On September 30, 2022 Aptorum Group Limited (NASDAQ: APM, Euronext Paris: APM) ("Aptorum Group" or the "Company"), a clinical stage biopharmaceutical company dedicated to meeting unmet medical needs in oncology, autoimmune and infectious diseases, reported that financial results for the six months ended June 30, 2022 (Press release, Aptorum, SEP 30, 2022, View Source [SID1234621593]).

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"During the first half of 2022, we remained focused on advancing the development of our therapeutic programs. As announced in early 2022, both our SACT-1 program (targeting neuroblastoma) and ALS-4 program (targeting MRSA), has announced the completion and positive Phase I clinical trial results. Our RPIDD program (liquid biopsy based approach to infectious disease rapid molecular diagnostics) are also progressing well into its clinical validation studies. We are excited to work towards the commencement of the relevant phase II clinical trials for our ALS-4 and SACT-1 programs and the continuation of the clinical validation of our RPIDD program. Finally, we are also pleased to have announced the commercialization of our NativusWell nutraceutical product, a novel supplement targeting woman’s health including menopausal symptoms, commencing in Hong Kong initially and to be followed into the European and US markets in due course," said Mr. Darren Lui, Chief Executive Officer and Executive Director of Aptorum Group Limited.

Clinical Pipeline Update and Upcoming Milestones

In January 2022, Aptorum Group announced the completion of the Phase I clinical trial for ALS-4,, a small molecule drug targeting methicillin-resistant staphylococcus aureus related infections. The Phase I clinical trial of ALS-4 was a randomized, double-blinded, placebo-controlled, single and multiple ascending dose study designed to evaluate safety, tolerability, and pharmacokinetics or orally administered ALS-4 in healthy male and female adult volunteers. Currently, the Company is preparing the relevant submission to the US FDA to initiate a Phase 2 clinical trials for skin and soft tissue infections and/or bacteraemia. The study treatments were well tolerated and no serious adverse events were observed and no relevant clinical changes in respect of vital signs and clinical laboratory test results.

In May 2022, Aptorum Group announced the finalized data from the Phase I clinical trial of SACT-1, a repurposed small molecule drug targeting Neuroblastoma and potentially other cancer types. The Phase I 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 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. SACT-1 also received Orphan Drug Designation from the US FDA.

In September 2022, Aptorum Group announced further results and updates from its RPIDD’s analytical and clinical validations in patient samples. It was further announced the company has taken steps to initiate the set up of a clinical laboratory in the state of California, which is targeted to require relevant CLIA certification.

In September 2022, Aptorum Group announced the commencement of sale of its NativusWell nutraceutical product targeting woman’s health and the launch of it’s initial sales launch in Hong Kong, to be followed in Europe and United States.

Corporate Highlights

In May 2022, Mr. Darren Lui has been appointed as the Chief Executive Officer. Mr. Lui was previously the Aptorum Group Limited’s President, successfully brought two small molecule drugs (ALS-4 and SACT-1) from discovery to their respective completion of two phase I clinical trials and currently in preparation for their next phase of clinical trials in human patients, as well as conducting ongoing clinical validations of the Company’s RPIDD program, a liquid biopsy based molecular diagnostics for infectious diseases.

In July 2022, Mr. Martin Siu has been appointed as the Head of Finance. Mr. Siu has over 19 years’ experience in the field of audit and regulatory consultancy and has supported a number of listed companies over the years.

Financial Results for the Six Months Ended June 30, 2022

Aptorum Group reported a net loss of $2.7 million for the six months ended June 30, 2022 compared to $17.1 million for the same period in 2021. The decrease in net loss in the current period was driven by an decrease of loss on investments in marketable securities, net of $7.5 million, and there was a gain on non-marketable investment of $5.6 million in 2022 while there was no such gain in the same period in 2021.

Research and development expenses were $4.5 million for the six months ended June 30, 2022 compared to $5.5 million for the same period in 2021. The decrease in research and development expenses was mainly due to the decrease in services provided by contracted research organizations as we are in a planning process for Phase 2 of our lead projects which does not require many services from contracted research organizations.

General and administrative fees were $2.4 million for the six months ended June 30, 2022 compared to $2.6 million for the same period in 2021. The decrease in general and administrative fees was primary due to the decrease in number of employees.

Legal and professional fees were $1.4 million for the six months ended June 30, 2022 compared to $1.2 million for the same period in 2021. The increase in legal and professional fees was mainly due to more share based compensation awards was granted for consultancy services when compared to the same period in 2021.

As of June 30, 2022, cash and restricted cash totalled approximately $7.2 million and total equity was approximately $15.6 million.

Aptorum Group expects that its existing cash and restricted cash together with undrawn line of credit facility from related parties, will enable it to fund its operating and capital expenditure requirements for at least the next 12 months.

On September 30, 2022 Hansa Biopharma AB (NASDAQ Stockholm: HNSA) reported that it will publish its interim report for January-September 2022 at 8:00 CET on October 20, 2022 (Press release, Hansa Biopharma, SEP 30, 2022, https://www.prnewswire.com/news-releases/hansa-biopharma-to-host-conference-call-to-provide-interim-results-for-january-september-2022-and-business-update-301637644.html [SID1234621592]). All interested parties are invited to participate in a telephone conference, which will include a presentation of the interim results and a business update, on the same date at 14:00 CET/8:00am EST. The event will be hosted by Hansa Biopharma’s CEO, Søren Tulstrup, and CFO, Donato Spota, and the presentation will be held in English.

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Slides used in the presentation will be live on the company website during the call under "Events & Presentations" and will also be made available online after the call. Link to presentation

On September 30, 2022 e-therapeutics plc (AIM: ETX) (OTC-QX: ETXPF), a company integrating computational power and biological data to discover life-transforming RNAi medicines, reported a fundraise of £13.5 million before expenses by way of a subscription for new ordinary shares of 0.1p each ("Ordinary Shares") in the Company (the "Subscription") at a price of 20p per Ordinary Share by funds managed by M&G Investment Management Limited ("M&G"), an institutional investor and an existing shareholder of the Company (Press release, e-Therapeutics, SEP 30, 2022, View Source [SID1234621591]).

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The fundraise provides ETX with the opportunity to generate value and accelerate the next stage of its growth, advancing the Company’s position in creating an entirely new template for drug discovery using computation to capture and model disease complexity, identify novel targets and design RNAi drugs against those targets that can be rapidly progressed to the clinic.

The net proceeds of the Subscription will be used to facilitate a number of initiatives to accelerate growth, with a focus on expanding the Company’s in-house pipeline of first-in-class RNAi candidates derived from ETX’s computational platform; further developing cell type-specific computational tools and datasets; and general working capital including additional headcount.

Ali Mortazavi, Chief Executive Officer of e-therapeutics, commented:

"I am pleased to announce the fundraise of £13.5 million and excited by the prospect of being able to accelerate the development of our in-house RNAi pipeline through enhanced investment in our therapeutic programmes, hepatocyte datasets and computational capabilities. This successful fundraise underlines ETX’s position at the intersection of computational approaches to drug discovery and genetic medicine, using RNA interference as our drug modality of choice. We are grateful for the support of our shareholders and look forward to delivering value from our platform technologies."

Michael Stiasny, Head of UK Equities at M&G Investments, commented:

"With the potential to re-shape the conventional drug discovery model, siRNA based therapies represent an extremely exciting new modality in medicine. We believe that e-therapeutics has a unique platform and strategically attractive IP in this space, combined with a strong computational edge, and are delighted to be increasing our long-term support for the Company."

The issue of new Ordinary Shares pursuant to the Subscription will be conditional on (i) the 67,500,000 shares ("Subscription Shares") being admitted to trading on AIM by not later than 8.00 a.m. on 6 October 2022, or such later time and/or date as the Company may agree (being not later than 8.00 a.m. on 14 October 2022) and (ii) the representations and warranties of the Company under the Subscription being true and accurate.

Highlights of the Fundraise

A total fundraise of £13.5 million before expenses, through the issue of the Subscription Shares at a price of 20p per Ordinary Share (the "Subscription Price"), equating to approximately 13.12% of the existing ordinary share capital
The Subscription Price represents a premium of approximately 12% to the closing middle market price of 17.825p per Ordinary Share on 29 September 2022, being the latest practicable date prior to the date and time of this announcement
The net proceeds of the Subscription will be used to facilitate a number of initiatives, with a focus on expanding the Company’s proprietary drug discovery and development platform capabilities and pipeline of computationally driven first-in-class RNAi medicines
The issue of the Subscription Shares pursuant to the Subscription will be conditional on (i) the Subscription Shares being admitted to trading on AIM by not later than 8.00 a.m. on 6 October 2022, or such later time and/or date as the Company may agree (being not later than 8.00 a.m. 14 October 2022) and (ii) the representations and warranties of the Company under the Subscription being true and accurate
M&G has the right to nominate a non-executive director to the board of ETX if it so wishes for so long as M&G holds the higher of at least 58,000,000 shares of the Company or 5% of the existing issued share Capital of the Company from time to time
Company Overview

The latest information on the Company and its recent progress is included in its Interim Report for the 6 months to 31 July 2022 which is also being announced today.

ETX is a UK-based company integrating computational power and biology information to discover life-transforming RNAi medicines. The Company’s technology uses computation to capture and model human biology, identify novel targets and design RNAi medicines against those targets that can be rapidly progressed to the clinic.

ETX’s proprietary Computational Biology Platform enables the generation and analysis of biological network models, providing a novel and mechanistic approach to drug discovery that explicitly considers the true complexity of biology and makes more reliable predictions from large complex data sets and ETX’s proprietary hepatocyte knowledge base, – the world’s most comprehensive and integrated hepatocyte-centric data and information resource. The Company generates, prioritises and tests millions of hypotheses in silico to identify better therapeutic targets with higher confidence.

ETX’s proprietary RNAi Platform enables the targeted delivery to hepatocytes in the liver and the specific silencing of novel disease-associated genes, identified by ETX’s Computational Biology Platform. The focus on hepatocytes offers the opportunity to work across a wide variety of diseases. The liver is a highly metabolically active organ which performs a key role in many biological processes and vital functions crucial for human health. ETX’s GalNAc-siRNA constructs have demonstrated compelling in vivo performance in terms of depth of gene silencing and duration of action.

ETX is progressing a pipeline of first-in-class pre-clinical RNAi candidates in several therapeutic areas including haematology, cardiovascular disease and non-alcoholic steatohepatitis ("NASH"). ETX has also partnered with biopharma companies such as Novo Nordisk, Galapagos NV and iTeos Therapeutics using its computational network biology approach across a diverse range of drug discovery projects.

On September 30, 2022 AIkido Pharma Inc. (NASDAQ: AIKI) ("AIkido" or the "Company") reported an update on the $3 million share repurchase program (the "Share Repurchase Program") authorized by the Company’s Board of Directors on January 21, 2022 (Press release, AIkido Pharma, SEP 30, 2022, View Source [SID1234621590]).

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In the framework of the Share Repurchase Program, the Company has repurchased 303,886 common shares in the period from January 21, 2022 up to and including September 29, 2022. The price per share range for the shares repurchased was $5.34 to $6.97.

Additional shares may be repurchased from time to time in open market transactions, or other means in accordance with Rule 10b5-1 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), and Rule 10b -18 of the Exchange Act. The timing, number of shares repurchased, and prices paid for the stock under this program will depend on general business and market conditions as well as corporate and regulatory limitations, including blackout period restrictions.

On September 30, 2022 Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. reported that the U.S. Food and Drug Administration (FDA) has approved LYTGOBI tablets for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements (Press release, Taiho, SEP 30, 2022, View Source [SID1234621589]).

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This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

"LYTGOBI is an effective, well-tolerated therapy for patients with intrahepatic CCA that can be taken orally," said Tim Whitten, President and CEO of Taiho Oncology, Inc. "This approval is an important milestone for patients and may provide hope for improved outcomes. As someone whose family has been impacted by cholangiocarcinoma, I’m acutely aware of the impact this disease can have on the patient and their loved ones."

As a whole, cholangiocarcinoma is an aggressive cancer of the bile ducts and is diagnosed in approximately 8,000 individuals each year in the U.S.1 This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) forms of the disease. Approximately 20% of patients diagnosed with CCA have the intrahepatic form of the disease.2,3 Within this 20%, approximately 10-16% of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation.4,5,6,7,8 LYTGOBI covalently binds to FGFR2 and inhibits the signaling pathway.9 The other approved FGFR inhibitors are reversible ATP-competitive inhibitors.10,11,12

"LYTGOBI is a key example of the potential of precision medicine in iCCA and represents another advance in the treatment of this rare and challenging disease," said medical oncologist Lipika Goyal, MD, MPhil, of the Massachusetts General Hospital Cancer Center and lead investigator of the pivotal study that led to the approval of LYTGOBI. "I am encouraged that treatment options continue to expand and evolve for this disease through the dedicated efforts of many over several years."

The approval of LYTGOBI is based on the results of the primary analysis of the FOENIX*-CCA2 trial, a global Phase 2 open-label trial evaluating 103 patients with unresectable, locally advanced or metastatic iCCA harboring FGFR2 gene rearrangements including fusions. In this trial, patients received LYTGOBI orally once daily at a dose of 20mg until disease progression or unacceptable toxicity.

The trial met its primary endpoint with an objective response rate of 42% as measured by independent central review. The median duration of response (DOR) was 9.7 months, with 72% of responses lasting at least six months. The most common (≥20%) adverse reactions were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, dry skin, arthralgia, dysgeusia, abdominal pain, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting.

LYTGOBI was discovered by Taiho Oncology’s parent company, Taiho Pharmaceutical, which continues to co-develop this product for other potential tumor types. "The Taiho group is working as one to optimize this agent for the patients who are waiting," said Teruhiro Utsugi, Senior Managing Director at Taiho Pharmaceutical.

About LYTGOBI
INDICATION AND USAGE
LYTGOBI is indicated for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Ocular Toxicity: Retinal Pigment Epithelial Detachment (RPED), which may cause symptoms such as blurred vision, occurred in 9% of 318 patients who received LYTGOBI across clinical trials. The median time to first onset of RPED was 40 days. Perform a comprehensive ophthalmological examination, including optical coherence tomography (OCT) of the macula, prior to initiation of therapy, every 2 months for the first 6 months, and every 3 months thereafter. For onset of visual symptoms, refer patients for ophthalmologic evaluation urgently, with follow-up every 3 weeks until resolution or discontinuation of LYTGOBI. Withhold or reduce the dose of LYTGOBI as recommended. Dry Eye/Corneal Keratitis: Among 318 patients who received LYTGOBI across clinical trials, dry eye occurred in 15% of patients. Treat patients with ocular demulcents as needed.
Hyperphosphatemia and Soft Tissue Mineralization: Hyperphosphatemia, which can cause soft tissue mineralization, calcinosis, nonuremic calciphylaxis, and vascular calcification was reported in 88% of 318 patients treated with LYTGOBI across clinical trials with a median time of onset of 5 days (range 3-117). Phosphate binders were received by 77% of patients who received LYTGOBI. Monitor for hyperphosphatemia throughout treatment. Initiate a low-phosphate diet and phosphate-lowering therapy when serum phosphate level is ≥5.5 mg/dL; initiate or intensify phosphate-lowering therapy when >7 mg/dL; reduce dose, withhold, or permanently discontinue LYTGOBI based on duration and severity of hyperphosphatemia.
Embryo-fetal Toxicity: LYTGOBI can cause fetal harm. Advise pregnant women of the potential risk to the fetus. Advise female patients of reproductive potential, and males with female partners of reproductive potential, to use effective contraception during treatment with LYTGOBI and for 1 week after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in 39% of patients receiving LYTGOBI, and in ≥2% of patients included pyrexia, gastrointestinal hemorrhage, ascites, musculoskeletal pain, and bile duct obstruction.
The most common adverse reactions (≥20%) were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, abdominal pain, dry skin, arthralgia, dysgeusia, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting.
The most common laboratory abnormalities (≥20%) were increased phosphate, increased creatinine, decreased hemoglobin, increased glucose, increased calcium, decreased sodium, decreased phosphate, increased alanine aminotransferase, increased alkaline phosphatase, decreased lymphocytes, increased aspartate aminotransferase, decreased platelets, increased activated partial thromboplastin time, decreased leukocytes, decreased albumin, decreased neutrophils, increased creatine kinase, increased bilirubin, decreased glucose, increased prothrombin international normalized ratio, and decreased potassium.
DRUG INTERACTIONS
Dual P-gp and Strong CYP3A Inhibitors: Avoid concomitant use of drugs that are dual P-gp and strong CYP3A inhibitors.
Dual P-gp and Strong CYP3A Inducers: Avoid concomitant use of drugs that are dual P-gp and strong CYP3A inducers.
USE IN SPECIFIC POPULATIONS
Lactation: Because of the potential for serious adverse reactions from LYTGOBI in breastfed children, advise women not to breastfeed during treatment and for 1 week after the last dose.