On October 28, 2020 Aethlon Medical, Inc. (Nasdaq: AEMD), a medical device technology company focused on developing products to diagnose and treat life and organ threatening diseases, reported financial results for its second quarter ended September 30, 2020 and provided an update on recent developments (Press release, Aethlon Medical, OCT 28, 2020, View Source [SID1234569261]).

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Company Updates

Aethlon Medical, Inc. (Company or Aethlon) is continuing the development of its proprietary Hemopurifier, which is a first in class therapeutic device designed for the single use depletion of cancer-promoting exosomes and circulating viruses. The Hemopurifier has previously been designated a Breakthrough Device by the FDA for the treatment of glycosylated viruses, including Ebola and other hemorrhagic fever viruses, and in late 2018 was additionally designated as a Breakthrough Device "…for the treatment of individuals with advanced or metastatic cancer who are either unresponsive to or intolerant of standard of care therapy, and with cancer types in which exosomes have been shown to participate in the development or severity of the disease….".

Aethlon has initiated its first clinical trial in patients with advanced and metastatic cancers. Under an Investigational Device Exemption (IDE) application approved by FDA in October 2019 this trial, termed an Early Feasibility Study (EFS – the device equivalent of a phase 1 study), in patients with advanced and/or metastatic head and neck cancer is being run at the UPMC Hillman Cancer Center in Pittsburgh, PA and has been approved by the UPMC Institutional Review Board (IRB) and is now open for enrollment. The EFS is designed to enroll 10-12 subjects and will investigate the combination of the Hemopurifier with standard of care pembrolizumab (Keytruda) in the front line setting.

As previously disclosed, the FDA has also approved an amendment to the Company’s open IDE for the Hemopurifier in life threatening viral infections, to allow for the treatment of patients with SARS-CoV-2/COVID-19 infection. This will allow for up to 40 of these patients to be treated under a New Feasibility Study protocol at up to 20 clinical sites in the U.S. The first sites for this trial have received IRB approval and the Company is currently recruiting additional sites. The Company has also recently treated one patient under an emergency use single patient pathway that allows for the use of an investigational product in patients who have essentially failed other treatment options. This patient successfully received eight Hemopurifier treatments of six hours each over nine days.

Financial Results for the Second Quarter Ended September 30, 2020

At September 30, 2020, we had a cash balance of approximately $14.5 million.

Consolidated operating expenses for the three months ended September 30, 2020 were approximately $1.77 million, compared to approximately $1.70 million for the three months ended September 30, 2019. This increase of approximately $70,000, or 4.1%, in the 2020 period was due to a an increase in general and administrative expenses of approximately $212,000, which was partially offset by a decreases in professional fees of approximately $106,000 and in payroll and related expenses of approximately $37,000.

The $212,000 increase in general and administrative expenses was primarily due to a $143,000 increase in lab supplies in connection with our ongoing effort to continue to build an inventory of Hemopurifiers for our clinical trials, and to a $54,000 increase in our clinical trial expenses.

The $106,000 decrease in our professional fees was primarily due to a $94,000 decrease in our legal fees and a $60,000 decrease in our accounting fees, which were partially offset by a $38,000 increase in scientific consulting expenses.

The $37,000 decrease in payroll and related expenses was due to the combination of a $159,000 reduction in stock-based compensation expense and a $122,000 increase in our cash-based compensation expense. The cash-based compensation increase was in turn due to additions to our headcount and to salary increases.

There was no other expense during the three months ended September 30, 2020. In the three months ended September 30, 2019, other expense primarily consisted of approximately $4,000 of losses on share for warrant exchanges.

As a result of the changes in revenues and expenses noted above, our net loss before noncontrolling interests increased to approximately $1.77 million for the three months ended September 30, 2020, from approximately $1.71 million for the three months ended September 30, 2019.

The unaudited condensed consolidated balance sheet for September 30, 2020 and the unaudited condensed consolidated statements of operations for the three and six month periods ended September 30, 2020 and 2019 follow at the end of this release.

Conference Call

The Company will hold a conference call today, Wednesday, October 28, 2020 at 4:30 p.m. Eastern Time to review financial results and recent corporate developments. Following management’s formal remarks, there will be a question and answer session.

Interested parties can register for the conference by navigating to View Source

Please note that registered participants will receive their dial in number upon registration.

Interested parties without internet access or unable to pre-register may dial in by calling:
PARTICIPANT DIAL IN (TOLL FREE): 1-844-836-8741
PARTICIPANT INTERNATIONAL DIAL IN: 1-412-317-5442

All callers should ask for the Aethlon Medical, Inc. conference call.

A replay of the call will be available approximately one hour after the end of the call through November 4, 2020. The replay can be accessed via Aethlon Medical’s website or by dialing 1-877-344-7529 (domestic) or 1-412-317-0088 (international) or Canada Toll Free at 1-855-669-9658. The replay conference ID number is 10149369.

On October 28, 2020 Nektar Therapeutics (Nasdaq: NKTR) reported that it will announce its financial results for the third quarter 2020 on Thursday, November 5, 2020, after the close of U.S.-based financial markets. Howard Robin, President and Chief Executive Officer, will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time (Press release, Nektar Therapeutics, OCT 28, 2020, View Source [SID1234569260]).

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The press release and live audio-only webcast of the conference call can be accessed through a link that is posted on the Home Page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through Tuesday, December 1, 2020.

On October 28, 2020 Asieris Pharmaceuticals, a China-based biotech company specializing in the development and commercialization of new drugs for the treatment of genitourinary tumors and related diseases, reported that Australian regulatory authorities have approved its Phase I clinical trial of APL-1501 (Press release, Asieris Pharmaceuticals, OCT 28, 2020, View Source [SID1234569258]). The objective of this Phase I study is to evaluate the safety, tolerability, and pharmacokinetic (PK) characteristics of APL-1501.

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APL-1501, which is independently developed by the Prodrug Accurate Drug Delivery (PADD) platform of Asieris, is an oral sustained-release product based on APL-1202 and will be the second generation product of APL-1202 to support new clinical development globally. APL-1501 inherits the unique oral administration of APL-1202 and the druggability of APL-1501 is improved by the design of prodrug molecules. Based on these, APL-1501 can improve patient compliance by reducing medication frequency, combined with the sustained-release technology which can contribute to controlled drug release and lengthen the drug plasma exposure time. APL-1501 has better pharmacokinetic (PK) characteristics so that this product is expected to not only treat bladder cancer, but also for other diseases such as prostate cancer, urinary tract infection, etc. Since APL-1501 is a product developed by Asieris independently, a comprehensive global intellectual property rights strategy has been developed to provide strong support for product life cycle managment and global expansion.

APL-1202 is being developed as a new drug of Asieris for the treatment of non-muscle invasive bladder cancer (NMIBC). It is the first oral and reversible methionine aminopeptidase II type (MetAP2) inhibitor currently under Phase III clinical development in the world. APL-1202 finished patient enrollment in pivotal registration trial in China in 2019. APL-1202 also completed the Phase I clinical trial in the US. Currently, the standard treatment of NMIBC is a Trans-Urethral Resection of Bladder Tumor (TURBT). Because of a high tumor recurrence rate after TURBT, intravesical chemo- or immune-therapies are required after the procedure. At present, choice of second-line treatment for relapsed patients is very limited. A radical cystectomy is the standard treatment for recurring high-risk NMIBC patients. No oral drugs have been approved for NMIBC to date.

"The approval of APL-1501 clinical trial in Australia is another important milestone on the journey of Asieris’ international clinical development", commented Dr. Kevin Pan, founder and CEO of Asieris. "As the second generation product of APL-1202, APL-1501 will bring better treatment choice to patients and reduce the pain and adverse reactions. Asieris will continue to adhere to the vision of ‘We strive for healthy and dignified lives’. We hope the innovative products will benefit more patients globally soon."

About APL-1501 and APL-1202

APL-1501 is developed by Asieris from its PADD technology platform as the second generation product of APL-1202, which has better pharmacokinetic characteristics. Besides the treatment of NMIBC, the product is expected to enter more therapeutic fields for clinical development, such as prostate cancer, urinary tract infection, etc.

APL-1202 is being developed as a new drug of Asieris for the treatment of non-muscle invasive bladder cancer (NMIBC) and is the first oral and reversible methionine aminopeptidase II type (MetAP2) inhibitor currently under Phase III clinical development in the world. In preclinical studies, APL-1202 has demonstrated both anti-angiogenic and anti-tumor activities as well as a potential synergistic effect with anti-BCG or epirubicin. APL-1202 is currently under clinical investigation for multiple indications. The treatment regimen with APL-1202 is convenient and well-tolerated, without causing pain or injury to the urethra, and may help some high-risk patients avoid radical cystectomy.

On October 28, 2020 Medidata, a Dassault Systèmes Company, reported that the US Food and Drug Administration (FDA) supported the use of a Medidata Synthetic Control Arm in a phase 3 registrational trial in recurrent glioblastoma (rGBM) (Press release, Medidata, OCT 28, 2020, View Source [SID1234569257]). This is a precedent setting acceptance of a hybrid external control (combining synthetic control arm patients with randomized patients) in a phase 3 trial in an indication that previously used traditional randomized controls.

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Medicenna Therapeutics, Corp. (NASDAQ: MDNA, TSX: MDNA), a clinical stage immunotherapy company, will use a hybrid external control arm in a phase 3 registrational trial in recurrent glioblastoma, an aggressive form of brain cancer. The hybrid external control arm will reduce the number of patients required to be assigned to the control therapy in the trial, will provide rigorous scientific data, and will enable speedier development of the product. The phase 2 single arm trial preceding this phase 3 study was also enhanced by a synthetic control arm and estimates of the treatment effects based on the synthetic control arm were part of the briefing information provided to the FDA for justification of the use in phase 3.

"We are pleased with FDA’s acceptance and look forward to creating a highly rigorous scientific comparison to support development of Medicenna’s MDNA55 for recurrent glioblastoma," said Ruthie Davi, vice president, Data Science, Acorn AI by Medidata. "There are no established therapies to prolong life for people suffering with rGBM, so the hybrid external control arm could provide great hope for patients with this disease."

"We are extremely impressed with the Acorn AI team for providing a scientifically rigorous rationale for the design of an innovative registration trial incorporating an external control arm for the treatment of recurrent glioblastoma (rGBM) with MDNA55. Their expertise and collaborative effort with thought leaders was instrumental in demonstrating to the FDA the validity of a well-designed external control in a registration trial," said Fahar Merchant, PhD, president and CEO, Medicenna Therapeutics, Corp. "The FDA’s acceptance of this unique design will expedite completion of the Phase 3 trial in rGBM, allowing earlier access of MDNA55 for a disease with poor prognosis and high unmet need."

Synthetic control arms are formed by carefully selecting patients from historical clinical trials to match the demographic and disease characteristics of the patients treated with the new investigational product. They have great potential to enhance single arm trials and enable more efficient randomized clinical trials in indications where the standard of care therapy is impracticable or unacceptable to patients.

Medidata is a wholly owned subsidiary of Dassault Systèmes, which with its 3DEXPERIENCE platform is positioned to lead the digital transformation of life sciences in the age of personalized medicine with the first end-to-end scientific and business platform, from research to commercialization.

On October 28, 2020 Scholar Rock Holding Corporation (Nasdaq: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported the pricing of an underwritten public offering of 2,948,718 shares of its common stock at a public offering price of $39.00 per share (Press release, Scholar Rock, OCT 28, 2020, View Source [SID1234569256]). In addition, in lieu of common stock to certain investors, Scholar Rock is offering pre-funded warrants to purchase 2,179,487 shares of its common stock at a purchase price of $38.9999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.0001 exercise price per share of each pre-funded warrant. The aggregate gross proceeds to Scholar Rock from this offering are expected to be approximately $200 million, before deducting underwriting discounts and commissions and other estimated offering expenses. In addition, Scholar Rock has granted the underwriters a 30-day option to purchase up to an additional 769,230 shares of common stock at the public offering price per share of the common stock, less the underwriting discounts and commissions. The offering is expected to close on November 2, 2020, subject to the satisfaction of customary closing conditions. All of the shares and pre-funded warrants are being offered by Scholar Rock.

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Scholar Rock intends to use the net proceeds from the offering to advance SRK-015 in Spinal Muscular Atrophy, including costs associated with preparing for and executing clinical trials (including a Phase 3 clinical trial), SRK-181 in cancer immunotherapy, development of its preclinical and discovery programs, as well as for working capital and other general corporate purposes.

J.P. Morgan Securities LLC, Jefferies LLC and Credit Suisse Securities (USA) LLC are acting as joint book-running managers for the offering. BMO Capital Markets Corp. is acting as lead manager for the offering.

The securities described above are being offered by Scholar Rock pursuant to a shelf registration statement on Form S-3 (No. 333-231920) that was declared effective by the Securities and Exchange Commission (SEC) on June 10, 2019. A preliminary prospectus supplement and accompanying prospectus and a free writing prospectus relating to and describing the terms of the offering were filed with the SEC on October 27, 2020 and October 28, 2020, respectively, and are available on the SEC’s website located at www.sec.gov. A copy of the final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and may be obtained, when available, by contacting: J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at 1-866-803-9204 or by email at [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at 877-547-6340 or by email at [email protected]; or Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037 or by email at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that state or jurisdiction.