On May 8, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer, autoimmune disease, asthma and allergic diseases, reported that company management will present at two upcoming conferences (Press release, Xencor, MAY 8, 2019, View Source [SID1234535955]):

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Bank of America Merrill Lynch 2019 Health Care Conference
Date: Wednesday, May 15, 2019
Location: Las Vegas, NV
Presentation Time: 4:20 p.m. PT
Berenberg Conference USA 2019
Date: Wednesday, May 22, 2019
Location: Tarrytown, NY
Presentation Time: 9:45 a.m. ET
A live webcast of the Bank of America Merrill Lynch conference will be available under "Events & Presentations" in the Investors section of the Company’s website located at View Source A replay of the event will be posted on the Xencor website approximately one hour after the live event and will be available for 90 days following the event.

On May 8, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported financial results for the quarter ended March 31, 2019, and provides an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 8, 2019, View Source [SID1234535954]).

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"Last quarter, we continued to lay the groundwork towards achieving our objective of having four programs in the clinic by year end," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We initiated our Phase 1 clinical trial for FA ‘Process B’ and are encouraged by the continued long-term clinical profile of patients receiving ‘Process A’ RP-L102. We are also encouraged by the strong preclinical data package of RP-A501 as it nears the clinic. In non-human primate studies, RP-A501 demonstrated robust transduction and LAMP2 protein expression in all four heart chambers, the key target organ for Danon disease."

"We remain focused on our core strategic goal of bringing transformative gene therapies to patients with devastating rare diseases, as quickly as possible. With the closing of our most recent equity raise, we are well capitalized to advance our pipeline and look forward to providing updates on two clinical programs by year end," Dr. Shah concluded.

Recent Pipeline and Corporate Updates

Presentation of preclinical and clinical updates at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2019 Annual Meeting. At ASGCT (Free ASGCT Whitepaper), Rocket’s oral and poster presentations highlighted promising data on four gene therapy programs: Fanconi Anemia (FA), Danon disease, Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD). Oral and poster presentations are available online: View Source
Patient dosing commences in Phase 1 clinical study for FA with improved "Process B". The Center for Definitive and Curative Medicine at Stanford is serving as the lead U.S. clinical site. "Process B" incorporates a modified cell enrichment process, transduction enhancers, and commercial-grade vector manufacturing and cell processing. Initial patients have received treatment under "Process B" at Stanford under a U.S.-focused Phase 1 study, with additional Phase 2 enrollment anticipated in the second half of 2019. Preliminary data from the Phase 1 "Process B" study are expected by the end of 2019.
Partnership with University of California, Los Angeles (UCLA) to lead U.S. clinical development efforts for LAD-I and Infantile Malignant Osteopetrosis (IMO) programs. UCLA and its Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research is serving as the lead U.S. clinical research center for the planned registrational clinical trial for LAD-I and also a lead U.S. clinical site for IMO.
Grant awarded from California Institute for Regenerative Medicine (CIRM). CIRM awarded Rocket a $6.5 million grant to support the development of RP-L201 for LAD-I. The grant will help fund the U.S. Phase 1/2 LAD-I registration-enabling study. The trial will evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene.
Rare Pediatric Disease designation for IMO. Rocket received Rare Pediatric Disease designation from the Food and Drug Administration (FDA) for RP-L401 for the treatment of IMO. The FDA defines a "rare pediatric disease" as a serious and life-threatening disease that affects less than 200,000 people in the U.S. that are aged between birth to 18 years. The Rare Pediatric Disease designation program allows for a Sponsor who receives an approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
Approximately $90.6 million secured in public equity offering. Rocket successfully closed an oversubscribed underwritten public offering of common stock for gross proceeds of approximately $90.6 million. Net of expenses, the Company received approximately $86.0 million in net proceeds.
Anticipated Milestones

FA (RP-L102)
Initial Phase 1 data under "Process B" (2H19)
Regulatory alignment on final endpoints for registration and Phase 2 study (2H19)
Additional data from RP-L102 (2020)
Danon Disease (RP-A501)
Initiation of Phase 1 study (2Q19)
Phase 1 data (2020)
Initiate Phase 2/registration-enabling study (2020)
LAD-I (RP-L201)
Initiation of registration-enabling Phase 1/2 study (2Q19)
Initial Phase 1 data (2H19)
Additional data from RP-L201 (2020)
PKD (RP-L301)
Initiation of Phase 1 study (2H19)
Data from Phase 1 (2020)
IMO (RP-L401)
Initiation of clinical study (1H20)
Upcoming Investor Conferences

Bank of America Merrill Lynch Global Healthcare Conference 2019. Rocket is scheduled to present on Wednesday, May 15, 2019, at 3:40 p.m. Pacific Time. Rocket will also participate in a Gene Therapy Panel on Wednesday, May 15, 2019, at 4:20 p.m. Pacific Time.
First Quarter 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of March 31, 2019, were $196.6 million.
Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $15.1 million for the three months ended March 31, 2019, compared to $5.7 million for the three months ended March 31, 2018. The increase was primarily driven by increases in manufacturing and process development expenses of $5.5 million and research agreement expenses of $1.9 million.
G&A expenses. General and administrative expenses were $3.8 million for the three months ended March 31, 2019, compared to $8.7 million for the three months ended March 31, 2018. The decrease was primarily due to certain stock compensation and severance termination expenses in Q-1, 2018 related to the reverse merger with Inotek Pharmaceuticals Corporation.
Net loss. Net loss was $19.5 million or $0.43 per share (basic and diluted) for the three months ended March 31, 2019, compared to $15.3 million or $0.42 per share (basic and diluted) for the three months ended March 31, 2018.
Shares outstanding. 45,114,437 million shares of common stock were outstanding as of March 31, 2019.
Financial Guidance

Cash position. As of March 31, 2019, we had cash, cash equivalents and investments of $196.6 million. On April 18, 2019, Rocket closed a public offering of 5,175,000 shares of common stock and received net proceeds of approximately $86.0 million. Considering the net proceeds from the public offering, Rocket expects such resources will be sufficient to fund its operations into the first half of 2021. As of April 30, 2019, we had cash, cash equivalents and investments of $273.1 million.

On May 8, 2019 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 Dr. Claus Møller, MD, Ph.D., the Company’s Chief Executive Officer will provide an overview and update on the Company’s business at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas (Press release, Y-mAbs Therapeutics, MAY 8, 2019, View Source [SID1234535953]). The presentation will take place on Wednesday, May 15, 2019, at 3:00 PM PDT, and the presentation deck will be made available on the Company website at the time of presentation.

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On May 8, 2019 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage immunotherapeutics company, reported that it will announce financial results for the first quarter ended March 31, 2019 before the market open and host a conference call on Wednesday, May 15, 2019 (Press release, Altimmune, MAY 8, 2019, View Source [SID1234535952]).

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Conference Call Details
Date: Wednesday, May 15
Time: 8:30 am Eastern Time
Domestic: 877-423-9813
International: 201-689-8573
Conference ID: 13690295
Webcast: View Source

On May 8, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2019 and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 8, 2019, http://ir.biocryst.com/news-releases/news-release-details/biocryst-reports-first-quarter-2019-financial-results [SID1234535951]).

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"In a year with many milestones across our multiple advancing programs of oral medicines for rare diseases, BioCryst has achieved significant progress in the first quarter and we look forward to reporting data from our APeX-2 trial in the second quarter and filing a new drug application by the end of the year," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We believe that oral BCX7353 could be transformative for many HAE patients and provide them with the opportunity for a normal life without the burden and discomfort of frequent injections and infusions," Stonehouse added.

First Quarter 2019 Corporate Developments

The company dosed the first patients in its APeX-J trial in Japan, designed to support potential Japanese approval of BCX7353 for the prevention of HAE attacks.

On March 4, 2019, the company announced that it is advancing BCX9930, an oral Factor D inhibitor, into Phase 1 clinical development in the second quarter of 2019 for the treatment of complement-mediated diseases.

On February 23, 2019, the company announced data from the completed ZENITH-1 trial (including the 250 mg and 500 mg dose cohorts) of BCX7353 for the acute treatment of HAE attacks at the annual meeting of the American Academy of Allergy, Asthma & Immunology. The company plans to commence a Phase 3 trial, ZENITH-2, in the summer of 2019.

On February 6, 2019, the company announced it had entered into a $100 million secured credit facility with MidCap Financial Trust pursuant to the terms and conditions of an amended and restated credit and security agreement.

On January 4, 2019, the company announced it had appointed Steve Aselage to its board of directors.

On January 2, 2019, the company announced the dosing of the first subject in a randomized, placebo-controlled Phase 1 clinical trial to evaluate intravenous galidesivir, its investigational broad-spectrum antiviral drug, in healthy volunteers.
Upcoming Key Milestones

HAE Program – BCX7353

Report 24-week safety and efficacy results from the Phase 3 APeX-2 clinical trial (Q2 2019)

Begin ZENITH-2, a Phase 3 clinical trial of oral BCX7353 (750 mg) for the acute treatment of HAE (Summer 2019)

File a new drug application for oral BCX7353 for the prevention of HAE attacks with the U.S. Food and Drug Administration (FDA) (Q4 2019)

File a marketing authorization application for oral BCX7353 for the prevention of HAE attacks with the European Medicines Agency (EMA) (Q1 2020)
Complement Factor D Inhibitor Program – BCX9930

Begin a Phase 1 trial of BCX9930, an oral Factor D inhibitor for treatment of complement-mediated diseases, in healthy subjects (Q2 2019)

Report Phase 1 results (Q4 2019)
ALK-2 Inhibitor Program – BCX9250

Begin a Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of FOP, in healthy subjects (2H 2019)
First Quarter 2019 Financial Results

For the three months ended March 31, 2019, total revenues were $5.9 million, compared to $4.0 million in the first quarter of 2018. The increase was primarily due to the recognition of $1.7 million of peramivir product sales to Green Cross, the company’s commercial partner in Korea, and an increase in revenue from galidesivir development under U.S. government contracts, and partially offset by lower royalty revenue.

Research and development (R&D) expenses for the first quarter of 2019 increased to $27.5 million from $18.4 million in the first quarter of 2018, primarily due to increased spending on the HAE, preclinical and galidesivir programs.

General and administrative (G&A) expenses for the first quarter of 2019 decreased to $6.2 million, compared to $7.6 million in the first quarter of 2018. The decrease was primarily due to approximately $4.7 million of merger-related costs that were incurred in the first quarter of 2018 but did not recur in 2019, offset by an overall increase in G&A expenses as the company prepares for the commercial launch of BCX7353.

Interest expense was $2.7 million in the first quarter of 2019, compared to $2.2 million in the first quarter of 2018 and was primarily associated with enhancements to the company’s secured credit facility in July 2018 and February 2019.

Net loss for the first quarter of 2019 was $31.1 million, or $0.28 per share, compared to a net loss of $25.8 million, or $0.26 per share, for the first quarter of 2018.

Cash, cash equivalents and investments totaled $121.6 million at March 31, 2019, and reflect a decrease from $128.4 million at December 31, 2018. Cash and investments reflect the proceeds from an enhancement to our secured credit facility in February 2019 and were partially offset by normal operating expenses. Operating cash use for the first quarter of 2019 was $27.1 million.

In February 2019, the company entered into a $100 million secured credit facility with MidCap Financial Trust which further enhanced the company’s cash position with $20 million of immediate additional non-dilutive capital and also provided additional financial flexibility through the ability to draw another $50 million of milestone-based non-dilutive capital.

Financial Outlook for 2019

BioCryst continues to expect net operating cash use to be in the range of $105 to $130 million, and its 2019 operating expenses to be in the range of $120 to $145 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 # 1777029. 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 # 1777029.