On August 3, 2022 Jubilant Therapeutics Inc., a biopharmaceutical Company advancing small molecule precision therapeutics to address unmet medical needs in oncology and autoimmune diseases, reported U.S. Food and Drug Administration (US FDA) clearance of the Investigational New Drug application (IND) for JBI-778, an oral, brain penetrant and selective protein arginine methyl transferase 5 (PRMT5) inhibitor, for the treatment of solid tumors with brain metastases and primary brain tumors including high-grade glioma (Press release, Jubilant Radiopharma, AUG 3, 2022, View Source [SID1234617428]).

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Jubilant Therapeutics Inc. announces US FDA clearance of& IND for JBI-778, an Oral, Brain Penetrant and Selective PRMT5 Inhibitor,& for treatment of solid tumors with brain metastases and primary brain tumors
Jubilant Therapeutics Inc. announces US FDA clearance of& IND for JBI-778, an Oral, Brain Penetrant and Selective PRMT5 Inhibitor,& for treatment of solid tumors with brain metastases and primary brain tumors
The Phase I/II trial is an open-label, two-part dose escalation and expansion study designed to define the safety profile, pharmacokinetics, optimal dosing and preliminary activity of JBI-778. The study population in the dose escalation phase will include patients with stable brain metastasis whose disease has failed prior standard therapy. Expansion cohorts will include patients with active brain metastases and high-grade gliomas.

Hari S Bhartia, Chairman, Jubilant Therapeutics Inc. shared on the announcement, "JBI-778 will be our second, highly selective oral drug candidate to enter clinical development following JBI-802. These two programs, along with several others partnered or in preclinical development, highlight Jubilant Therapeutics’ proven discovery engine and structure-based drug discovery expertise."

Syed Kazmi, Chief Executive Officer, Jubilant Therapeutics Inc. said, "JBI-778 was engineered by our drug discovery team to be a PRMT5 substrate-competitive and brain penetrant drug candidate to address primary brain tumors and brain metastases which currently have limited treatment options. The incidence of brain metastasis is increasing due to improved therapies, increased imaging of neurologically asymptomatic patients and patients living longer. Our team has developed a unique capability to optimize brain penetration for precision oncology therapeutics. In addition to JBI-778, we are also advancing an oral brain penetrant PDL1 inhibitor, JBI 2174, which is on the IND-track to potentially treat primary CNS cancers among others."

About JBI-778

JBI-778 is a potent and selective brain penetrant inhibitor of protein arginine methyl transferase 5 (PRMT5), which is overexpressed in many cancers. JBI-778 is in development for the treatment of patients with advanced cancer with brain metastasis, and patients with high-grade glioma all of whom have limited treatment options. It has a unique mechanism of action compared to existing PRMT5 inhibitors by being substrate-competitive and S-adenosylmethoinine (SAM) cooperative, combined with a high brain exposure that enables targeting of both primary brain tumors and CNS metastasis. The substrate competitive profile appears to provide enhanced selectivity in the biological system by not interfering with the functions of SAM and shows a good tolerability profile in toxicological studies.

On August 3, 2022 Aethlon Medical, Inc. (Nasdaq: AEMD), a company developing medical therapeutics to treat cancer and life-threatening infectious disease, reported that it will issue financial results for its first quarter ended June 30, 2022, at 4:15 p.m. EST on Tuesday, August 9, 2022 (Press release, Aethlon Medical, AUG 3, 2022, https://www.prnewswire.com/news-releases/aethlon-medical-to-release-first-quarter-financial-results-and-host-conference-call-on-august-9-2022-301599228.html [SID1234617427]).

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Management will host a conference call on Tuesday, August 9, 2022 at 4:30 p.m. EST 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.

A replay of the call will be available approximately one hour after the end of the call through September 9, 2022. 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 2740523.

On August 3, 2022 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the second quarter ended June 30, 2022 (Press release, Vanda Pharmaceuticals, AUG 3, 2022, View Source [SID1234617426]).

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"During the second quarter we made significant progress towards commercializing our products and improving access to HETLIOZ for patients with Non-24," said Mihael H. Polymeropoulos, M.D., Vanda’s President, CEO and Chairman of the Board. "We advanced our clinical pipeline, with significant clinical milestones expected in the coming quarters, and we are preparing our NDA for tradipitant in gastroparesis and our sNDA for HETLIOZ in insomnia."

Financial Highlights

Second Quarter of 2022

Total net product sales from HETLIOZ and Fanapt were $64.4 million in the second quarter of 2022, a 5% decrease compared to $67.9 million in the second quarter of 2021.

HETLIOZ net product sales were $41.2 million in the second quarter of 2022, a 7% decrease compared to $44.5 million in the second quarter of 2021, due in part to continued reimbursement challenges for prescriptions for patients with Non-24.

Fanapt net product sales were $23.2 million in the second quarter of 2022, a 1% decrease compared to $23.4 million in the second quarter of 2021.

Net income was $2.6 million in the second quarter of 2022 compared to $9.7 million in the second quarter of 2021.
First Six Months of 2022

Total net product sales from HETLIOZ and Fanapt were $124.6 million in the first six months of 2022, a 5% decrease compared to $130.6 million in the first six months of 2021.

HETLIOZ net product sales were $78.2 million in the first six months of 2022, a 7% decrease compared to $83.9 million in the first six months of 2021, due in part to continued reimbursement challenges for prescriptions for patients with Non-24.

Fanapt net product sales were $46.4 million in the first six months of 2022, a 1% decrease compared to $46.7 million in the first six months of 2021.

Net loss was $3.9 million in the first six months of 2022 compared to net income of $18.3 million in the first six months of 2021.

Cash, cash equivalents and marketable securities (Cash) was $440.9 million as of June 30, 2022, representing an increase to Cash of $44.4 million compared to June 30, 2021.
Key Operational Highlights

HETLIOZ (tasimelteon)

Clinical trials for HETLIOZ in delayed sleep phase disorder (DSPD) and sleep disturbances in autism spectrum disorder (ASD) are currently enrolling patients.

Vanda is preparing for the submission of a supplemental New Drug Application (sNDA) for HETLIOZ in the treatment of insomnia.

Since November 2021, more than 15 states have revised or agreed to revise their Medicaid prior authorization criteria to broaden access to HETLIOZ for patients with Non-24 and patients with nighttime sleep disturbances in Smith-Magenis Syndrome (SMS).

In July 2022, an Administrative Law Judge struck down a Medicare Part D plan policy that blocked HETLIOZ coverage for sighted Non-24 patients. Vanda intends to advocate with other Part D plans to challenge similar policies and improve HETLIOZ access for Non-24 patients.

In January 2022, Vanda settled its HETLIOZ patent litigation against one of the Abbreviated New Drug Application (ANDA) defendants. The trial for the consolidated lawsuit against the remaining defendants was held in March 2022. A decision is expected from the court by the end of 2022.
Tradipitant

Vanda is continuing to conduct an open-label safety study for tradipitant in gastroparesis and continues to receive requests from patients seeking access to tradipitant through the Expanded Access program that has multiple patients who have taken tradipitant for more than a year.

Vanda recently held a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) to discuss the planned New Drug Application (NDA) submission for tradipitant in the short-term treatment of nausea in gastroparesis. Vanda is preparing for the submission of the NDA for this indication.

The Phase III study of tradipitant in the treatment of motion sickness is approximately 30% enrolled.
Fanapt (iloperidone)

Enrollment of the Phase III clinical study of Fanapt in acute manic episodes in patients with bipolar disorder is close to being fully enrolled. The study is a placebo controlled four-week evaluation of approximately 400 patients at sites in the U.S. and Europe. Results are expected by the end of 2022.
VQW-765

The Phase II clinical study of a single-dose treatment of VQW-765 to alleviate social/performance anxiety is fully enrolled. Results are expected by the end of 2022.
GAAP Financial Results

Net income was $2.6 million in the second quarter of 2022 compared to net income of $9.7 million in the second quarter of 2021. Diluted net income per share was $0.05 in the second quarter of 2022 compared to diluted net income per share of $0.17 in the second quarter of 2021.

Net loss was $3.9 million in the first six months of 2022 compared to net income of $18.3 million in the first six months of 2021. Diluted net loss per share was $0.07 in the first six months of 2022 compared to diluted net income per share of $0.32 in the first six months of 2021.

2022 Financial Guidance

Conference Call

Vanda has scheduled a conference call for today, Wednesday, August 3, 2022, at 4:30 PM ET. During the call, Vanda’s management will discuss the second quarter 2022 financial results and other corporate activities. Investors can call 1-800-715-9871 (domestic) or 1-646-307-1963 (international) and use passcode number 4304469. A replay of the call will be available on Wednesday, August 3, 2022, beginning at 8:30 PM ET and will be accessible until Wednesday, August 10, 2022 at 8:30 PM ET. The replay call-in number is 1-800-770-2030 for domestic callers and 1-609-800-9909 for international callers. The passcode number is 4304469.

The conference call will be broadcast simultaneously on Vanda’s website, www.vandapharma.com. Investors should click on the Investors tab and are advised to go to the website at least 15 minutes early to register, download, and install any necessary software or presentations. The call will also be archived on Vanda’s website for a period of 30 days.

On August 3, 2022 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported that it has commenced an underwritten public offering of shares of its common stock (Press release, Poseida Therapeutics, AUG 3, 2022, View Source [SID1234617425]). In addition, Poseida expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of its common stock offered in the offering. All of the shares to be sold in the offering are to be sold by Poseida. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.

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Piper Sandler and William Blair & Company, L.L.C. are acting as joint book-running managers for the offering. Cantor Fitzgerald & Co. and BTIG, LLC are also acting as book-running managers for the offering.

The proposed offering is being made pursuant to a shelf registration statement on Form S-3, including a base prospectus, previously filed and declared effective by the Securities and Exchange Commission (the "SEC"). The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A copy of the preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available for free on the website of the SEC at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying prospectus relating to the offering may be obtained, when available, by contacting Piper Sandler, Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924 or by email at [email protected]; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by telephone at (800) 621-0687 or by email at [email protected].

This press release shall 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 other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On August 3, 2022 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that the European Medicines Agency (EMA) issued a positive opinion on the Company’s application for orphan drug designation for tamibarotene for the treatment of myelodysplastic syndrome (MDS) (Press release, Syros Pharmaceuticals, AUG 3, 2022, View Source [SID1234617423]). Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with azacitidine in the SELECT-MDS-1 Phase 3 trial for RARA-positive patients with newly diagnosed higher-risk MDS (HR-MDS).

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Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to tamibarotene in MDS in February 2022.

"We are pleased that the EMA has issued a positive opinion for orphan drug designation for tamibarotene as it represents an important milestone for MDS patients, who have an urgent need for effective, tolerable, and convenient treatment options," said David A. Roth, M.D., Syros’ Chief Medical Officer. "We believe tamibarotene has the potential to change the current standard of care and become the first therapy for a targeted population in HR-MDS. We continue to advance our SELECT-MDS-1 trial and are looking forward to announcing pivotal data in late 2023 or early 2024."

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. The EMA’s orphan designation is available to companies developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. Medicines that meet the EMA’s orphan designation criteria qualify for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase and access to centralized marketing authorization.

The ongoing SELECT-MDS-1 Phase 3 clinical trial is evaluating the safety and efficacy of tamibarotene in combination with azacitidine for RARA-positive patients with newly diagnosed HR-MDS. Data from the pivotal trial are expected in the fourth quarter of 2023 or the first quarter of 2024, with a potential new drug application filing expected in 2024.

Syros is also evaluating tamibarotene in combination with azacitidine and venetoclax for RARA-positive patients with newly diagnosed unfit acute myeloid leukemia (AML), for which tamibarotene had previously received orphan drug designation from both the FDA and EMA. Data from the safety lead-in portion of the SELECT-AML-1 Phase 2 trial is expected in the second half of this year.