here’s a look.

COVID-19-Related

Regeneron Pharmaceuticals announced early data from its Phase I/II/III trial of its antibody cocktail, REGN-COV2, against COVID-19. The analysis demonstrated the cocktail of two antibodies against SARS-CoV-2 decreased viral load and alleviated symptoms faster in non-hospitalized COVID-19 patients. It also demonstrated positive trends in decreasing medical visits. The study is still ongoing.

Moderna published second interim analysis of its open-label Phase I trial of its COVID-19 vaccine, mRNA-1273, in The New England Journal of Medicine. This particular data focused on two age cohorts, one ages 56-70 and the other 71+. It presented results through Day 57, which was one month after the second dose. The vaccine requires two doses 28 days apart. This data was an extension of the company’s Phase I safety trial, which was conducted in people aged 18 to 55. It evaluated two doses of the vaccine, 25 micrograms and 100 micrograms. The extension study evaluated 40 adults. The results indicated that the older volunteers who received two injections of the 100-microgram dose 28 days apart produced immune responses about equivalent to what was observed in younger adults.

Covaxx dosed the first healthy volunteers in its Phase I trial of UB-612, its vaccine candidate for COVID-19. The study began in Taiwan. It will test the vaccine in 60 healthy male and female adults from 20 to 55 years of age in three groups of 20 subjects.

FSD Pharma received the go-ahead from the FDA to initiate a Phase II trial of its FSD201 (ultramicronized palmitoylethanolamide, or ultramicronized PEA) to treat COVID-19. Dosing is expected to start in October. They believe the therapy can address the “over-exuberant inflammatory response” that leads to a cytokine storm.

Johnson & Johnson posted an interim analysis of its Phase I/II trial of its COVID-19 vaccine candidate, JNJ-78436735, demonstrating a strong neutralizing antibody response in almost all participants. The vaccine was also well-tolerated.

Inovio Pharmaceuticals reported the FDA had placed a partial clinical hold on its planned Phase II/III trial of its COVID-19 vaccine candidate, INO-4800. The FDA had additional questions about the candidate itself as well as the Cellectra 2000 delivery device. It wasn’t related to any adverse events and the Phase I trial is expected to continue.

Windtree Therapeutics announced the FDA had accepted its IND application for a Phase II trial of lyo lucinactant, the company’s KL4 surfactant drug in COVID-19 associated lung injury and ARDS patients. The company hopes to launch the study in four to five U.S. sites and begin in the next several weeks.

Non-COVID-19-Related

Biogen presented new data from the Spinraza (nusinersen) clinical development program. New data from the ENDEAR/SHINE and NURTURE studies reinforced the importance of early spinal muscular atrophy (SMA) treatment. They also announced enrollment is completed for Part A of the DEVOTE trial, which is evaluating a higher dose of Spinraza.

Bristol Myers Squibb presented Phase III data from its Opdivo (nivolumab) in muscle-invasive urothelial carcinoma (MIUC). Opdivo is the company’s immune checkpoint inhibitor. The trial, CheckMate-274 compared Opdivo against placebo in 709 patients at high risk of recurrence after radical surgery. In the interim analysis, the primary endpoints of disease-free-survival (DFS) in all patients and in a subset of PD-L1 expressing patients were both met.

PTC Therapeutics announced two-year data from Part 1 of the FIREFISH trial of Evrysdi (risdiplam) in spinal muscular atrophy (SMA). The study showed continued improvement and achievement of motor milestones. The drug was approved in August for SMA patients two months and older.

MeiraGTx Holdings announced nine-month results from the ongoing Phase I/II trial of AAV-RPGR for X-linked retinitis pigmentosa (XLRP). The drug is being jointly developed by MeiraGTx and Janssen Pharmaceuticals, a J&J company. AAV-RPGR is an investigational gene therapy in XLRP caused by mutations in the eye specific form of the RPGR gene (RPGR ORF15). It delivers functional copies of the RPGR gene to the subretinal space.

Junshi Biosciences announced that an Independent Monitoring Committee (IDMC) decided its Phase III JUPITER-02 trial had met its pre-specified primary endpoint at the interim analysis. The trial was evaluating their PD-L1 checkpoint inhibitor toripalimab in combination with gemcitabine/cisplatin as a first-line treatment for recurrent or metastatic nasopharyngeal carcinoma (NPC). The drug significantly extended progression-free survival over gemcitabine/cisplatin alone.

Arcutis Biotherapeutics announced topline data from its Phase II trial of ARQ-154 (topical roflumilast foam) for seborrheic dermatitis. Dosing the therapy once a day for eight weeks demonstrated statistically significant improvement compared to a placebo on key efficacy endpoints in patients with moderate-to-severe seborrheic dermatitis, including the primary endpoint, which showed an Investigator Global Assessment success rate of 73.8% compared to 40.9% for vehicle.

Celyad Oncology is collaborating with Merck & Co. on a Phase Ib KEYNOTE-B79 clinical trial of Celyad’s non-gene edited allogeneic CAR-T candidate, CYAD-101, following FOLFIRI preconditioning chemotherapy, with Merck’s checkpoint inhibitor Keytruda (pembrolizumab) in refractory metastatic colorectal cancer (mCRC) with microsatellite stable (MSS)/mismatch-repair proficient (pMMR) disease.

Genentech released two-year data from Part 1 of the pivotal FIREFISH trial of Evrysdi (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The data demonstrated continued improvement and achievement of motor milestones.

Myovant Sciences announced additional secondary subgroup endpoint data of its Phase III HERO trial of relugolix in advanced prostate cancer. The drug did not achieve statistical superiority for castration resistance-free survival compared to leuprolide acetate in men with metastatic disease through 48 weeks. The drug is under Priority Review by the FDA for the treatment of men with advanced prostate cancer.

iLiAD Biotechnologies presented positive topline data from its Phase IIb trial of BPZE1, a vaccine against pertussis (whooping cough). The vaccine met both primary endpoints of overall safety and induction of mucosal immunity.

TargetCancer Foundation and Bayer in partnership with Foundation Medicine enrolled the first patients in the TCF-001 TRACK Study. TRACK’s goal is to provide individualized treatment recommendations to patients with rare cancers, informed by genomic analysis and in consultation with leading rare cancer clinicians and researchers. All the participants’ treatments and responses will be tracked prospectively for at least one year, and patient outcomes will be analyzed based on molecularly-informed treatment decisions.

IntraBio announced positive data from its multinational clinical trial of IB1001 for Niemann-Pick disease Type C (NPC). The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), as well as topline secondary endpoints, such as improvement in symptoms, functioning, and quality of life. IB1001, N-acetyl-L-leucine, is an oral modified amino acid which can restore neuronal function and protect against or delay disease progression in multiple neurological circuits in the brain.

Solid Biosciences announced the FDA had lifted the clinical hold on its IGNITE DMD Phase I/II clinical trial. It was placed on hold in November 2019 over safety concerns. The trial is evaluating SGT-001, a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy for Duchenne muscular dystrophy (DMD). SGT-001 delivers microdystrophin, a synthetic dystrophin gene, that encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins.

Athira Pharma dosed the first patient in its Phase II/III LIFT-AD trial of ATH-1017 for mild-to-moderate Alzheimer’s disease. ATH-1017 is a small molecule designed to enhance the activity of Hepatocyte Growth Factor (HGF) and its receptor, MET, which are expressed in normal CNS function.

Selecta Biosciences and Swedish Orphan Biovitrum (Sobi) announced topline data for the Phase II COMPARE trial of SEL-212 for chronic refractory gout. SEL-212 is a combination of Selecta’s ImmTOR immune tolerance platform and a therapeutic uricase enzyme (pegadricase) and was compared to Krystexxa (pegloticase). The data showed a numerically higher response rate for 3 to 6 months combined on the primary endpoint but did not meet the primary endpoint of statistical superiority; it did show a statistically significant higher response rate during month 3, and numerically higher response rate during month 6. It demonstrated a statistically significant greater overall reduction in mean SUA levels.

Imago Biosciences dosed the first patient in the Phase IIb trial of bomedemstat for essential thrombocythemia (ET). Bomedemstat is an oral small molecule that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme vital in cancer stem/progenitor cells.

Apellis Pharmaceuticals presented post hoc analysis of the Phase II trial of intravitreal pegcetacoplan (APL-2) for treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The data indicated the drug may have the potential to slow the progression of nascent GA, the early stage of the disease that precedes atrophy, in areas of the retina outside of GA lesions. Pegcetacoplan is a targeted C3 therapy designed to regulate excessive complement activation.

Windtree Therapeutics dosed the first patient in its Phase II trial of istaroxime in patients experiencing early cardiogenic shock. Istaroxime is a first-in-class dual mechanism drug designed to improve both systolic and diastolic cardiac function. It is a positive inotropic agent that increases myocardial contractility through inhibition of Na+/K+-ATPase with a complimentary mechanism that facilitates myocardial relaxation through the activation of the SERCA2a calcium pump, on the sarcoplasmic reticulum enhancing calcium reuptake from the cytoplasm.

Otonomy completed enrollment in its Phase III trial of Otividex in Menieres disease. Meniere’s disease is a disorder of the inner ear that can lead to vertigo and hearing loss. Otividex is a sustained-exposure formulation of the steroid dexamethasone.

AiViva Biopharma began dosing patients with AIV001 for its Phase I/II trial for superficial or nodular basal cell carcinoma (BCC). AIV001 is an intradermally administered formulation of a multi-kinase inhibitor using the company’s proprietary delivery technology.

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