On May 2, 2022 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) ("Aptorum Group" or "Aptorum"), a clinical-stage biopharmaceutical company, reported the finalized data from the Phase 1 clinical trial of SACT-1, a repurposed small molecule drug targeting Neuroblastoma and potentially other cancer types (Press release, Aptorum, MAY 2, 2022, View Source [SID1234613322]).

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Following the announcement of the Phase 1 clinical trial of SACT-1 in January 2022, Aptorum is pleased to announce further data updates from the trial conducted by an independent clinical contract research organization. The Phase 1 clinical trial of SACT-1 was an open-label, randomized, 3-period, 3-sequence, single-dose crossover bioavailability and food effect study of SACT-1 (oral suspension) in healthy adult volunteers. The primary objective of this study was to assess the relative bioavailability of 150 mg of SACT-1 (oral suspension) under fasted and fed conditions. The secondary objectives were to evaluate the safety, tolerability and any potential QT prolongation after a single oral administration of 150mg of the studied drug under fasted and fed conditions in healthy adult subjects. The study treatments were well tolerated and no subjects were discontinued from study participation because of adverse events. No serious adverse events were reported during the study. The phase 1 clinical data also suggested that any QT interval after oral administration of SACT-1 at 150mg was well within clinically acceptable limits. Regarding the relative bioavailability under the Fed vs Fasted condition, the AUC0-tlast, AUC0-∝ and Cmax ratio of SACT-1 were determined to be 189.87%, 189.43%, and 205.25% respectively.

Dr. Clark Cheng, Chief Medical Officer and Executive Director of Aptorum Group, commented: "Further to our previous announcements, we are very encouraged by the impressive safety data even at a relatively high dosage. The relative bioavailability data also enabled us to estimate the starting dose for pediatric neuroblastoma patients via PK modeling. We are planning to meet with the US FDA for an end of Phase 1 meeting as soon as possible and are targeting for submission for a Phase 1b/2a clinical trial in neuroblastoma patients."

About SACT-1

SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma. SACT-1’s mechanism has been investigated in our preclinical studies to enhance tumor cell death and suppress MYCN expression (a common clinical diagnosis in high-risk or relapsed neuroblastoma patients where an amplification of MYCN is usually observed). SACT-1 is designed to be used especially in combination with standard-of-care chemotherapy.

On May 2, 2022 Johnson & Johnson (NYSE: JNJ) reported that it will participate in the Bernstein 38th Annual Strategic Decisions Conference on Wednesday, June 1st, at the New York Hilton Midtown in New York (Press release, Johnson & Johnson, MAY 2, 2022, View Source;johnson-to-participate-in-the-bernstein-38th-annual-strategic-decisions-conference-301537487.html [SID1234613338]). Joaquin Duato, Chief Executive Officer will represent the Company in a session scheduled at 10:00 a.m. (Eastern Time).

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This webcast will be available to investors and other interested parties by accessing the Johnson & Johnson website at www.investor.jnj.com.

A webcast replay will be available approximately 48-hrs after the live webcast.

On May 2, 2022 Boston Scientific Corporation (NYSE: BSX) reported that it will participate in Bank of America’s Global Healthcare Conference on Thursday, May 12 (Press release, Boston Scientific, MAY 2, 2022, View Source [SID1234613275]).

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Dan Brennan, executive vice president and chief financial officer, and Lauren Tengler, vice president, Investor Relations, will participate in a 30-minute question-and-answer session with the host analyst at approximately 8:00 a.m. PT. A live webcast of the session will be available on the Investor Relations section of the Boston Scientific website at investors.bostonscientific.com.

The replay of the webcast will be accessible at investors.bostonscientific.com beginning approximately one hour following the completion of the event.

On May 2, 2022 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported two abstracts have been accepted for presentation at the American Society of Gene and Cell Therapy ("ASGCT") 25th Annual Meeting, taking place virtually and in person in Washington, D.C., May 16-19, 2022 (Press release, NeuBase Therapeutics, MAY 2, 2022, View Source [SID1234613291]).

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NeuBase will present new preclinical data on the biodistribution in key tissues of the company’s lead candidate, NT-0231.F, a precision genetic medicine in development to treat myotonic dystrophy, type 1 ("DM1"). NeuBase recently presented data at the 2022 MDA Clinical & Scientific Conference demonstrating that systemic administration of NT-0231.F in the HSALR model achieves clinically relevant molecular and functional rescue, including rescue of the spliceopathy, resolution of nuclear aggregates, and reversal of myotonia (delayed muscle relaxation after contraction). Pharmacokinetic studies of NT-0231.F in wild-type BALB/C mice showed a single IV or SC dose achieved high volume of distribution.

The presentations are listed below, and the full abstracts are available on the ASGCT (Free ASGCT Whitepaper) meeting website. All times are listed in Eastern Time (ET).

Title: Pharmacokinetics, Biodistribution, and CNS Penetration of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy-Type 1 Following Systemic Administration Systemic Administration in Mice
Presenter: Noel Monks, Ph.D.
Session Date/Time: Tuesday, May 17, 5:30 PM – 6:30 PM
Poster Board Number: Tu-90
Session Title: Oligonucleotide Therapeutics II
Room: Hall D
Abstract Number: 585

Title: Pharmacology, Biodistribution and Tolerability of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy, Type 1
Presenter: Sandra Rojas-Caro, M.D.
Session Date/Time: Wednesday, May 18, 5:30 PM – 6:30 PM
Poster Board Number: W-175
Session Title: Musculo-skeletal Diseases
Room: Hall D
Abstract Number: 1049

About NeuBase’s DM1 Program
Patients with DM1 suffer from cognitive deficits and muscle pathology caused by a trinucleotide expansion in the DMPK gene which, when transcribed, results in an RNA hairpin structure that sequesters RNA splice proteins. NeuBase’s DM1 investigational genetic therapy, NT-0231.F, targets mutant DMPK pre-mRNA with a novel peptide-nucleic acid ("PNA") pharmacophore and is designed to selectively engage with the toxic RNA hairpin structure, release the splicing proteins, and restore RNA splicing and downstream protein production. The PNA pharmacophore is conjugated to NeuBase’s novel delivery technology that is designed for broad distribution, including into the deep brain, with the potential for a whole body, disease-modifying solution for DM1. For more information, visit www.neubasetherapeutics.com/pipeline/.

On May 2, 2022 Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing and commercializing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, reported topline results from the six-week, Phase 2a, open-label, single-arm AESOP trial of avasopasem evaluating its ability to reduce the incidence of severe acute radiation-induced esophagitis in patients with lung cancer receiving concurrent chemoradiotherapy (Press release, Galera Therapeutics, MAY 2, 2022, View Source [SID1234613307]).

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The multicenter Phase 2a trial enrolled 39 patients (62 screened) with unresectable Stage 3A/3B or post-operative Stage 2B non-small cell (NSCLC) or limited-stage small cell (SCLC) lung cancers. Thirty-five patients completed treatment with 60 gray of intensity-modulated radiation therapy (IMRT) plus chemotherapy over six weeks. Of these 35 patients, 29 received at least five weeks of 90 mg of avasopasem on the days they underwent IMRT. These 29 patients were evaluated as the pre-specified per protocol population. Patients enrolled in this trial were considered at high risk for developing esophagitis due to the amount of radiation planned to be delivered to the esophagus.1 Patients were assessed and classified according to NCI-CTCAE criteria.2

Incidence of esophagitis by grade and timepoint in the AESOP trial (per protocol, n=29):

Grading Scale for Esophagitis per NCI Criteria
Grade 1 Asymptomatic; clinical or diagnostic observations only; intervention not indicated
Grade 2 Symptomatic; altered eating/swallowing; oral supplements indicated
Grade 3 Severely altered eating/swallowing; tube feeding, TPN, or hospitalization indicated
Grade 4 Life-threatening consequences; urgent operative intervention indicated
Grade 5 Death
Only two of the 29 patients (7%) experienced Grade 3 esophagitis at any time, with neither patient experiencing Grade 3 for more than one week. No patients experienced Grade 4 or 5 esophagitis at any point during the trial. These data compare favorably to the literature in which approximately 20-30 percent of these patients experienced Grade 3 or 4 esophagitis.3 Avasopasem was generally well tolerated. The adverse events experienced are comparable to those expected with chemoradiotherapy.

"These encouraging results demonstrate avasopasem’s potential to meaningfully reduce radiotherapy-induced Grade 3 or worse esophagitis," said Mel Sorensen, M.D., Galera’s President and CEO. "Patients with lung cancer undergoing chemoradiotherapy are at high risk of severe and potentially life-threatening esophagitis, including an inability to eat or swallow, severe pain, ulceration, infection, bleeding and weight loss, and there are no established drug therapies. Following the positive Phase 3 results of avasopasem in radiotherapy-induced severe oral mucositis (SOM), we believe these results in esophagitis support the safety and efficacy of avasopasem as a potential therapy to prevent the most severe forms of radiotherapy-induced toxicities."

Approximately 50,000 lung cancer patients undergo standard-of-care chemoradiotherapy every year in the U.S. and are at risk of developing esophagitis.

About Radiotherapy-Induced Esophagitis
Radiotherapy-induced esophagitis is a common and debilitating adverse effect that develops in patients receiving radiotherapy, most commonly for lung, esophageal, breast or head and neck cancers or for lymphoma. Radiotherapy-induced esophagitis is inflammation, edema, erythema, and erosion of the mucosal surface of the esophagus caused by radiotherapy. Esophagitis can be life-threatening, and symptoms include an inability to swallow, severe pain, ulceration, infection, bleeding and weight loss and may require hospitalization. There are currently no FDA-approved drugs and no established guidelines for the treatment of radiotherapy-induced esophagitis.

About Avasopasem
Avasopasem manganese (avasopasem, or GC4419) is a selective small molecule dismutase mimetic in development for the reduction of radiation-induced severe oral mucositis (SOM) in patients with locally advanced head and neck cancer (HNC) and for the reduction of radiation-induced esophagitis in patients with lung cancer. The FDA has granted Fast Track and Breakthrough Therapy designations to avasopasem for the reduction of SOM induced by radiotherapy, with or without systemic therapy.

About the Phase 2a AESOP Trial
The AESOP trial is an open-label, multicenter trial designed to evaluate the ability of avasopasem to reduce the incidence of radiotherapy-induced esophagitis in patients receiving chemoradiotherapy for unresectable Stage 3A/3B or post-operative Stage 2B non-small cell lung cancer, or small cell lung cancer treatable with chemoradiotherapy. For more information, please visit View Source