On August 1, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported its financial results and corporate update for the quarter ended June 30, 2019 (Press release, Ultragenyx Pharmaceutical, AUG 1, 2019, View Source [SID1234538022]).

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"The U.S. launch of Crysvita continues to be strong with the number of patients on reimbursed therapy increasing significantly this quarter and the number of unique prescribers continuing to grow steadily," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "Earlier this week we submitted the New Drug Application for UX007, which marks significant progress in bringing this important therapy to patients, and we are advancing our gene therapy platform with updates expected from the two clinical-stage programs in the third quarter."

Second Quarter 2019 Financial Results

Net Revenues

For the second quarter of 2019, Ultragenyx reported $24.1 million in total revenue, including $20.2 million in Crysvita revenue. Crysvita revenue includes $17.3 million in collaboration revenue in the North American profit share territory, $1.9 million in royalty revenue in the European territory from Kyowa Kirin Co., and product revenue in other regions of $1.0 million. Mepsevii (vestronidase alfa) product revenue for the second quarter of 2019 was $3.2 million and UX007 named patient revenue was $0.6 million. Ultragenyx also recognized $0.1 million in revenue from its research agreement with Bayer.

Revenue for the six months ended June 30, 2019 was $42.3 million, including $34.7 million in Crysvita revenue. Crysvita revenue includes $29.2 million in collaboration revenue in the North American profit share territory, $3.9 million in royalty revenue in the European territory from Kyowa Kirin Co., and product revenue for Crysvita in other regions of $1.6 million. Mepsevii product revenue for the six months ended June 30, 2019 was $5.9 million and UX007 named patient revenue was $1.3 million. Ultragenyx also recognized $0.4 million in revenue from its research agreement with Bayer in the first half of 2019.

Operating Expenses

Total operating expenses for the second quarter of 2019 were $136.6 million compared with $107.7 million for the same period in 2018, including a $15.6 million research and development expense in the second quarter of 2019 from the Arcturus collaboration amendment, and non-cash stock-based compensation of $22.2 million and $19.6 million in the second quarter of 2019 and 2018, respectively. Total operating expenses for the six months ended June 30, 2019 were $254.0 million compared with $214.9 million for the same period in 2018, including a $15.6 million research and development expense from the Arcturus collaboration amendment in the second quarter of 2019, and non-cash stock-based compensation of $42.4 million and $38.4 million in the first half of 2019 and 2018, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its pipeline.

For the second quarter of 2019, Ultragenyx reported a net loss of $99.2 million, or $1.72 per share, basic and diluted, compared with a net loss for the second quarter of 2018 of $52.7 million, or $1.06 per share, basic and diluted. For the six months ended June 30, 2019, net loss was $195.9 million, or $3.54 per share, basic and diluted, compared with a net loss for the same period in 2018 of $22.5 million, or $0.46 per share, basic and diluted. The loss for the second quarter of 2019 and for the six months ended June 30, 2019 is net of a $9.8 million unrealized gain from the fair value adjustment on the investment in Arcturus equity. The loss for the second quarter of 2018 is net of a $40.3 million gain from Ultragenyx’s portion of the sale of the priority review voucher (PRV) received with the Crysvita U.S. Food and Drug Administration (FDA) approval. In addition to the Crysvita PRV, the loss for the first six months of 2018 also is net of the $130.0 million gain from the sale of the PRV received with the Mepsevii FDA approval. The net loss for the first six months of 2019 reflected cash used in operations of $184.8 million compared to $165.6 million for the same period in 2018.

Cash, Cash Equivalents and Investments

Cash, cash equivalents and available-for-sale investments were $618.3 million as of June 30, 2019, which factors in the $30 million paid in the Arcturus collaboration agreement.

Recent Updates and Upcoming Milestones

Crysvita Commercial Progress in X-Linked Hypophosphatemia (XLH)

Strong U.S. launch continues, with approximately 960 patients on reimbursed commercial therapy in the United States at the end of the second quarter 2019.
UX007 NDA submitted to FDA for the treatment of Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Ultragenyx submitted a New Drug Application (NDA) to the FDA earlier this week. The FDA previously granted Rare Pediatric Disease Designation and Fast Track designation, which enables eligibility for Priority Review, if relevant criteria are met. The company expects to hear back from FDA on submission acceptance and review designation within 60 days.
Clinical-stage Gene Therapy Programs Advance

DTX301 Phase 1/2 study in ornithine transcarbamylase (OTC) deficiency: Previous data from Cohorts 1 and 2 demonstrated that the two responders maintained ureagenesis levels above normal for 78 and 52 weeks, respectively. There have been no infusion-related adverse events and no treatment-related serious adverse events reported.
DTX401 Phase 1/2 Study in glycogen storage disease type Ia (GSDIa): Previous data from Cohort 1 demonstrated that all three patients showed improvements in glucose control reflected by prolonged time to hypoglycemia during a controlled fasting challenge. All patients have sustained their reductions in cornstarch compared to baseline, and continue to maintain normal glucose levels throughout the day and overnight. There have been no infusion-related adverse events and no treatment-related serious adverse events reported.
Ultragenyx plans to provide updates from the third dose cohort of the DTX301 study in OTC and the second dose cohort of the DTX 401 study in GSDIa in the third quarter of 2019.
Corporate Updates

Expanded collaboration with Arcturus Therapeutics to develop additional nucleic acid therapies. The research collaboration and license agreement with Arcturus Therapeutics now includes the discovery and development of mRNA, DNA, and siRNA therapeutics for up to 12 rare disease targets.
Erik Harris named Chief Commercial Officer. In June 2019, Ultragenyx promoted Erik Harris to Executive Vice President and Chief Commercial Officer. Mr. Harris joined Ultragenyx in 2017 as Senior Vice President, Head of North American Commercial Operations, leading the launches of Crysvita and Mepsevii. In his new role, Mr. Harris will be responsible for all commercial operations in North America, Europe, and Latin America.
Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, August 1, 2019, at 2 p.m. PT/ 5 p.m. ET to discuss second quarter 2019 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 6298416. The replay of the call will be available for one year.

On August 1, 2019 Adamis Pharmaceuticals Corporation (Nasdaq: ADMP), a specialty biopharmaceutical company focused on developing and commercializing products in various therapeutic areas, including respiratory disease, allergy and opioid overdose, reported the pricing of its previously announced underwritten public offering of 12,000,000 shares of its common stock and warrants to purchase up to 12,000,000 shares of its common stock (Press release, Adamis Pharmaceuticals, AUG 1, 2019, View Source [SID1234538038]). Each share of common stock is being sold together with a warrant to purchase one share of common stock for a combined public offering price of $1.00, resulting in gross proceeds of approximately $12,000,000, before deducting underwriting discounts and commissions and other estimated offering expenses payable by the company and excluding the proceeds from the exercise of any warrants. All shares of common stock and warrants to purchase common stock to be sold in the public offering are being sold by Adamis. The warrants will be exercisable commencing on the date of issuance, will expire five years from the date of issuance, and have an exercise price of $1.15 per share, subject to certain adjustments. The shares of common stock and warrants will be purchased together but will be issued separately and will be immediately separable upon issuance.

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The offering is expected to close on August 5, 2019, subject to the satisfaction of customary closing conditions. The company has also granted the underwriters a 30-day option to purchase up to 1,800,000 additional shares of its common stock and/or warrants to purchase up to 1,800,000 additional shares of its common stock.

Raymond James & Associates, Inc. is acting as the sole book-running manager for the offering. Maxim Group LLC is acting as lead manager for the offering.

The company intends to use the net proceeds from this offering for general corporate purposes, which may include, without limitation, expenditures relating to research, development and clinical trials relating to its products and product candidates, manufacturing, capital expenditures, hiring additional personnel, acquisitions of new technologies or products, the repayment, refinancing, redemption or repurchase of existing or future indebtedness or capital stock and working capital.

The securities described above are being offered by the company pursuant to a "shelf" registration statement on Form S-3 (File No. 333-226100) previously filed with and declared effective by the Securities and Exchange Commission (the "SEC") on July 18, 2018. A preliminary prospectus supplement and the related prospectus have been filed with the SEC and are available on the SEC’s website at www.sec.gov. A final prospectus supplement and an accompanying prospectus related to the offering will be filed with the SEC and will be available on the SEC’s website located at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained by contacting Raymond James & Associates, Inc., Attention: Equity Syndicate, 880 Carillon Parkway, St. Petersburg, Florida, or by telephone at (800) 248-8863, or e-mail at [email protected].

Before investing in the offering, you should read in their entirety the prospectus supplement and the accompanying prospectus and the other documents that the company has filed with the SEC that are incorporated by reference in the prospectus supplement and the accompanying prospectus, which provide more information about the company and the offering.

This press release does not constitute an offer to sell or a solicitation of an offer to buy any securities described herein, 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 registration or qualification under the securities laws of any such state or jurisdiction.

On August 1, 2019 SenesTech, Inc. (NASDAQ: SNES), a developer of proprietary technologies for managing animal pest populations through fertility control, reported that will report financial results for its second quarter of 2019, ended June 30, 2019, after the market close on Tuesday, August 13, 2019 (Press release, SenesTech, AUG 1, 2019, View Source [SID1234538054]). The Company has scheduled a conference call that same day, Tuesday, August 13, 2019, at 5:00 pm ET, to review the results.

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Second Quarter 2019 Conference Call Details

Date and Time: Tuesday, August 13, 2019 at 5:00 pm ET

Call-in Information: Interested parties can access the conference call by dialing (844) 308-3351 or (412) 317-5407.

Live Webcast Information: Interested parties can access the conference call via a live Internet webcast, which is available in the Investor Relations section of the Company’s website at View Source." target="_blank" title="View Source." rel="nofollow">View Source

To Ask a Question: There will be three options to ask a question during the call:

Questions can be asked live during the call-in portion of the conference call.
The live webcast will feature an option to submit questions in writing during the event.
If you are unable to attend the event, you can submit a question in advance to [email protected].
Replay: A teleconference replay of the call will be available for three days at (877) 344-7529 or (412) 317-0088, confirmation #10134038. A webcast replay will be available in the Investor Relations section of the Company’s website at View Source for 90 days.

About SenesTech
SenesTech is changing the paradigm of pest management by targeting the root cause of the problem: reproduction.

ContraPest is an innovative technology with an approach

On August 1, 2019 Rakuten Medical, Inc., a clinical-stage, global biotechnology company developing precision-targeted cancer therapies based on its proprietary Photoimmunotherapy (PIT) platform, reported that it has raised approximately US $100 million on July 31, 2019, in a Series C-1 Preferred Stock financing from Rakuten, Inc., a global leader in internet services headquartered in Japan (Press release, Rakuten Medical, AUG 1, 2019, View Source [SID1234538083]). This investment is increasing Rakuten’s equity share of Rakuten Medical to 22.6 percent.

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Rakuten Medical intends to use the additional injection of capital to expand the development of the company’s oncology-focused PIT platform, strengthen its business and commercial functions, and provide resources to further identify and evaluate new investigational compounds and indications for the potential treatment of other types of cancers using PIT.

"The additional investment from Rakuten solidifies our commitment to accelerating Rakuten Medical’s business and commercial development," said Mickey Mikitani, chairman and CEO of Rakuten Medical. "I believe we can cultivate a sustainable health care ecosystem to provide patients with safe and easy access to, and better care in, the treatment of cancer, regardless of their nationality or income. Culturally, the technology industry has revolutionized society by being bold, action-driven and innovative; and we hope to do the same at Rakuten Medical by exploring opportunities that will combine Rakuten’s technology expertise with Rakuten Medical’s first-in-class Photoimmunotherapy platform."

Based on the additional investment, Rakuten Medical will become an equity-method affiliate of Rakuten in the third quarter of this fiscal year.

About Photoimmunotherapy
Photoimmunotherapy (PIT) is an investigational, anti-cancer treatment platform that is comprised of a drug and device combination that utilizes monoclonal antibodies conjugated to a dye (IRDye 700DX). Transient excitation of IRDye 700Dx with non-thermal red light (690 nm) is believed to result in anti-cancer activity, which is mediated by biophysical processes that may compromise the membrane integrity of cells. The requirement of targeted binding of antibody-IR700 conjugate to a specific antigen on the cell surface and subsequent illumination is believed to result in rapid and selective cell killing and tumor necrosis with minimal effects on surrounding normal tissue. PIT may also lead to the systemic induction of innate and adaptive immunity.

On August 1, 2019 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the second quarter ended June 30, 2019 (Press release, Agios Pharmaceuticals, AUG 1, 2019, View Source [SID1234537982]).

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"In the second quarter we demonstrated our ability to execute across all areas of our business. Our commercial team continues to deliver on the AML launch for TIBSOVO and prepare for our first potential solid tumor launch on the heels of the positive ClarIDHy study in cholangiocarcinoma," said Jackie Fouse, Ph.D., chief executive officer at Agios. "On the clinical side, we continued to broaden our IDH program into earlier lines of AML therapy and expand our PKR programs into new disease areas while enrolling the PK deficiency pivotal studies. In addition, we completed dose escalation in our AG-270 Phase 1 trial and are on track to initiate the next phase of development. The great progress we made during the quarter keeps us on track to deliver on our remaining 2019 milestones and drive further value across our portfolio."

SECOND QUARTER 2019 HIGHLIGHTS & RECENT PROGRESS

Received approval from the U.S. Food and Drug Administration (FDA) on May 2, 2019 for single agent TIBSOVO for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) with an IDH1 mutation who are ≥ 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Announced the Phase 3 ClarIDHy trial of TIBSOVO in advanced previously treated cholangiocarcinoma patients with an IDH1 mutation met its primary endpoint, demonstrating improvement in progression-free survival by independent radiology review compared with patients who received placebo.
Presented updated data from the Phase 1 studies of TIBSOVO in frontline AML, both as a monotherapy and in combination with azacitidine, and the first data from the perioperative study of TIBSOVO and vorasidenib in glioma at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data presentations can be found here.
Began dosing patients in the Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in advanced lymphoma.
Completed the single agent dose-escalation portion of the ongoing Phase 1 study of AG-270 in methylthioadenosine phosphorylase (MTAP)-deleted tumors.
Appointed Orlando Oliveira to the role of senior vice president and general manager, international. Mr. Oliveira will be responsible for building and leading the company’s operations outside of the U.S. in support of the expected launch of TIBSOVO in Europe and potentially other select markets.
KEY UPCOMING MILESTONES

The company plans to achieve the following key milestones in the remainder of 2019:

Oncology:

Submit a supplemental new drug application to the FDA for TIBSOVO for advanced previously treated IDH1 mutant cholangiocarcinoma by year-end.
Initiate a registration-enabling Phase 3 study of vorasidenib in low-grade glioma with an IDH mutation by year-end.
Initiate expansion phase for the Phase 1 study of AG-270 in MTAP-deleted tumors, including two combination arms with AG-270 and taxanes in non-small cell lung cancer and pancreatic ductal adenocarcinoma, in the third quarter.
Rare Genetic Diseases:

Complete enrollment in two global pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end 2019:
ACTIVATE-T: A single-arm trial of up to 40 regularly transfused patients
ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions
Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.
ANTICIPATED 2019 DATA PRESENTATIONS

Full data from the Phase 3 ClarIDHy study of TIBSOVO in IDH1 mutant advanced previously treated cholangiocarcinoma have been submitted for presentation at the European Society for Medical Oncology Congress taking place in Barcelona, Spain from September 27-October 1, 2019.
Data from the single agent dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with MTAP-deleted tumors have been submitted to the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) taking place in Boston from October 26-30, 2019.
Data from IDH and PKR programs have been submitted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place in Orlando, Fla. from December 7-10, 2019, including new data from the extension phase of the Phase 2 DRIVE PK study of mitapivat in adults with PK deficiency and important translational data from the Phase 1 combination study of TIBSOVO and azacitidine in frontline AML.
SECOND QUARTER 2019 FINANCIAL RESULTS

Revenue: Total revenue for the second quarter of 2019 was $26.2 million, which includes $13.7 million of net product revenue from U.S. sales of TIBSOVO, $9.0 million in collaboration revenue and $2.7 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene. This compares to revenue of $40.4 million for the second quarter of 2018, which included recognition of a $15 million milestone from Celgene related to Celgene’s filing of an MAA to the EMA for IDHIFA and $12.4 million from the signing of the CStone collaboration.

Cost of Sales: We began U.S. sales of TIBSOVO in the third quarter of 2018. Cost of sales were $0.3 million for the second quarter of 2019.

Research and Development (R&D) Expenses: R&D expenses were $107.4 million for the second quarter of 2019 compared to $86.7 million for the second quarter of 2018. The increase in R&D expense was primarily attributable to vorasidenib Phase 3 low grade glioma trial start-up costs, the mitapivat pivotal program in PK deficiency and Phase 2 study in thalassemia, and clinical trial activity related to the ongoing Phase 1 trials for AG-270 and AG-636.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $32.4 million for the second quarter of 2019 compared to $26.6 million for the second quarter of 2018. The increase in SG&A expense was primarily attributable to costs to support commercialization of TIBSOVO and personnel costs related to increased headcount.

Net Loss: Net loss was $109.9 million for the second quarter of 2019 compared to $68.7 million for the second quarter of 2018.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2019 were $624.0 million compared to $805.4 million as of December 31, 2018. The net decrease of $181.4 million in cash position was primarily driven by net expenditures to fund operations, including a onetime cash expense of $19.2 million for bonus payouts during the first quarter. The company expects that its cash, cash equivalents and marketable securities as of June 30, 2019, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2019 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 9319039. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.