Clinical Catch-Up: Genentech Announces Positive Data, Pfizer Develops New Vaccine

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It was yet another busy week for clinical trial news, including FDA approvals, new indications, trial failures and more. For that and more clinical news, continue reading. 

COVID-19-Related

Beyond Air presented positive safety and efficacy data from the LungFit PRO pilot study of high-concentration inhaled nitric oxide (NO) for Community-Acquired Viral Pneumonia (CAVP), including COVID-19. The treatment was well tolerated overall, with improved efficacy parameters.

Sorrento Therapeutics received clearance from the FDA for its IND for IV STI-9167 (Covishield). This is the IV formulation of the company’s STI-9199 neutralizing antibody against COVID-19.

Veru announced results from the Phase II trial of oral sabizabulin in severe COVID-19 patients at high risk for ARDS. Based on the overwhelming efficacy of a planned interim analysis, the data indicated a clinically meaningful reduction in deaths in those patient populations.

AB Science SA announced the continuation of the Phase II trial of masitinib in combination with isoquercetin in COVID-19 after the recommendation of the DSMB. The drug combination is being evaluated in hospitalized patients with moderate COVID-19 or severe COVID-19. The DSMB recommended continuation without restrictions in moderate patients. The company plans to continue the study only in moderate patients.

Non-COVID-19-Related

Ocugen reported that the independent Data and Safety Monitoring Board (DSMB) for its Phase I/II trial of Ocu400, its gene therapy for Retinitis Pigmentosa (RP), recommended the study proceed with enrolling more patients. The trial is evaluating the modifier gene therapy Ocu400 for RP caused by mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes. It had recently dosed its first patient. The DSMB recommended the company continue enrolling the remaining subjects in the current cohort at the target dose level.

Mustang Bio announced updated interim data from the ongoing Phase I/II trial of MB-106 for r/r B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia. MB-106 is a CD-20 targeted, autologous CAR T cell therapy. The data demonstrated high efficacy and a very favorable safety profile.

Athenex presented data from the Anchor Phase I trial of KUR-502, its allogeneic CD19 CAR-NK T cells in relapsed or refractory lymphoma and leukemia. One NHL patient had stable diseases, and a second with ALL had progressive disease at the four-week assessment. Two complete responses (CRs) persisted for more than six months.

Priothera announced the FDA cleared its IND to begin its pivotal Phase IIb/III trial of mocravimod (Mo-Trans) in acute myeloid leukemia patients undergoing allogeneic hematopoietic stem cell transplant (HSCT). Mocravimod is a synthetic S1PR modulator.

Nkarta announced positive preliminary Phase I data from independent dose finding studies of two of its CAR natural killer (NK) products, Nkx101 and Nkx019. The Nkx101 was in r/r AML and myelodysplastic syndrome patients. Nkx019 was in r/r B cell malignancies, including one patient with aggressive diffuse large B cell lymphoma (DLBCL) and one with mantle cell lymphoma (MCL).

Venus Concept treated the first patient in a U.S. Investigational Device Exemption (IDE) trial of its Aime next-generation robotic technology for the treatment of moderate to severe facial wrinkles. Aime is a robotic platform that provides micro-coring for medical aesthetic applications.

Eliem Therapeutics announced Etx-810 failed to hit its primary endpoint in a Phase IIa trial. The drug is being developed to treat diabetic peripheral neuropathic pain.

Quoin Pharmaceuticals received FDA clearance for its IND for Qrx003 for Netherton Syndrome. Qrx003 is a topical lotion formulated with its proprietary delivery technology. It contains a broad-spectrum serine protease inhibitor designed to perform the function of Lekti.

Bausch & Lomb presented data from the Phase III trial of NOV03 (perfluorohexyloctane), a first-in-class eye drop for dry eye disease associated with Meibomian gland dysfunction (MGD). The drug hit both primary endpoints.

Applied Molecular Transport announced positive topline Phase II results from the Fillmore monotherapy trial of AMT-101 in chronic pouchitis. AMT-101 is a once-daily, GI-selective, oral fusion of IL-10 and AMT’s proprietary carrier molecule.

Rockwell Medical announced its partner, Wanbang Biopharmaceuticals, enrolled the final patient in its Phase III trial for Triferic (ferric pyrophosphate citrate) Dialysate in China. The drug is being tested in hemodialysis patients for maintaining hemoglobin.

Lyra Therapeutics treated the first patient in its Phase II Beacon study of LYR-220 in post-surgical chronic rhinosinusitis patients. The drug is designed to deliver six months of continuous anti-inflammatory medication in the sinonasal passages.

Gamida Cell reported that the FDA had lifted its clinical hold on its cryopreserved formulation of GDA-201. GDA-201 is an off-the-shelf cell therapy candidate to treat follicular and diffuse large B cell lymphomas. The company expects to launch a Phase I/II trial of the drug for those indications this year.

Protagonist Therapeutics reported its Phase II trial of PN-943 for moderate-to-severe ulcerative colitis failed to hit the primary endpoint at a higher dosage. However, patients who received 150 mg had 13% better remission outcomes compared to placebo, and participants receiving the lower dose also achieved 16% better outcomes. The drug is an oral, gut-restricted, alpha-4-beta-7-integrin antagonist.

Pfizer and Valneva announced positive Phase II data of its VLA15, a vaccine to prevent Lyme disease (LD). The trial included 585 participants ages 5 to 17 years and evaluated the immunogenicity and safety of the vaccine after a two or three-dose series.

Novaliq announced data from the second pivotal Phase III Essence-2 trial of CyclASol for dry eye disease. The drug is the first topical preservative-free, water-free, anti-inflammatory and immunomodulating ophthalmic solution, containing 0.1% cyclosporine in a novel vehicle. The trial confirmed the drug has consistent and superior therapeutic effects on the ocular surface in patients with dry eye disease.

Biomind Labs dosed the first patient in its Phase I/IIa trial of BMND01 for treatment-resistant depression. The drug is a N, N-Dimethyltryptamine (DMT) inhalable formulation.

Arrowhead Pharmaceuticals dosed the first patients in the Phase II Gateway study of ARO-ANG3 for patients with homozygous familial hypercholesterolemia (HoFH). The drug is an RNA interference therapeutic designed to silence the hepatic expression of angiopoietin-like protein 3 (ANGPTL3).

Cellectar Biosciences reported an iDMC completed its planned futility/efficacy assessment of its Phase IIb trial of iopofosine in Waldenstrom’s macroglobulinemia and unanimously recommended continuation. The primary endpoint is a major response rate defined as a partial response or better in patients that receive a minimum total body dose of 60 miCi with secondary endpoints of treatment-free survival, duration of response and progression-free survival.

Mundipharma and Cidara Therapeutics presented results from the Phase III restore trial of rezafungin for Candidemia and/or invasive Candidiasis. The drug demonstrated positive efficacy and safety. Rezafungin is a next-generation once-weekly echinocandin.

SOTIO dosed the first patient in its Phase I/II Claudio-01 trial of SOT102 in gastric and pancreatic cancer. The drug is an antibody-drug conjugate.

Avadel Pharmaceuticals presented interim data from its ongoing RESTORE open-label extension/switch trial of FT218. The drug is an investigational formulation of sodium oxybate to be taken once daily at bedtime for excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy.

Shenzhen HighTide Biopharmaceutical dosed the first patient in a Phase II trial of HTD1801 for Type 2 diabetes in China. HTD1801 is a first-in-class new molecular entity with pleiotropic effects that target both the metabolic pathways critical to developing type 2 diabetes and pathogenic factors underlying liver dysfunction and the progression of nonalcoholic fatty liver disease.

Tryp Therapeutics dosed the first patient in a Phase II trial of TRP-8802 in combination with psychotherapy for Binge Eating Disorder (BED). The drug is synthetic psilocybin.

Zymeworks completed enrollment in its Phase III Herizon-BTC-01 trial of zanidatamab monotherapy in patients with previously treated advanced or metastatic HER2-amplified biliary tract cancers (BTC), including gallbladder cancer and cholangiocarcinoma (bile duct cancer). Zanidatamab is a bispecific antibody that simultaneously binds two non-overlapping epitopes of HER2.

Transcenta Holding Ltd. dosed the first patient in China in a Phase I trial of TST002 for osteoporosis. TST002 (blosozumab) is a humanized anti-sclerostin monoclonal antibody.

Alzinova announced the DSMB completed a planned assessment of the ongoing ALZ-191 Phase Ib trial for Alzheimer’s disease. The antibody targets neurotoxic amyloid beta oligomers. The DSMB offered a favorable opinion on the drug’s safety.

Sedana Medical AB enrolled the first patient in its U.S. clinical studies. Two identical Phase III trials, INSPiRE-ICU 1 and 2, are evaluating the efficacy and safety of inhaled sedation with isoflurane delivered via Sedaconda ACD.

BioArctic and Eisai published data suggesting that treatment with its lecanemab slowed the rate of disease progression in Alzheimer’s disease. The drug is an anti-amyloid-beta protofibril antibody.

BioCorRx initiated enrollment for its Phase I trial of BICX104 for opioid use disorder (OUD). The drug is an implantable, biodegradable naltrexone pellet.

AnaptysBio announced positive topline data from a Phase I trial of ANB032. The drug is an anti-BTLA agonist antibody. The data demonstrated favorable safety, tolerability and a rapid and sustained PK and PD profile.

Harmony Biosciences initiated a Phase III trial of pitolisant in adults with idiopathic hypersomnia (IH). IH is a rare and chronic neurological disease marked by excessive daytime sleepiness. Wakix is a first-in-class drug approved by the FDA for excessive sleepiness or cataplexy in adults with narcolepsy. It is a selective histamine 3 receptor antagonist/inverse agonist.

Innate Pharma announced AstraZeneca dosed the first patient in its Phase III PACIFIC-9 trial of durvalumab with monalizumab or oleclumab in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who have not progressed after definitive platinum-based concurrent chemoradiation therapy (CRT). Monalizumab is Innate’s lead partnered asset, a potentially first-in-class immune checkpoint inhibitor targeting NKG2A receptors expressed on tumor-infiltrating cytotoxic CD8+ T cells and NK cells. Durvalumab is AstraZeneca’s Imfinzi, an anti-PD-L1 checkpoint inhibitor. Oleclumab is AstraZeneca’s experimental anti-CD73 monoclonal antibody.

Genentech announced new three-year data from its Firstfish study, including one-year data from the open label extension of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed about 91% of infants treated with the drug were alive after three years of treatment.

Finch Therapeutics received approval from the FDA to continue its Phase III Prism4 trial of CP101 for recurrent C. difficile infections. The hold was issued in February, which wanted more details on how COVID-19 donor samples would be shipped to the vendor.

Lyra Therapeutics presented additional new data from its Lantern Phase II trial of LYR-210 in adults with chronic rhinosinusitis. LYR-210 utilizes the company’s proprietary XTreo platform to enable six months of local, intra-nasal, anti-inflammatory therapy. It is a bioresorbable polymeric matrix that provides up to six months of continuous mometasone furoate therapy.

CytoSorbents enrolled the first patient in the DrugSorb-ATR Antithrombotic Removal System for intraoperative removal of apixaban and rivaroxaban during the STAR-D trial cardiothoracic surgery. Apixaban and rivaroxaban are direct oral anticoagulants.

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