On November 3, 2021 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the third quarter ended September 30, 2021 (Press release, Vanda Pharmaceuticals, NOV 3, 2021, View Source [SID1234594279]).

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"Vanda is approaching another pivotal moment in its 18-year history with the upcoming completion of the Phase III study of tradipitant in gastroparesis and anticipated results by the end of this year. We believe that tradipitant has the potential to become the first new product in 40 years for the treatment of gastroparesis, a serious and debilitating disorder that severely impacts the lives of many people. Additionally, the launch of HETLIOZ and HETLIOZ LQ for nighttime sleep disturbances in people with Smith-Magenis Syndrome is progressing well as we are beginning our broad awareness campaign. Across all of our programs, patient access to innovative treatments remains a key goal of Vanda. We are currently working to address the escalating reimbursement delays for HETLIOZ, which are especially impacting Non-24 patients with light perception, and we are confident in our ability to drive positive outcomes for patients. I am proud of the Vanda team for continuing to advance Vanda’s innovation objectives and creating value for patients and all of the company’s stakeholders," said Mihael H. Polymeropoulos, M.D., Vanda’s President, CEO and Chairman of the Board.

Financial Highlights

Third Quarter of 2021

Total net product sales from HETLIOZ and Fanapt were $70.1 million in the third quarter of 2021, a 16% increase compared to $60.3 million in the third quarter of 2020.
HETLIOZ net product sales were $45.6 million in the third quarter of 2021, a 15% increase compared to $39.6 million in the third quarter of 2020.
Fanapt net product sales were $24.5 million in the third quarter of 2021, an 18% increase compared to $20.7 million in the third quarter of 2020.
Income before taxes was $10.7 million in the third quarter of 2021 compared to $8.4 million in the third quarter of 2020.
First Nine Months of 2021

Total net product sales from HETLIOZ and Fanapt were $200.7 million in the first nine months of 2021, an 11% increase compared to $180.5 million in the first nine months of 2020.
HETLIOZ net product sales were $129.5 million in the first nine months of 2021, an 11% increase compared to $116.5 million in the first nine months of 2020.
Fanapt net product sales were $71.2 million in the first nine months of 2021, an 11% increase compared to $64.0 million in the first nine months of 2020.
Income before taxes was $33.8 million in the first nine months of 2021 compared to $20.7 million in the first nine months of 2020.
Cash, cash equivalents and marketable securities (Cash) was $406.0 million as of September 30, 2021, representing an increase to Cash of $57.4 million compared to September 30, 2020.

Key Operational Highlights

Tradipitant

Enrollment of the randomized portion of the Phase III clinical study of tradipitant in gastroparesis is complete. The randomized portion of the study is a 12-week study of approximately 200 patients with idiopathic or diabetic gastroparesis. Results are expected by the end of 2021. Enrollment of the open label portion of the study is ongoing with more than 250 patients enrolled.
HETLIOZ (tasimelteon)

Reimbursement challenges with HETLIOZ prescriptions for patients with Non-24 have increased significantly. While Vanda is working to address these challenges and is confident in its ability to successfully navigate the current environment, Vanda is revising its HETLIOZ net product sales guidance to $170 to $190 million from the prior HETLIOZ net product sales guidance of $180 to $200 million.
In December 2020, the FDA approved HETLIOZ capsule and liquid formulations for the treatment of adults and children, respectively, with nighttime sleep disturbances in Smith-Magenis Syndrome (SMS).1 To date, more than 90 patients with SMS have been prescribed HETLIOZ or HETLIOZ LQ and a broad awareness campaign is expected to begin during the fourth quarter of 2021.
A Phase III clinical study of HETLIOZ in delayed sleep phase disorder (DSPD) is currently enrolling patients. The study has a 28-day randomized evaluation period and plans to enroll approximately 300 patients. DSPD is likely the most prevalent circadian-rhythm sleep disorder, affecting approximately 1% of the population, and there is no FDA approved treatment at this time.2
Fanapt (iloperidone)

A Phase III clinical study of Fanapt in acute bipolar mania disorder is currently enrolling. The study is a placebo controlled four-week evaluation of approximately 400 patients at sites in the U.S. and Europe.
A repeat-dose clinical pharmacology study of the long acting injectable (LAI) formulation of Fanapt is ongoing. A Phase III study of the LAI formulation for the treatment of acute schizophrenia is planned to follow the pharmacology study.
Evaluation of P88, the active metabolite of iloperidone, has been initiated. P88 has the potential to improve the clinical profile of Fanapt and create sustained, long-term value in the treatment of psychiatric disorders.
GAAP Financial Results

Income before taxes was $10.7 million in the third quarter of 2021 compared to $8.4 million in the third quarter of 2020. Net income was $7.8 million in the third quarter of 2021 compared to net income of $5.9 million in the third quarter of 2020. Diluted net income per share was $0.14 in the third quarter of 2021 compared to diluted net income per share of $0.11 in the third quarter of 2020.

Income before taxes was $33.8 million in the first nine months of 2021 compared to $20.7 million in the first nine months of 2020. Net income was $26.1 million in the first nine months of 2021 compared to net income of $15.1 million in the first nine months of 2020. Diluted net income per share was $0.46 in the first nine months of 2021 compared to diluted net income per share of $0.28 in the first nine months of 2020.

2021 Financial Guidance

Vanda is updating its 2021 financial guidance and expects to achieve the following financial objectives in 2021:

Conference Call

Vanda has scheduled a conference call for today, Wednesday, November 3, 2021, at 4:30 PM ET. During the call, Vanda’s management will discuss the third quarter 2021 financial results and other corporate activities. Investors can call 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 5961858. A replay of the call will be available on Wednesday, November 3, 2021, beginning at 7:30 PM ET and will be accessible until Wednesday, November 10, 2021 at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 5961858.

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.

References

Refer to Company press release titled "FDA Approves HETLIOZ (tasimelteon) for the Treatment of Nighttime Sleep Disturbances in Smith-Magenis Syndrome" issued on December 1, 2020. View Source
P.J. Murphy, Delayed Sleep-Phase Type, Encyclopedia of Sleep, Academic Press, 2013, Pages 22-25, ISBN 9780123786111, View Source

On November 3, 2021 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal ("GI") diseases in areas of high unmet need, reported financial results for the third quarter ended September 30, 2021 (Press release, Synthetic Biologics, NOV 3, 2021, View Source [SID1234594278]).

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Recent Developments:

Initiated a Phase 1, placebo-controlled, multiple ascending dose clinical study of SYN-020 intestinal alkaline phosphatase ("IAP")
Continuing enrollment in the Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant ("HCT") recipients
Current cash position of approximately $72.1 million
Current cash runway expected to provide funding to complete Phase 1b/2a clinical trial of SYN-004, clinical trials of SYN-020 through proof-of-concept, and other key milestones into 2023
Anticipated Milestones:

Topline data readout from the first antibiotic cohort of the SYN-004 Phase 1b/2a clinical trial is expected during Q1 2022
Expect to announce topline data readout from the Company’s Phase 1 MAD clinical trial of SYN-020 during Q2 2022
"During the third quarter, we significantly advanced our portfolio of GI and microbiome-focused clinical programs," said Steven A. Shallcross, Chief Executive Officer of Synthetic Biologics. "We were pleased to initiate a Phase 1 multiple ascending dose clinical trial of our SYN-020 IAP program during the third quarter of 2021. At this time, the first cohort of 8 study participants is nearing completion with dosing of the second cohort of 8 study participants expected to begin shortly thereafter, pending a safety review. We anticipate reporting topline data from this clinical trial during the second quarter of 2022. This study follows our recent Phase 1 single ascending dose ("SAD") clinical trial of SYN-020, in which SYN-020 was well tolerated and demonstrated a favorable safety profile at all doses. Both clinical trials are designed to support the advancement of SYN-020 in multiple potential therapeutic indications, including radiation enteropathy, celiac disease, non-alcoholic fatty liver disease (NAFLD), and age-related metabolic and inflammatory diseases. We continue to view SYN-020 as a multi-indication platform program, which has the potential to help address a considerable need for innovative new therapies targeting disorders that stem from immune and inflammatory damage to the intestine."

Mr. Shallcross continued, "We also continue to progress the SYN-004 Phase 1b/2a clinical trial in allogeneic hematopoietic cell transplant ("HCT") recipients. Patient screening and enrollment is ongoing at the Washington University School of Medicine in St. Louis ("Washington University"). As a result, we currently anticipate announcing topline results from the first of three antibiotic cohorts during the first quarter of 2022. We believe SYN-004 has the potential to address a significant unmet medical need by improving outcomes for allogeneic HCT recipients. Overall, we remain very excited about the potential for each of our clinical programs and near-term clinical milestones that we believe could drive significant value for shareholders."

Clinical Development and Operational Update

Announced commencement of the Company’s Phase 1, placebo-controlled, multiple ascending dose clinical trial of SYN-020
The ongoing Phase 1 MAD clinical trial is intended to evaluate the safety, tolerability and biodistribution of SYN-020 upon repeated dosing in up to 32 healthy adult volunteers.
The study is divided into 4 sequential cohorts of 8 participants, with SYN-020 (5 mg, 15, mg, 45 mg or 75 mg) given orally twice daily for 14 days.
At this time, the first cohort of 8 study participants is nearing completion and dosing of the second cohort of 8 study participants is expected to begin shortly thereafter, pending a safety review.
A safety review will be conducted at the end of each cohort to determine whether progression to the next higher dose cohort is permissible.
A topline data readout from this clinical trial is anticipated during the second quarter of 2022, pandemic conditions permitting.
A previously completed Phase 1 single ascending dose clinical study of SYN-020 enrolled 24 healthy adult volunteers into four cohorts with SYN-020 given orally as a single dose ranging from 5 mg to 150 mg. Analyses of preliminary data demonstrated that SYN-020 maintained a favorable safety profile, was well tolerated at all dose levels, and no adverse events were attributed to study drug. No serious adverse events were reported.
Both studies are intended to support the development of SYN-020 in multiple potential clinical indications including radiation enteropathy, celiac disease, NAFLD, as well as indications supported by the Company’s collaboration with Massachusetts General Hospital.
Enrollment in the Company’s Phase 1b/2a clinical trial of SYN-004 in allogeneic HCT recipients for the prevention of acute graft-versus-host-disease ("aGVHD") remains ongoing
The Phase 1b/2a clinical trial comprises a single center, randomized, double-blind, placebo-controlled clinical trial of oral SYN-004 in up to 36 evaluable adult allogeneic HCT recipients.
The goal of this clinical trial is to evaluate the safety, tolerability, and potential absorption into the systemic circulation (if any) of oral SYN-004 administered to allogeneic HCT recipients who receive an IV beta-lactam antibiotic to treat fever.
Study participants will be enrolled into three sequential cohorts and administered a different study-assigned IV beta-lactam antibiotic. Eight participants in each cohort will receive SYN-004 and 4 will receive placebo.
Safety and pharmacokinetic data for each cohort will be reviewed by an independent Data and Safety Monitoring Committee ("DSMC"), which will make a recommendation on whether to proceed to the next IV beta-lactam antibiotic.
A topline data readout for the first of three antibiotic cohorts is anticipated during the first quarter of 2022, pandemic conditions permitting.
Quarter Ended September 30, 2021 Financial Results

General and administrative expenses increased by 9% to approximately $1.3 million for the three months ended September 30, 2021, from approximately $1.2 million for the three months ended September 30, 2020. This increase is primarily due to higher insurance costs, audit fees and registration fees offset by lower legal costs and vacation expense. The charge related to stock-based compensation expense was $83,000 for the three months ended September 30, 2021, compared to $67,000 for the three months ended September 30, 2020.

Research and development expenses increased by 116% to approximately $2.0 million for the three months ended September 30, 2021, from approximately $900,000 for the three months ended September 30, 2020. This increase is primarily the result of increased clinical trial expenses as we continued dosing patients in the Phase 1b/2a clinical trial of SYN-004 and by higher indirect program costs for the three months ended September 30, 2021, including an increase in manufacturing costs for SYN-020. We anticipate research and development expense to increase as our ongoing clinical trials continue to enroll patients. The charge related to stock-based compensation expense was $19,000 for the three months ended September 30, 2021, compared to $15,000 related to stock-based compensation expense for the three months ended September 30, 2020.

Other income was $2,000 for the three months ended September 30, 2021, compared to other income of $134 for the three months ended September 30, 2020. Other income for the three months ended September 30, 2021 and 2020 is primarily comprised of interest income.

Cash and cash equivalents as of September 30, 2021 totaled $72.1 million, an increase of $65.9 million from December 31, 2020.

Conference Call

Synthetic Biologics will hold a conference call today, Wednesday, November 3, 2021, at 4:30 p.m. (EST). The dial-in information for the call is as follows, U.S. toll free: 1-888-347-5280 or International: +1 412-902-4280. Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source." target="_blank" title="View Source." rel="nofollow">View Source An archive of the call will be available for replay at the same URL, View Source, for 90 days after the call.

On November 3, 2021 ANI Pharmaceuticals, Inc. (NASDAQ: ANIP) ("ANI" or the "Company") reported the pricing of an underwritten public offering of 1,500,000 shares of its common stock at a public offering price of $50.00 per share (Press release, ANI Pharmaceuticals, NOV 3, 2021, View Source [SID1234594277]). The gross proceeds of the offering to the Company are expected to be $75 million, before deducting the underwriting discounts and commissions and other offering expenses. In addition, ANI granted the underwriters a 30-day option to purchase up to an additional 225,000 shares of common stock at the public offering price, less underwriting discounts and commissions.

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The closing of the offering is expected to occur on or about November 8, 2021, subject to the satisfaction of customary closing conditions.

Guggenheim Securities is acting as lead book-running manager for the offering. Raymond James is also acting as book-running manager for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering.

The securities described above are being offered by ANI pursuant to a shelf registration statement on Form S-3 (File No. 333-239771) which was initially filed by the Company with the Securities and Exchange Commission (the "SEC") on July 9, 2020 and was declared effective by the SEC on July 17, 2020.

A preliminary prospectus supplement relating to the offering was filed with the SEC on November 3, 2021 and is available on the SEC’s website at View Source The final prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website. Before investing in the offering, you should read each of the prospectus supplement and the accompanying prospectus relating to the offering in their entirety as well as the other documents that the Company has filed with the SEC that are incorporated by reference in the prospectus supplement and the accompanying prospectus relating to the offering, which provide more information about the Company and the offering. Copies of the final prospectus supplement, when available, and accompanying prospectus relating to the offering may be obtained from Guggenheim Securities, LLC Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017 or by telephone at (212) 518-9544, or by email at [email protected], and from Raymond James & Associates, Inc. Attention: Equity Syndicate, 880 Carillon Parkway, St. Petersburg, Florida 33716, by telephone at (800) 248-8863 or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, 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 any such state or jurisdiction.

On November 3, 2021 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, and The Royal Marsden NHS Foundation Trust, one of the United Kingdom’s (UK) leading cancer research organizations, reported that they have agreed to enter into a partnership to establish an in-house liquid biopsy testing service using Guardant Health’s industry-leading proprietary digital sequencing platform (Press release, Guardant Health, NOV 3, 2021, View Source [SID1234594275]).

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The service is expected to become operational at the end of 2022 and will be available for clinical research and clinical care. This will be the first UK hospital for Guardant Health to house a dedicated liquid biopsy testing facility for cancer diagnostics.

"We are delighted to announce this partnership with Guardant Health, establishing a state-of-the-art liquid biopsy testing facility. In a clinical diagnostic setting, it will provide many of our patients with more rapid access to bespoke diagnostic testing, leading to earlier, faster, and more accurate diagnosis, along with targeted treatment selection and monitoring," said Professor Michael Hubank, Director of Clinical Genomics (Research) at The Royal Marsden NHS Foundation Trust. "The opening of this service will also allow us to significantly increase our capacity for research using liquid biopsies. Bringing expanded capacity for genomic testing, the facility will help us identify more people with cancer for clinical trials based on targeted treatments, improving outcomes for patients across the UK and beyond."

It is estimated that there were around 375,000 new cancer cases in the UK from 2016-20181, with approximately 166,000 cancer deaths in the UK from 2016-2018.1 Guardant Health’s tests are used by oncologists around the world to guide treatment decisions across solid tumor cancers, and by pharmaceutical companies and academic researchers in clinical trials to accelerate precision medicine drug development. Guardant Health has achieved CE Mark and U.S. Food and Drug Administration approval for its Guardant360 CDx assay for tumor mutation profiling, also known as comprehensive genomic profiling (CGP), in patients with any solid cancerous tumor.

"We are pleased to announce this partnership with The Royal Marsden NHS Foundation Trust, which will enable clinicians to quickly and accurately detect relevant tumor alterations with a blood test to guide personalized treatment plans and provide researchers with diagnostic tools to support clinical research and innovation," said Helmy Eltoukhy, Guardant Health Co-CEO. "This partnership further advances our goal of ensuring all patients with cancer have access to the latest innovations to inform their treatment management to enable the best possible outcomes."

On November 3, 2021 ANI Pharmaceuticals, Inc. (NASDAQ: ANIP) ("ANI") reported that it intends to offer and sell shares of its common stock in an underwritten public offering (Press release, ANI Pharmaceuticals, NOV 3, 2021, View Source [SID1234594274]). All of the shares to be sold in the offering will be offered by ANI. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. In addition, ANI intends to grant the underwriters a 30-day option to purchase up to an additional 15% of shares of its common stock offered in the public offering.

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Guggenheim Securities is acting as book-running manager.

ANI anticipates using the net proceeds from the offering to fund its Cortrophin commercialization efforts, including, but not limited to, sales and marketing and consulting expenses related thereto, and for general corporate purposes. We may also use a portion of the net proceeds to in-license, acquire or invest in additional businesses, technologies, products or assets.

The securities described above are being offered by ANI pursuant to a shelf registration statement on Form S-3 (File No. 333-239771) which was initially filed by the Company with the Securities and Exchange Commission (the "SEC") on July 9, 2020, and was declared effective by the SEC on July 17, 2020.

The securities will be offered only by means of a prospectus supplement and accompanying prospectus relating to the offering that form a part of the registration statement. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at View Source Copies of the final prospectus supplement, when available, and accompanying prospectus relating to the offering may be obtained from Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, 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 any such state or jurisdiction.