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Ultragenyx Reports First Quarter 2019 Financial Results and Corporate Update

On May 6, 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 March 31, 2019 (Press release, Ultragenyx Pharmaceutical, MAY 6, 2019, View Source [SID1234535789]).

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"We are seeing strong, sustained momentum from the Crysvita launch in the United States, driven by increased breadth and depth of prescribers treating both adults and children with XLH," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We are also on track to submit UX007 for LC-FAOD for regulatory review in the coming months, and are advancing our gene therapy platform with key data readouts expected from two clinical programs. In addition, we plan to move three preclinical programs into the clinic in 2020."

First Quarter 2019 Financial Results

Net Revenues

For the first quarter of 2019, Ultragenyx reported $18.2 million in total revenue. Ultragenyx recognized $14.5 million in total Crysvita revenue. This includes $11.9 million in collaboration revenue in the U.S. profit share territory and $2.0 million in royalty revenue in the European territory from the collaboration and license agreement with Kyowa Hakko Kirin. Net product sales for Crysvita in other regions were $0.6 million. Mepsevii (vestronidase alfa) product revenue for the first quarter of 2019 was $2.7 million, and UX007 named patient revenue was $0.7 million. Ultragenyx recognized $0.3 million in revenue from its research agreement with Bayer.

Operating Expenses

Total operating expenses for the first quarter of 2019 were $117.4 million compared with $107.2 million for the same period in 2018, including non-cash stock-based compensation of $20.2 million and $18.8 million in the first quarter 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 first quarter of 2019, Ultragenyx reported a net loss of $96.8 million, or $1.82 per share, basic and diluted, compared with a net income for the first quarter of 2018 of $30.3 million, or $0.63 per basic share and $0.62 per diluted share. The income for the first quarter of 2018 included the $130.0 million gain from the sale of the priority review voucher (PRV). The net loss for the first quarter of 2019 reflected cash used in operations of $95.8 million compared to $89.5 million for the same period in 2018.

Cash, Cash Equivalents and Investments

Cash, cash equivalents and investments were $715.3 million as of March 31, 2019.

Recent Updates and Upcoming Milestones

Crysvita in X-Linked Hypophosphatemia (XLH)

Strong launch continues in the United States, with approximately 730 patients on reimbursed commercial therapy at the end of the first quarter 2019.

Crysvita was approved in Brazil for the treatment of XLH in adults and pediatric patients one year of age and older. Crysvita is approved in three key regions of the world including North America, Europe, and now the first country in Latin America.

UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Fast Track Designation and Rare Pediatric Disease Designation granted by U.S. Food and Drug Administration (FDA). Fast Track designation allows for early and frequent communication with the FDA, and also enables eligibility for Priority Review, if relevant criteria are met.

Ultragenyx is on track to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in mid-2019. The submission will include data from a company-sponsored Phase 2 study of UX007 in 29 patients, data from a long-term safety and efficacy extension study in 75 patients, a retrospective medical record review of 20 original compassionate use patients, data from 70 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect of triheptanoin on cardiac function.

DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

DTX301 data from first two dose cohorts demonstrate long-term normalization of ureagenesis. The two responders in Cohort 1 and 2 have now maintained ureagenesis levels above normal for 52 and 78 weeks.

DTX301 Phase 1/2 data from the third-dose cohort are expected in mid-2019.

DTX401 Gene Therapy in Glycogen Storage Disease Type Ia (GSDIa)

Positive longer-term results from the first cohort of the Phase 1/2 clinical study of DTX401 gene therapy in GSDIa. After 24 weeks of treatment, all three patients have either maintained or further increased their time to hypoglycemia during the controlled fasting challenge compared to baseline. All three patients continue to show a clinical

response with additional improvements in glucose control reflected by prolonged time to hypoglycemia during a controlled fasting challenge. Patients have currently reduced cornstarch intake by approximately 50 to 90% and continue to maintain normal glucose levels throughout the day and overnight. There were no infusion-related adverse events and no treatment-related serious adverse events reported.

DTX401 Phase 1/2 data from the second-dose cohort are expected in mid-2019.

Corporate

Equity financing of approximately $330 million: In March 2019, we completed an underwritten public offering, with net proceeds of approximately $330 million. We intend to use a portion of the net proceeds to build our own GMP gene therapy manufacturing facility to reduce the costs and increase the speed with which we can execute on our gene therapy programs.

Analyst and Investor Day: In April 2019, we held an Analyst and Investor Day that provided an update on recent corporate and pipeline updates, including a deeper dive on the gene therapy platform and three preclinical programs with near-term INDs expected in 2020.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Monday, May 6, 2019, at 2 p.m. PT/ 5 p.m. ET to discuss first 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 3666589. The replay of the call will be available for one year.

Cellectar Reports First Quarter 2019 Financial Results and Provides a Corporate Update

On May 6, 2019 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the first quarter ended March 31, 2019, and provided a corporate update (Press release, Cellectar Biosciences, MAY 6, 2019, View Source [SID1234535788]).

First Quarter and Recent Corporate Highlights

·Announced additional positive top-line results from a relapsed/refractory multiple myeloma cohort in the ongoing Phase 2 clinical study of CLR 131, the Company’s lead product candidate. In the cohort, CLR 131 achieved a 30% overall response rate in the first 10 evaluable patients. Patients received one 30-minute infusion of 25mCi/m2 and CLR 131 represented, on average, seventh line treatment. The Company previously announced an overall response rate of 33% as the fourth line average treatment in patients with R/R diffuse large B-cell lymphoma (DLBCL) also receiving the single, 25mCi/m2 dose of CLR 131.

·Announced median overall survival (mOS) of 22 months in Cohorts 1-4 of the Company’s ongoing Phase 1 clinical trial evaluating CLR 131 for the treatment of relapsed/refractory multiple myeloma. The mOS results were from 15 patients, all of whom were heavily pretreated and averaged five prior lines of systemic therapy.

·Received an exemption from the U.S. Food and Drug Administration (FDA) to the Import Alert placed on the Centre for Probe Development and Commercialization (CPDC) for the use of CLR 131 in connection with the Company’s pediatric Investigational New Drug Application (IND). This exemption has allowed Cellectar to initiate patient enrollment in its Phase 1 pediatric study for the treatment of select relapsed or refractory solid tumors including neuroblastoma, lymphomas and malignant brain tumors.

"The data we have seen thus far for CLR 131 are very compelling with demonstrated activity in at least 3 hematologic cancers. We continue to make significant progress in the clinic and are excited to now provide this promising treatment to pediatric cancer patients," said James Caruso, president and CEO of Cellectar. "We believe CLR 131 has the potential to be a meaningful part of the treatment regimine for patients battling life-threatening cancers and look forward to continuing to provide updates on our studies throughout 2019."

First Quarter Financial Highlights

Research and Development Expense: Research and development expense for the three months ended March 31, 2019 was $2.3 million, compared to $2.1 million in the three months ended March 31, 2018. The overall increase in research and development expense of $184,000, or 8%, was primarily a result of an increase in clinical project costs of approximately $285,000 related to the start-up of the pediatric study. Manufacturing and related costs increased as a result of an increase in patient recruitments for the on-going clinical trials. Pre-clinical studies decreased as some studies were concluding. The general research and development costs were relatively consistent.

General and Administrative Expense: General and administrative expense for the three months ended March 31, 2019 was approximately $1,321,000, compared to approximately $1,329,000 in the three months ended March 31, 2018 and remained relatively consistent.

Net Loss: Net loss for the three months ended March 31, 2019 was $(3.6) million, or a loss of $(0.76) per diluted share, compared to a net loss of $(3.5) million, or a loss of $(2.07) per diluted share, in the three months ended March 31, 2018.

Cash and Cash Equivalents: As of March 31, 2019, cash and cash equivalents were approximately $10.5 million compared to $13.3 million as of December 31, 2018. The Company believes this cash balance is adequate to fund our pipeline development and operations into the first quarter 2020.

About CLR 131

CLR 131 is a small-molecule, cancer-targeting radiotherapeutic PDC (proprietary phospholipid drug conjugate) designed to deliver cytotoxic radiation directly and selectively to cancer cells and cancer stem cells. CLR 131 is our lead therapeutic PDC product candidate and is currently being evaluated in both Phase 2 and Phase 1 clinical studies. In December 2014, the FDA granted orphan drug designation for CLR 131 for the treatment of multiple myeloma. In 2018, the FDA granted orphan drug and rare pediatric disease designations for CLR 131 for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. The FDA previously accepted our IND application for a Phase 1 open-label, dose-escalating study to evaluate the safety and tolerability of a single intravenous administration of CLR 131 in up to 30 children and adolescents with cancers including neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors.

Magenta Therapeutics Launches Proposed Public Offering

On May 6, 2019 Magenta Therapeutics, Inc. (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported that it has launched a proposed public offering of 4,250,000 shares of its common stock (Press release, Magenta Therapeutics, MAY 6, 2019, View Source [SID1234535786]).

All shares of common stock will be offered by Magenta. Magenta expects to grant the underwriters a 30-day option to purchase up to an additional 637,500 shares of common stock. Magenta intends to use the net proceeds of the offering to advance its lead conditioning programs through preclinical studies and into Phase 1 clinical trials; to advance its first-line mobilization program, MGTA-145, through Phase 1 and into Phase 2 clinical trials; and to advance its cell therapy program, MGTA-456, through Phase 2 and into Phase 3 clinical trials. The remainder of the net proceeds will be used for research and development, working capital, and general corporate purposes. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the proposed offering may be completed, or as to the actual size or terms of the proposed offering.

J.P. Morgan, Goldman Sachs & Co. LLC and Cowen are acting as joint book running managers for the offering. Wedbush PacGrow is acting as lead manager for the offering.

The proposed offering will be made only by means of a prospectus. A preliminary prospectus supplement and accompanying prospectuses relating to and describing the terms of this offering has been filed with the U.S. Securities and Exchange Commission (the "SEC"). When available, copies of the preliminary prospectus may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 866-803-9204; Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 212-902-9316 or by emailing [email protected]; Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, telephone: 631-274-2806.

Important Information

A registration statement relating to these securities has been filed with the SEC but has not yet become effective. These securities may not be sold, nor may offers to buy be accepted, prior to the time the registration statement becomes effective. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any offer or sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

Magenta Therapeutics Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

On May 6, 2019 Magenta Therapeutics, Inc. (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported the closing of its previously announced public offering of 4,887,500 shares of its common stock, including 637,500 shares of common stock sold pursuant to the underwriters’ full exercise of their option to purchase additional shares, at a price to the public of $13.25 per share (Press release, Magenta Therapeutics, MAY 6, 2019, View Source [SID1234535785]). The total gross proceeds from the offering (before deducting underwriters’ discounts and commissions and estimated offering expenses) are approximately $64.8 million. All shares of common stock were offered by Magenta.

J.P. Morgan, Goldman Sachs & Co. LLC and Cowen acted as joint book running managers for the offering. Wedbush PacGrow acted as lead manager for the offering.

Important Information

A registration statement relating to the offering has been filed with the United States Securities and Exchange Commission ("SEC") and became effective on May 1, 2019. Copies of the prospectus can be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 866-803-9204; Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 212-902-9316 or by emailing [email protected]; Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, telephone: 631-274-2806.

This press release does not constitute an offer to sell or a solicitation of an offer to buy, nor will there be any offer or sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

Veloxis Pharmaceuticals Announces Financial Results for the First Three Months of 2019

On May 6, 2019 Veloxis Pharmaceuticals A/S (OMX: VELO) reported its Interim Report for the first three months of 2019 (Press release, Veloxis Pharmaceuticals, MAY 6, 2019, View Source [SID1234535782]). This Company Release should be read in conjunction with Veloxis’s full Interim Report for the first three months of 2019, which is attached to this release and also available on Veloxis’s website at: View Source

Product revenue for Q1 2019 was tUSD 14,261, an increase of 96% compared with the same period last year.
US revenue increased 91% to tUSD 12,285
EU revenue increased 133% to tUSD 1,961
Over 91% of US transplant centers have utilized Envarsus since its launch.
Cash balance, tUSD 28,394 on March 31.
Veloxis reported a net income of tUSD 111 for the first quarter of 2019 compared to a net loss of tUSD 4,030 for the same period in 2018.
In connection with the Interim Report, Veloxis’s CEO, Craig Collard said:

"Veloxis is off to a terrific start in 2019 as sales of Envarsus continue to accelerate through the first quarter. We are seeing increased interest from the transplant community driven largely by the approval of the de novo indication in December 2018. We are very excited about what this means for the Envarsus franchise long-term and look forward to a successful year."

Outlook for 2019

Veloxis maintains its 2019 outlook of revenues to be in the range of USD 58 – 68 million and operating income before accounting for stock compensation in the range of USD 4 – 10 million.

Conference Call

A conference call will be held tomorrow, May 7, 2019 at 4:00 p.m. CET (Denmark); 3:00 p.m. GST (London); and 10:00 a.m. EST (New York).

To access the live conference call, please dial one of the following numbers:

DK: +45 32 72 75 18

UK: +44 (0) 203 009 5710

US: +1 917 720 0178

Confirmation Code: 7877813

Following the conference call, a recording will be available on the Company’s website: View Source

About Envarsus

Envarsus is a novel formulation of tacrolimus designed using advance technology which allows for increased bioavailability and controlled, smooth delivery, resulting in in once daily dosing, a lower total daily dose requirement, and lower peak concentrations with less fluctuation.

In addition to the prophylaxis of organ rejection in kidney transplant patients converted from tacrolimus, Envarsus is now FDA-approved for use in de novo kidney transplant patients as of December 2018. That means more patients, including hard-to-treat patients such as rapid metabolizers, can benefit from once-daily controlled-release Envarsus. Envarsus is marketed as Envarsus XR in the Unites States.