On August 6, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that on August 5, 2020, the Company completed the submission of its Biologics License Application ("BLA") under the FDA’s Rolling Review process for omburtamab (Press release, Y-mAbs Therapeutics, AUG 6, 2020, View Source [SID1234563033]). Omburtamab is an investigational, monoclonal antibody that targets B7-H3, an immune checkpoint molecule that is widely expressed in tumor cells of several cancer types. The omburtamab BLA is for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma. The submission is based on the safety and efficacy results of the pivotal Phase 2 studies 101 and 03-133, which the Company expects to present at a venue later this year.

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"I am excited to see the completion of Y-mAbs’ second BLA submission this year in neuroblastoma. As children treated for high-risk systemic neuroblastoma potentially experience longer systemic remissions, we expect more patients eventually relapsing with brain metastasis and there is currently no standard therapy available for these patients. We believe this is a key milestone for families facing CNS/leptomeningeal metastasis from neuroblastoma and for Y-mAbs. As the father of a long-term high-risk neuroblastoma survivor with CNS/Leptomeningeal metastasis, I know how important this potentially is for families faced with brain metastasis from high-risk neuroblastoma." stated Thomas Gad, Founder, Chairman and President.

Dr. Claus Moller, Chief Executive Officer, continued, "We believe omburtamab can potentially address a significant unmet medical need for children with CNS/leptomeningeal metastasis from neuroblastoma, and we look forward to working with the FDA to bring omburtamab to appropriate patients. Omburtamab is also being tested in a Phase 2 study for desmoplastic small round cell tumor and we are currently planning a Phase 2 study for diffuse intrinsic pontine glioma, as we believe omburtamab could potentially be developed for wider compartmental use."

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests related to the compound and Y-mAbs.

On August 6, 2020 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that it has entered into a clinical trial collaboration with AstraZeneca (LSE/STO/NYSE: AZN) to evaluate the combination of patritumab deruxtecan (U3-1402), a HER3 directed DXd antibody drug conjugate (ADC), and TAGRISSO (osimertinib), an epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), in patients with EGFR-mutated advanced or metastatic non-small cell lung cancer (NSCLC) (Press release, Daiichi Sankyo, AUG 6, 2020, View Source [SID1234563016]).

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There are no HER3 directed therapies approved for the treatment of NSCLC or any cancer. The frequency of HER3 overexpression in EGFR-mutated NSCLC has been reported to be as high as 75 percent, and there is evidence that HER3 expression may be associated with resistance to TKIs.[1],[2]

"The majority of patients with activating mutations of EGFR, or EGFR-mutated NSCLC, overexpress the HER3 protein in the cancer cells, and there is evidence that HER3 expression is a passenger marker of resistance to TKIs. Clinical and preclinical data, as well as biomarker expression and resistance mechanism research, support the further evaluation of patritumab deruxtecan and TAGRISSO as a treatment combination for patients with EGFR-mutated NSCLC who have progressed after treatment with a TKI, typically TAGRISSO," said Gilles Gallant, BPharm, PhD, FOPQ, Senior Vice President, Global Head, Oncology Development, Oncology R&D, Daiichi Sankyo. "This clinical trial collaboration supports our goal to optimize development of patritumab deruxtecan in patients with EGFR-mutated metastatic NSCLC to further improve current standards of care. Daiichi Sankyo is pleased to begin this focused collaboration with AstraZeneca on this important aspect of patritumab deruxtecan development."

"In this trial, we will explore a new potential way to treat patients with advanced disease by combining TAGRISSO with patritumab deruxtecan, a HER3 directed ADC," said Cristian Massacesi, Senior Vice President, Head of Late Stage Development Oncology R&D, AstraZeneca. "This combination approach represents one of our strategies of addressing tumor resistance. As we work to continue maximizing the benefit TAGRISSO can bring to patients, we look forward to collaborating with Daiichi Sankyo in this new area of study."

About the Study

Under the terms of the agreement, Daiichi Sankyo will sponsor and conduct a multicenter, open-label, two-part phase 1 study evaluating patritumab deruxtecan and TAGRISSO as both a first-line and second-line combination treatment in patients with advanced or metastatic NSCLC with an EGFR exon 19 deletion or L858R mutation.

The first part of the study (dose escalation) will assess the safety and tolerability of different dosing combinations of patritumab deruxtecan and TAGRISSO to determine the recommended combination dose. The second part of the study (dose expansion) will include a first-line and second-line cohort that will further evaluate the anti-tumor activity and safety of the combination. Patients enrolled in the first-line cohort will receive patritumab deruxtecan and TAGRISSO combination treatment, and patients in the second-line cohort will be randomized 1:1 to receive treatment with patritumab deruxtecan alone or in combination with TAGRISSO. Up to 258 patients will be enrolled into the study, which will be conducted in North America, Europe, and Asia including Japan.

The primary study objectives for the dose escalation part of the study include the assessment of the safety and tolerability of patritumab deruxtecan and TAGRISSO. The primary objective for the dose expansion part of the study for both cohorts is the assessment of anti-tumor activity as measured by objective response rate (ORR) and as assessed by independent central review.

Unmet Need in Non-Small Cell Lung Cancer (NSCLC)

Lung cancer is the most common cancer and is the leading cause of cancer mortality worldwide with an estimated 2.1 million new cases of lung cancer in 2018 and 1.8 million deaths.[3] Non-small cell lung cancer (NSCLC) accounts for 80 to 85 percent of all lung cancers.[4]

Mutations in the epidermal growth factor receptor (EGFR) gene are among the most frequently observed genomic alterations in NSCLC, affecting approximately 14 to 30 percent of patients with NSCLC.[5] For patients with EGFR-mutated NSCLC, targeted therapy with EGFR TKIs offers higher response rates, overall survival and progression-free survival compared to chemotherapy.[6] However, most patients eventually develop resistance to the TKIs, and new treatment approaches including combination strategies designed to overcome TKI resistance are needed.[7]

About HER3

HER3 is a member of the EGFR family of tyrosine kinase receptors, which are associated with normal as well as aberrant cell proliferation and survival.2 HER3 expression has been associated with an increased incidence of metastases and reduced survival in patients with NSCLC with expression frequency reported to be as high as 75 percent.1 A majority of EGFR-mutated NSCLCs show some level of HER3 expression.[8],[9] Currently, no HER3 directed therapies are approved for NSCLC or any cancer.

About Patritumab Deruxtecan (U3-1402)

Patritumab deruxtecan (U3-1402) is one of three lead DXd antibody drug conjugates (ADC) in the oncology pipeline of Daiichi Sankyo.

ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy ("payload") to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, patritumab deruxtecan is comprised of a human anti-HER3 antibody attached to a topoisomerase I inhibitor payload by a tetrapeptide-based linker. It is designed to target and deliver chemotherapy inside cancer cells that express HER3 as a cell surface antigen.

Patritumab deruxtecan is currently being evaluated in a phase 1 study in previously treated patients with metastatic or unresectable NSCLC. Patritumab deruxtecan is also being evaluated in a phase 1/2 study in patients with HER3 expressing metastatic breast cancer.

Patritumab deruxtecan is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

On August 6, 2020 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company is scheduled to present at the virtual Canaccord 40th Annual Growth Conference on Thursday, August 13, 2020 at 1:00 p.m. ET (Press release, Agios Pharmaceuticals, AUG 6, 2020, View Source [SID1234562986]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On August 6, 2020 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biotechnology company, reported the pricing of the previously announced underwritten registered public offering of 4,000,000 of its common shares at a price to the public of $5.00 per share (Press release, DiaMedica, AUG 6, 2020, View Source [SID1234562985]). DiaMedica has also granted the underwriters a 30-day option to purchase up to an additional 600,000 common shares, at the public offering price, less underwriting discounts and commissions. All of the common shares are being offered by DiaMedica. The offering is expected to close on or about August 10, 2020, subject to the satisfaction of customary closing conditions.

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DiaMedica expects the gross proceeds from the offering to be approximately $20 million, before deducting the underwriting discount and other estimated offering expenses payable by DiaMedica. Net proceeds, after the underwriting discount, but before estimated expenses of the offering payable by DiaMedica, are expected to be approximately $18.8 million. As previously announced, DiaMedica intends to use the net proceeds from the offering to continue its clinical and product development activities, including the addition of a new cohort III to its REDUX study to be comprised of participants with Type II diabetes mellitus with chronic kidney disease, hypertension and albuminuria, and for other working capital and general corporate purposes.

Guggenheim Securities, LLC is acting as lead book-running manager for the offering. Craig-Hallum Capital Group LLC is acting as joint book-running manager and National Securities Corporation, a wholly owned subsidiary of National Holdings Corporation (NASDAQ: NHLD), is acting as lead manager.

The securities described above are being offered by DiaMedica pursuant to a shelf registration statement on Form S-3 (File No. 333-235775) previously filed with and declared effective by the U.S. Securities and Exchange Commission (SEC). A preliminary prospectus supplement and accompanying prospectus relating to and describing the terms of the offering were filed with the SEC on August 5, 2020. The final prospectus supplement and the accompanying prospectus relating to this offering will be filed with the SEC and, when available, may be obtained by visiting the SEC’s website at www.sec.gov or by contacting 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].

On August 6, 2020 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2020, and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 6, 2020, View Source [SID1234562975]).

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"BioCryst is currently in an exciting transformation from a company primarily focused on R&D to one that is about to launch its first oral drug to patients with HAE (ORLADEYO) this year, generating meaningful revenue starting next year, with global peak sales potential of greater than half a billion dollars. In parallel, we plan to fill our pipeline with a single molecule, BCX9930, by investing to accelerate this program across multiple rare disease indications based on the proof of concept data we have," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We expect to end the year with ORLADEYO approved in the U.S. and Japan, additional data at 200 mg and 400 mg with BCX9930 in PNH patients, and clinical data from galidesivir in COVID-19 patients," Stonehouse added.

Program Updates and Key Milestones

Hereditary Angioedema (HAE) Program – ORLADEYO: Oral, once-daily treatment for prevention of HAE attacks

BioCryst expects three regulatory approvals for ORLADEYO in Q4 2020 and early 2021.

The U.S. Food and Drug Administration (FDA) is reviewing a new drug application for ORLADEYO and has set an action date of December 3, 2020, under the Prescription Drug User Fee Act (PDUFA).

In Japan, ORLADEYO is being reviewed under Sakigake designation. The Pharmaceutical and Medical Devices Agency (PMDA) has confirmed their regulatory review schedule and the company expects an approval decision in December 2020.

On March 30, 2020, the company announced that the European Medicines Agency (EMA) had validated its marketing authorization application (MAA) submission for ORLADEYO and begun its formal review of the MAA under the centralized procedure. The company expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) within approximately 12 months from MAA validation.

BioCryst has completed significant preparations to support the launch of ORLADEYO in the U.S.

The company has attracted an accomplished U.S. rare disease sales team, which averages 20 years in pharmaceutical sales and nearly a decade of rare disease experience.

The company is well-positioned in terms of product supply and inventory on-hand to support the launch and anticipated global demand for ORLADEYO.

On June 9, 2020, the company announced that it had established an expanded access program with ORLADEYO for patients with HAE in the United States. Through this program, physicians may be able to request ORLADEYO for HAE patients who do not have access to the product through a clinical trial.

On June 6, at the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress, the company presented new data highlighting the burden of therapy faced by HAE patients taking currently available injectable prophylactic medication. Patients taking oral, once daily ORLADEYO experienced sustained decreases in their attack frequency and improvements in quality of life scores over 48 weeks. ORLADEYO was also safe and generally well-tolerated over 48 weeks in both the APeX-2 and APeX-S clinical trials.
"Our ongoing dialogue and research with HAE patients and physicians continue to reinforce their strong demand for an oral, once-daily medicine that is safe and provides the significant and sustained attack reduction we are seeing with ORLADEYO in our clinical program. Nearly half of patients in our APeX-2 and APeX-S trials have prior experience with injectable or infused therapies and most have chosen to remain on ORLADEYO," said Charlie Gayer, chief commercial officer of BioCryst.

Complement Oral Factor D Inhibitor Program – BCX9930

On August 3, 2020, the company announced that the FDA has granted Fast Track designation for BCX9930 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). According to the FDA, the purpose of the Fast Track designation is to get important new drugs to the patient earlier by facilitating the development, and expediting the review of, drugs to treat serious conditions and fill an unmet medical need.

On May 6, 2020, the company reported proof of concept data for BCX9930 in three treatment-naïve PNH patients in the lowest dose cohort of 50 mg (14 days) and 100 mg (14 days) twice-daily. BCX9930 inhibited complement and was safe and generally well tolerated.

Based on the investigators’ assessment, three patients receiving 50 mg and 100 mg twice daily experienced clinical benefit, continued on therapy and are now receiving BCX9930 therapy at higher doses. An additional treatment-naïve PNH cohort, which starts at 200 mg (14 days) followed by 400 mg (14 days) twice-daily, has also begun enrollment. The company expects to report data from treatment-naïve PNH patients receiving 200 mg / 400 mg twice daily in Q3 2020.

The company plans to report data from PNH patients who are poor responders to C5 inhibitors receiving 200 mg / 400 mg twice daily by the end of 2020.

Based on the safety and proof-of-concept data generated to date in PNH patients, the company is working closely with key opinion leaders in hematology and nephrology to map the development strategy across a broad set of indications.
"We plan to meet with regulators later this year to confirm our advanced development plan for BCX9930 in PNH. Because the alternative pathway of complement is also directly related to several complement-mediated diseases, we plan to broaden and accelerate the BCX9930 program to patients in multiple disease areas as quickly as possible," said Dr. William Sheridan, chief medical officer of BioCryst.

Coronavirus Antiviral Program – Galidesivir (BCX4430)

In response to the COVID-19 pandemic, the company is conducting a randomized, double-blind, placebo-controlled clinical trial (NCT03891420) in Brazil to assess the safety, clinical impact and antiviral effects of galidesivir in COVID-19 patients. The company expects to provide information from Part 1 of this ongoing clinical trial by the end of Q3 2020.

The galidesivir program is being substantially funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and the Biomedical Advanced Research and Development Authority (BARDA). The goal of the galidesivir clinical program is to evaluate clinical activity that would support additional government investment and advanced development of galidesivir, including additional drug supply.
Additional Updates

The company remains on track to report data in 2H 2020 from its ongoing Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of fibrodysplasia ossificans progressiva (FOP), in healthy subjects.
Second Quarter 2020 Financial Results

For the three months ended June 30, 2020, total revenues were $2.9 million, compared to $1.4 million in the second quarter of 2019. The increase was primarily due to an increase in collaboration revenue under U.S. government development contracts.

Research and development (R&D) expenses for the second quarter of 2020 were $27.5 million, similar to $27.7 million in the second quarter of 2019. During the second quarter of 2020, R&D spending increased on the company’s complement-mediated diseases and galidesivir programs, which was offset by a reduction in spend on the ORLADEYO program as the company approaches commercial launch.

Selling, general and administrative (SG&A) expenses for the second quarter of 2020 increased to $13.9 million, compared to SG&A expenses of $8.7 million in the second quarter of 2019. The increase was primarily due to increased spending on commercial activities and medical affairs to support the U.S. commercial launch of ORLADEYO in 2020.

Interest and other income were $2.8 million in the second quarter of 2020, compared to $0.5 million in the second quarter of 2019. The increase was primarily due to recognition of income from our arbitration proceedings.

Interest expense was $2.9 million in the second quarter of 2020, compared to $3.0 million in the second quarter of 2019.

Net loss for the second quarter of 2020 was $38.6 million, or $0.24 per share, compared to a net loss of $37.6 million, or $0.34 per share, for the second quarter of 2019.

Cash, cash equivalents and investments and restricted cash totaled $191.6 million at June 30, 2020, and reflect an increase from $137.8 million at December 31, 2019 and $114.6 million at March 31, 2020. Operating cash use for the second quarter of 2020 was $31.8 million.

Financial Outlook for 2020

With the additional capital raised in Q2 and the safety and proof-of concept data generated to-date with BCX9930 in PNH patients, the company is investing in accelerated development of BCX9930 and expects full year 2020 net operating cash use to be in the range of $150 to $165 million, and its operating expenses to be in the range of $180 to $195 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 5142479. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 5142479.