On August 12, 2020 KAHR Medical, a cancer immunotherapy company developing novel bi-functional fusion proteins, reported that the U.S. Food and Drug Administration has cleared its investigational new drug (IND) application for the Company’s lead product, DSP107, a second generation CD47x41BB targeting compound that simultaneously target cancer cells, weaken their innate defenses and activate an effective, local response of both innate and adaptive immunity (Press release, KAHR Medical, AUG 12, 2020, View Source [SID1234563454]).

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Under this IND, the Company intends to initiate a Phase I/II clinical trial to evaluate the safety, pharmacokinetics (PK) and pharmacodynamics (PD) of DSP107 as a monotherapy and in combination with Roche’s PD-L1-blocking checkpoint inhibitor (CPI) atezolizumab (Tecentriq) in patients with advanced solid tumors. The study will be conducted at multiple centers in the United States and site activation activities are currently underway.

"Receiving clearance from the FDA to advance our lead immuno-oncology program to the clinic marks a significant milestone for KAHR as we transform into a clinical-stage company," said Yaron Pereg, PhD, CEO, KAHR Medical. "DSP107, with its unique dual mechanism of action and its excellent safety profile with no hematological toxicities has the potential to become a best-in-class CD47 therapy. We are proud of our significant progress in recent years and look forward to initiating the Phase I/II study in the upcoming weeks for the benefit of patients suffering from challenging to treat cancers," added Dr. Pereg.

The planned Phase I/II study will evaluate the safety, pharmacokinetics (PK) and pharmacodynamics (PD) of DSP107 in advanced solid tumors. The preliminary efficacy of both DSP107 monotherapy and combination therapy with atezolizumab will also be evaluated in patients with advanced non-small-cell lung carcinoma (NSCLC) who progressed after treatment with PD-1/PD-L1 inhibitors. The study will be conducted under a clinical collaboration with Roche.

About DSP107

DSP107 targets CD47-overexpressing tumors, simultaneously blocking macrophage inhibitory signals and delivering an immune costimulatory signal to tumor antigen-specific, activated T-cells. CD47 is overexpressed on many cancer cells and binds SIRPα on immune phagocytic cells to produce a "don’t eat me" signal. DSP107 binds CD47 on cancer cells, blocking interaction with SIRPα and thus, blocking the "don’t eat me signal". Simultaneously, DSP107 binds 41BB on T-cells, stimulating their activation. These activities lead to targeted immune activation through both macrophage and T-cell mediated tumor destruction.

On August 12, 2020 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, metabolic, autoimmune and other major diseases, reported with Eli Lilly and Company ("Lilly",NYSE: LLY) that the National Medical Products Administration (NMPA) of China has accepted the supplemental New Drug Application (sNDA) for TYVYT (sintilimab injection) in combination with Gemzar (gemcitabine) and platinum as first-line therapy in squamous non-small cell lung cancer (squamous NSCLC) (Press release, Innovent Biologics, AUG 12, 2020, View Source [SID1234563453]). Recently, the NMPA accepted sNDA for TYVYT (sintilimab injection) as first-line therapy in non-squamous NSCLC on Apr 23, 2020.

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The sNDA was based on the analysis of a randomized, double-blind, Phase 3 clinical study (ORIENT-12)—TYVYT (sintilimab injection) or placebo in combination with Gemzar (gemcitabine) and platinum as first-line therapy for advanced or metastatic squamous NSCLC. Based on the analysis conducted by the Independent Data Monitoring Committee (IDMC), TYVYT (sintilimab injection) in combination with Gemzar (gemcitabine) and platinum demonstrated a statistically significant improvement in progression-free survival (PFS) compared with placebo in combination with Gemzar (gemcitabine) and platinum, which met the pre-defined efficacy criteria. The safety profile is consistent with previously reported sintilimab studies, and no new safety signals were identified. Detailed data will be released in an upcoming international academic conference and journal.

Professor Caicun Zhou, Head of Department of Oncology, Shanghai Pulmonary Hospital, stated: "We are pleased to see that sintilimab in combination with chemotherapy has met predefined primary endpoint in ORIENT-12 study. There still exists large unmet medical needs in squamous NSCLC patients. Globally, ORIENT-12 has demonstrated for the first time survival benefit by treatment with PD-1 inhibitor in combination with gemcitabine and platinum in first-line squamous NSCLC. The result of this study has great clinical value."

Dr. Hui ZHOU, Vice President of Medical Science and Strategy Oncology of Innovent, stated: "For Innovent, this is the second double-blind, randomized Phase 3 study of NSCLC that met primary endpoint following ORIENT-11, a randomized Phase 3 study of 1L non-squamous NSCLC (NCT03607539). This is also the second sNDA of TYVYT (sintilimab injection) for first-line NSCLC indication. The acceptance of the sNDA by NMPA represents further progress in covering first-line NSCLC disease area. We will actively cooperate with the regulatory authority to bring the high quality therapy to more patients with advanced squamous NSCLC as soon as possible."

"Lilly and Innovent are committed to bring new anti-tumor treatments, developed with global standards, to patients in China. TYVYT (sintilimab injection) is the first success of this partnership. After being included in the NRDL for relapsed or refractory classical Hodgkin’s lymphoma, the acceptance of the sNDA for new indication of NSCLC is another important milestone of TYVYT (sintilimab injection) in the field of oncology therapy," said Dr. Li WANG, Senior Vice-President of Lilly China and Head of Lilly China Drug Development and Medical Affairs. "ORIENT-12 provides new combination option of chemotherapy for squamous NSCLC patients and we look forward to bringing this new treatment solution to lung cancer patients in China as soon as possible. In the future, we will continue to strengthen cooperation with Innovent to further explore TYVYT (sintilimab injection)’s potential in the field of immuno-oncology therapy and the potential benefits TYVYT (sintilimab injection) may bring to more patients."

About squamous NSCLC

Lung cancer is a malignancy with the highest morbidity and mortality in China. NSCLC accounts for approximately 80-85% of all lung cancer diagnosis. About 70% of NSCLC are locally advanced or metastatic at initial diagnosis, rendering the patients with no chance of radical resection. Meanwhile, even after radical surgery the patients still have a high chance of recurrence and eventually die from disease progression. About 35% of patients with NSCLC in China are of squamous subtype without driver genes. There are limited treatment options available to them.

About ORIENT-12 Study

ORIENT-12 is a randomized, double-blind, Phase 3 clinical study to evaluate the efficacy and safety of TYVYT (sintilimab injection) or placebo in combination with Gemzar (gemcitabine) and platinum as first-line therapy for advanced or metastatic squamous NSCLC (ClinicalTrials.gov, NCT03629925). The primary endpoint is progression-free survival (PFS) assessed by Independent Radiographic Review Committee based on RECIST v1.1. The key secondary endpoints include overall survival (OS) and safety profiles.

A total of 357 subjects have been enrolled in ORIENT-12 study and randomized at a 1:1 ratio to receive either sintilimab 200mg or placebo in combination with Gemzar (gemcitabine) and platinum every 3 weeks for up to 4 or 6 cycles, followed by either sintilimab or placebo maintenance therapy. The subjects will receive treatment until radiographic disease progression, unacceptable toxicity or any other conditions that require treatment discontinuation. Conditional crossover is permitted.

About ORIENT-11 Study

ORIENT-11 is a randomized, double-blind, Phase 3 clinical study to evaluate the efficacy and safety of TYVYT (sintilimab injection) or placebo in combination with ALIMTA (pemetrexed) and platinum as first-line therapy for advanced or recurrent non-squamous NSCLC without sensitizing EGFR mutation or ALK rearrangement (ClinicalTrials.gov, NCT03607539). The primary endpoint is progression-free survival (PFS) assessed by Independent Radiographic Review Committee based on RECIST v1.1. The key secondary endpoints include overall survival (OS) and safety profile.

A total of 397 subjects have been enrolled in ORIENT-11 study and randomized in a 2:1 ratio to receive either sintilimab 200mg or placebo in combination with ALIMTA (pemetrexed) and platinum every 3 weeks for up to 4 cycles, followed by either sintilimab or placebo plus ALIMTA maintenance therapy. The subjects will receive treatment until radiographic disease progression, unacceptable toxicity or any other conditions that require treatment discontinuation. Conditional crossover is permitted.

This study has met primary endpoint and NMPA accepted the sNDA for TYVYT (sintilimab injection) as first-line therapy in non-squamous NSCLC on Apr 23, 2020.

About TYVYT (Sintilimab Injection)

TYVYT (sintilimab injection), an innovative drug developed with global quality standards jointly developed in China by Lilly and Innovent, has been granted marketing approval by the NMPA for relapsed or refractory classic Hodgkin’s lymphoma after at least two lines of systemic chemotherapy, and included in the 2019 Guidelines of Chinese Society of Clinical Oncology for Lymphoid Malignancies. TYVYT (sintilimab injection) is the only PD-1 inhibitor that has been included in the new Catalogue of the National Reimbursement Drug List (NRDL) in November 2019.

In April 2020, the NMPA accepted the sNDA for TYVYT (sintilimab injection) in combination with ALIMTA (pemetrexed) and platinum as first-line therapy in non-squamous non-small cell lung cancer (NSCLC). In May 2020, TYVYT (sintilimab injection) combined with Gemzar (gemcitabine for injection) and platinum chemotherapy met the predefined primary endpoint in the Phase 3 ORIENT-12 study as first-line therapy in patients with locally advanced or metastatic squamous NSCLC, and TYVYT (sintilimab injection) monotherapy met the primary endpoint in the ORIENT-2 study as second-line therapy in patients with advanced or metastatic esophageal squamous cell carcinoma as well. In August, the NMPA accepted the sNDA for TYVYT (sintilimab injection) in combination with Gemzar (gemcitabine for injection) and platinum chemotherapy as first-line therapy in patients with locally advanced or metastatic squamous NSCLC.

TYVYT (sintilimab injection) is a type of immunoglobulin G4 monoclonal antibody, which binds to PD-1 molecules on the surface of T-cells, blocks the PD-1/ PD-Ligand 1 (PD-L1) pathway and reactivates T-cells to kill cancer cells. Innovent is currently conducting more than 20 clinical studies for sintilimab injection to evaluate its safety and efficacy in a wide variety of cancer indications, including more than 10 registration or pivotal clinical studies. Meanwhile, we are actively developing TYVYT globally.

On August 12, 2020 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for population sequencing and cancer, reported the pricing of its previously announced underwritten public offering of 6,578,947 shares of its common stock at a price to the public of $19.00 per share (Press release, Personalis, AUG 12, 2020, View Source [SID1234563452]). Gross proceeds to Personalis from the offering are expected to be $125 million, before deducting underwriting discounts and commissions and estimated offering expenses.

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In addition, the underwriters have been granted a 30-day option to purchase up to an additional 986,842 shares of common stock from a selling stockholder on the same terms and conditions. Personalis will not receive any proceeds from any sale of shares by the selling stockholder.

BofA Securities, Citigroup and Cowen are acting as joint book-running managers. BTIG, Needham & Company and Oppenheimer & Co. are acting as co-managers.

A shelf registration statement relating to the shares being sold in this offering was filed with the U.S. Securities and Exchange Commission on July 2, 2020, and was declared effective on July 10, 2020. A preliminary prospectus supplement and accompanying prospectus relating to the offering was filed with the SEC and are available for free on the SEC’s website located at View Source When available, electronic copies of the final prospectus supplement and accompanying prospectus relating to the offering may be obtained from BofA Securities, Inc., Attention: Prospectus Department, NC1‐004‐03‐43, 200 North College Street, 3rd floor, Charlotte, NC 28255‐0001, or by emailing [email protected]; Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at 1-800-831-9146; or Cowen and Company, LLC, c/o Broadridge Financial Solutions, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, New York 11717, telephone: 631‐274‐2806.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, 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 August 12, 2020 Atomwise Inc., which uses artificial intelligence to help academic and commercial scientists discover new medicines, reported that it has raised $123 million to expand and build its own pipeline of experimental drugs (Press release, Atomwise, AUG 12, 2020, View Source [SID1234563442]).

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Based in San Francisco, Atomwise is one of several startups seeking to use AI to speed drug development and uncover medicines that would be difficult to discover through other means. Others include Deep Genomics Inc., which in January said it had raised $40 million to develop treatments for conditions such as the rare disorder.

On August 12, 2020 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the second quarter ended June 30, 2020 (Press release, Vanda Pharmaceuticals, AUG 12, 2020, View Source [SID1234563217]).

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"We are very proud of the achievements of our team, especially the record commercial performance, even in the face of the challenges presented by the pandemic. We are also very excited with the progress of the HETLIOZ SMS applications as we get closer to providing a therapeutic solution to patients with SMS," said Mihael H. Polymeropoulos, M.D., Vanda’s President and CEO.

Key Financial and Corporate Highlights

Total revenues from HETLIOZ and Fanapt were $62.2 million in the second quarter of 2020, a 5% increase compared to $59.1 million in the second quarter of 2019.
HETLIOZ net product sales were $41.6 million in the second quarter of 2020, a 10% increase compared to $37.8 million in the second quarter of 2019.
Fanapt net product sales were $20.6 million in the second quarter of 2020, a 3% decrease compared to $21.2 million in the second quarter of 2019.
Cash, cash equivalents and marketable securities (Cash) were $339.8 million as of June 30, 2020, representing an increase to Cash of $47.2 million compared to June 30, 2019.
Net income was $8.7 million in the second quarter of 2020 compared to net income of $11.5 million in the second quarter of 2019.
Key Product and Pipeline Highlights

Products

Vanda is encouraged by its record commercial performance during the second quarter of 2020. Vanda continues to implement marketing and sales strategies aimed at supporting continued growth and minimizing the impact of disruptions caused by the COVID-19 pandemic, including the launch of a Fanapt for schizophrenia direct-to-consumer campaign at the end of the second quarter of 2020.

Pipeline

The COVID-19 pandemic has impacted clinical research globally, including Vanda’s previously reported clinical trials. The tradipitant gastroparesis and motion sickness programs have resumed, while recruitment for the tradipitant atopic dermatitis program, as well as the HETLIOZ delayed sleep phase disorder study and Fanapt bipolar disorder and long acting injectable studies, is currently on hold.

Tradipitant

An Individual Patient Expanded Access protocol (VP-VLY-686-3303) for tradipitant in gastroparesis was approved by the U.S. Food and Drug Administration (FDA) and the patient was enrolled in July 2020. Under this protocol, this patient will receive tradipitant treatment for a period of up to six months, which may be extended upon review by the FDA.
The gastroparesis Phase III clinical study (VP-VLY-686-3301) resumed recruitment. Enrollment in this 200-person study is expected to be completed in the first half of 2021 with a New Drug Application (NDA) filing projected for later that year.
The protocol for the pivotal Phase III motion sickness study was discussed with the FDA at the end of Phase II meeting, and the FDA agrees with the adequacy of the program design to support an application. Preparations for this study have begun with the boat trip portion of the study expected to commence as soon as local restrictions are lifted.
Patient enrollment in the Phase III clinical study (ODYSSEY VLY-686-3501) of tradipitant in COVID-19 Acute Respiratory Distress Syndrome (ARDS) is ongoing and an interim analysis will be conducted to determine next steps.
HETLIOZ (tasimelteon)

The Smith-Magenis Syndrome (SMS) marketing authorization applications were accepted by the FDA for priority review with a Prescription Drug User Fee Act (PDUFA-VI) target action date of December 1, 2020.1
The FDA appeals process related to the sNDA for HETLIOZ for the treatment of jet lag disorder is ongoing.
Key Publications

The article "Efficacy and Safety of Tradipitant in Patients with Diabetic and Idiopathic Gastroparesis in a Randomized, Placebo-Controlled Trial" was accepted for publication in the July 2020 issue of Gastroenterology.2
The article "Efficacy of Tasimelteon (HETLIOZ) in the Treatment of Jet Lag Disorder Evaluated in an 8-h Phase Advance Model; a Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial" was published in the July 2020 issue of Frontiers in Neurology.3
GAAP Financial Results

Net income was $8.7 million in the second quarter of 2020, compared to net income of $11.5 million in the second quarter of 2019. Diluted net income per share was $0.16 in the second quarter of 2020, compared to diluted net income per share of $0.21 in the second quarter of 2019.

2020 Financial Guidance

Vanda will continue to assess the impact of the evolving pandemic on its business and operations and will provide future updates to its financial guidance as necessary. The financial guidance previously communicated by Vanda is shown below. In addition, Vanda provides an update to Year-end 2020 Cash.

Full Year 2020

Financial Objectives

Full Year 2020

Guidance

Total revenues

$240 to $260 million

HETLIOZ net product sales

$155 to $165 million

Fanapt net product sales

$85 to $95 million

Year-end 2020 Cash

Greater than $340 million as compared to prior
guidance of greater than $320 million

Conference Call

Vanda has scheduled a conference call for today, Wednesday, August 5, 2020, at 4:30 PM ET. During the call, Vanda’s management will discuss the second quarter 2020 financial results and other corporate activities. Investors can call 1-888-771-4371 (domestic) or 1-847-585-4405 (international) and use passcode 49854840.

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:

1 Refer to Company press release titled "FDA Accepts and Grants Priority Review of Vanda’s Applications for HETLIOZ (tasimelteon) in the Treatment of Smith-Magenis Syndrome" issued on August 3, 2020. View Source

2 Carlin, J. L., Lieberman, V. R., Dahal, A., Keefe, M. S., Xiao, C., Birznieks, G., Abell, T. L., Lembo, A., Parkman, H., & Polymeropoulos, M. H. (2020). Efficacy and safety of tradipitant in patients with diabetic and idiopathic gastroparesis in a randomized, placebo-controlled trial. Gastroenterology. Advance online publication. View Source

3 Polymeropoulos, C. M., Mohrman, M. A., Keefe, M. S., Brzezynski, J. L., Wang, J., Prokosch, L. S., Polymeropoulos, V. M., Xiao, C., Birznieks, G., & Polymeropoulos, M. H. (2020). Efficacy of tasimelteon (Hetlioz) in the treatment of jet lag disorder evaluated in an 8-h phase advance model; a multicenter, randomized, double-blind, placebo-controlled trial. Frontiers in Neurology, 11, 611. View Source