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BioMarin to Host Conference Call Tuesday, May 28, 2019 to Provide a Valoctocogene Roxaparvovec Phase 2 and Phase 3 Update

On May 27, 2019 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), reported that management will host a conference call and webcast on Tuesday, May 28 at 8:00am ET to discuss program updates (Press release, BioMarin, MAY 27, 2019, https://www.prnewswire.com/news-releases/biomarin-to-host-conference-call-tuesday-may-28-2019-to-provide-a-valoctocogene-roxaparvovec-phase-2-and-phase-3-update-300857066.html [SID1234536588]).

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Conference Call and Webcast Information
Interested parties may access the live video webcast that will include audio and a slide presentation via the investor section of the BioMarin website, www.biomarin.com. A replay of the meeting will be archived on the site for one week.

For those who want access to the audio portion only, please use the dial-in information below:

U.S. / Canada Dial-in Number: (866) 502-9859
International Dial-in Number: (574) 990-1362
Conference ID: 2295086

Replay Dial-in Number: (855) 859-2056
Replay International Dial-in Number: (404) 537-3406
Conference ID: 2295086

Medicure Reports Financial Results for Quarter Ended March 31, 2019

On May 27, 2019 Medicure Inc. ("Medicure" or the "Company") (TSXV:MPH, OTC:MCUJF), a pharmaceutical company, reported its results from operations for the quarter ended March 31, 2019 (Press release, Medicure, MAY 27, 2019, View Source [SID1234536587]).

Quarter Ended March 31, 2019 Highlights:

Recorded net revenue from the sale of AGGRASTAT (tirofiban hydrochloride) of $4.8 million during the quarter ended March 31, 2019 compared to $6.1 million for the quarter ended March 31, 2018;

$55.5 million in cash and short-term investments as at March 31, 2019;

Adjusted earnings before interest, taxes, depreciation and amortization (EBITDA1) for the quarter ended March 31, 2019 was negative $1.7 million compared to adjusted EBITDA of $927,000 for the quarter ended March 31, 2018; and

Net loss for the quarter ended March 31, 2019 was $2.8 million compared to net income of $1.4 million for the quarter ended March 31, 2018.
Financial Results

Net revenues for the three months ended March 31, 2019 were $4.9 million compared to $6.1 million for the three months ended March 31, 2018. Net revenues from AGGRASTAT for the three months ended March 31, 2019 were $4.8 million compared to $6.1 million for the three months ended March 31, 2018. Additionally, ReDSTM point of care system ("ReDSTM"), contributed $103,000 of net revenue for the three months ended March 31, 2019.

The Company continued to experience strong patient market share held and strong hospital demand for AGGRASTAT during the three months ended March 31, 2019, however increases in volume compared to the three months ended March 31, 2018 were offset by increased price competition that resulted in lower discounted prices for AGGRASTAT throughout the quarter.

Diversification of revenues remains an important aspect of the Company’s focus with Medicure concentrating on the sales and marketing of AGGRASTAT, growing the sales of ZYPITAMAGTM (pitavastatin) and marketing the recently licensed ReDSTM device system.

Adjusted EBITDA for the three months ended March 31, 2019 was negative $1.7 million compared to $927,000 for the three months ended March 31, 2018. The decrease in adjusted EBITDA for the three months ended March 31, 2019 is the result of the higher selling, general and administration expenses caused by the incurrence of additional costs relating to the commercial organization due to the Company’s additional products, higher cost of goods sold attributable to an increased volume of AGGRASTAT sold and lower revenues experienced during the quarter ended March 31, 2019.

Net loss for the three months ended March 31, 2019 was $2.8 million or $0.18 per share. This compares to net income of $1.4 million or $0.09 per share for the three months ended March 31, 2018. Similar to the decrease in adjusted EBITDA, the net loss for the three months ended March 31, 2019 is the result of higher selling, general and administration expenses, higher cost of goods sold and lower revenues experienced during the quarter. Foreign exchange loss relating to a decrease in the value of the U.S. dollar experienced during the quarter ended March 31, 2019 also contributed to the net loss.

At March 31, 2019, the Company had unrestricted cash and short-term investments totaling $55.5 million compared to $71.9 million as of December 31, 2018. The decrease in cash is primarily due to the investment made in Sensible Medical Innovations Ltd. and a decrease in the value of the U.S. dollar as at March 31, 2019 compared to December 31, 2018. Cash flows used in operating activities for the three months ended March 31, 2019 totaled $1.9 million.

All amounts referenced herein are in Canadian dollars unless otherwise noted.

Notes

(1) The Company defines EBITDA as "earnings before interest, taxes, depreciation, amortization and other income or expense" and Adjusted EBITDA as "EBITDA adjusted for non-cash and non-recurring items". The terms "EBITDA" and "Adjusted EBITDA", as it relates to the quarters ended March 31, 2019 and 2018 results prepared using International Financial Reporting Standards ("IFRS"), do not have any standardized meaning according to IFRS. It is therefore unlikely to be comparable to similar measures presented by other companies.

Conference Call Info:

Topic: Medicure’s Q1 2019 Results

Call date: Tuesday, May 28, 2019

Time: 7:30 AM Central Time (8:30 AM Eastern Time)

Canada toll-free: 1 (888) 465-5079 Canada toll: 1 (416) 216-4169

United States toll-free: 1 (888) 545-0687

Passcode: 6562291#

Webcast: This conference call will be webcast live over the internet and can be accessed from the Medicure investor relations page at the following link: View Source

You may request international country-specific access information by e-mailing the Company in advance. Management will accept and answer questions related to the financial results and operations during the question-and-answer period at the end of the conference call. A recording of the call will be available following the event at the Company’s website.

Last Patient Enrolled in the Phase 2 Trial with Transgene’s TG4010 + Nivolumab + Chemotherapy for the First-Line Treatment of Advanced Lung Cancer (NSCLC)

On May 27, 2019 Transgene (Paris:TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of solid tumors, reported that the last patient has been included in the Phase 2 trial evaluating TG4010 in combination with Opdivo (nivolumab) and chemotherapy as a first-line treatment for advanced non-squamous non-small cell lung cancer (NSCLC) with low or no expression of PD-L1 by the tumor cells (Press release, Transgene, MAY 27, 2019, View Source [SID1234536586]).
Transgene confirms that the study’s primary endpoint (objective response rate – ORR) on a minimum of 35 evaluable patients will be reported in Q4 2019.

The Phase 2 clinical trial is exploring the tolerability and efficacy of the combination regimen of Transgene’s TG4010, an investigational active immunotherapy against MUC1 tumor-associated antigen, with Bristol-Myers Squibb’s immune checkpoint inhibitor, Opdivo (nivolumab), which acts by overcoming immune suppression, and standard platinum doublet chemotherapy.

This multi-center single-arm trial has enrolled patients both in the USA and Europe.

The trial has overall ORR as primary endpoint. The study will also assess the safety and tolerability of the regimen together with other efficacy and immunological parameters. More information on the trial can be found on clinicaltrials.gov (NCT03353675).

This trial is being conducted by Transgene under a clinical collaboration agreement with Bristol-Myers Squibb, which is supplying nivolumab.

"We are looking forward to reporting the first efficacy data of our active immunotherapy TG4010, with nivolumab and chemotherapy as a first-line treatment of advanced lung cancer for patients whose tumors express low or undetectable levels of PD-L1", said Maud Brandely, Chief Medical Officer of Transgene. "Today anti-PD-1 therapy is relatively less effective in this large subset of NSCLC patients. With this triple combination regimen, we aim to significantly improve treatment outcomes in this major oncology indication."

The combination of TG4010 immunotherapy and chemotherapy has demonstrated significant efficacy in terms of increased response rate, progression-free survival and overall survival in a randomized, double-blind, placebo-controlled Phase 2b trial in first-line treatment of patients with advanced non-squamous NSCLC (Quoix et al. Lancet Oncol. 2015).

About TG4010
TG4010 is an active immunotherapy that has been designed to express the coding sequences of the MUC1 tumor-associated antigen and the cytokine, Interleukin-2 (IL2). It is based on a modified Vaccinia virus (MVA) and has been shown to induce an immune response against MUC1 expressing tumors, such as non-small cell lung cancer (NSCLC). Its mechanism of action and excellent safety profile make TG4010 a very suitable candidate for combinations with other therapies, including immune checkpoint inhibitors and chemotherapy. The combination of TG4010 immunotherapy and chemotherapy has demonstrated significant efficacy in terms of progression-free survival and overall survival in patients with advanced stage NSCLC (Quoix et al. Lancet Oncol. 2015).
TG4010 is being investigated for the first-line treatment of NSCLC in combination with nivolumab and chemotherapy in patients whose tumors express low or undetectable levels of PD-L1 (NCT03353675).

About Non-Small Cell Lung Cancer (NSCLC)
Lung cancer is one of the most common malignancies worldwide with an estimated 2.1 million new cases annually. It is also a leading cause of cancer-related deaths, accounting for an estimated 1.7 million deaths (Source: GLOBOCAN 2018). Advanced lung cancer remains one of the cancer types with the worst prognosis (five-year survival rate for advanced NSCLC of less than 5%), underlining the still unmet need in this disease despite recent progress.

bridgebio pharma announces filing of registration statement for proposed initial public offering

On May 24, 2019 BridgeBio Pharma, Inc., a clinical-stage biopharmaceutical company focused on genetic diseases, reported that it has filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission (SEC) relating to a proposed initial public offering of shares of its common stock (Press release, BridgeBio, MAY 24, 2019, View Source [SID1234576266]). The number of shares to be offered and the price range for the proposed offering have not yet been determined. BridgeBio has applied to list its common stock on the Nasdaq Global Market under the symbol "BBIO."

J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC, Jefferies LLC, SVB Leerink LLC, KKR Capital Markets LLC, Piper Jaffray & Co., Mizuho Securities USA LLC, BMO Capital Markets Corp. and Raymond James & Associates, Inc. will act as book-running managers for the proposed offering.

The proposed offering will be made only by means of a prospectus. Copies of the preliminary prospectus relating to the proposed offering may be obtained, when available, for free by visiting EDGAR on the SEC’s website at www.sec.gov. Alternatively, copies of the prospectus, when available, may be obtained for free from the offices of J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 1-866-803-9204 or by emailing [email protected]; Goldman, Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526 or by emailing [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, telephone: 1-877-547-6340, or by emailing [email protected]; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 01220, telephone: 1-800-808-7525, ext. 6132, or by emailing [email protected]. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed.

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 the solicitation of an offer to buy these securities, 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.

Myovant Sciences Provides Corporate Updates and Reports Financial Results
for Fourth Fiscal Quarter and Full Fiscal Year Ended March 31, 2019

On May 24, 2019 Myovant Sciences (NYSE: MYOV), a clinical-stage healthcare company focused on developing and commercializing innovative therapies for women’s health and prostate cancer, reported corporate updates and reported financial results for the fourth fiscal quarter and full fiscal year ended March 31, 2019 (Press release, Myovant Sciences, MAY 24, 2019, View Source [SID1234536585]).

"Earlier this month, Myovant reached a significant milestone with the announcement of positive data from LIBERTY 1, the first of two Phase 3 studies of once daily relugolix combination therapy in women with uterine fibroids and heavy menstrual bleeding, which met its primary efficacy endpoint and six key secondary endpoints," said Lynn Seely, M.D., President and Chief Executive Officer of Myovant Sciences. "These positive results bring us one step closer to realizing the potential of relugolix combination as a new treatment option for the millions of women suffering from this debilitating disease. We look forward to reporting data from LIBERTY 2, the second Phase 3 study designed to confirm these results, next quarter and results from our Phase 3 studies in endometriosis and prostate cancer over the next three quarters."
Fourth Fiscal Quarter 2018 and Recent Business Highlights
Relugolix Phase 3 Clinical Programs

On May 14, 2019, Myovant announced positive results from the LIBERTY 1 Phase 3 study evaluating relugolix combination therapy (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg) once daily in women with uterine fibroids and heavy menstrual bleeding. Results from the Phase 3 LIBERTY 2 study are expected in the third quarter of calendar year 2019, and, provided the LIBERTY 2 study is successful, Myovant plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the fourth quarter of calendar year 2019.
MVT-602 Clinical Program

Myovant completed a dose-finding pharmacokinetic/pharmacodynamic Phase 2a study of MVT-602, a kisspeptin-1 receptor agonist, in healthy women undergoing a controlled ovarian stimulation protocol. Top-line results are expected to be presented at the European Society of Human Reproduction in Vienna, Austria in June.
Corporate

In the fourth quarter of fiscal year 2018, Myovant issued and sold 1,203,000 common shares for aggregate net proceeds of $26.7 million pursuant to its "at-the-market" equity offering program. In April 2019, Myovant issued and sold an additional 106,494 common shares for aggregate net proceeds of $2.5 million pursuant to the "at-the-market" equity offering program.

Fourth Fiscal Quarter and Full Fiscal Year 2018 Financial Summary
Research and development (R&D) expenses for the quarter ended March 31, 2019, were $59.0 million compared to $40.1 million for the comparable period in 2018. R&D expenses for the fiscal year ended March 31, 2019, were $222.6 million, compared to $116.8 million for the prior fiscal year. The increase for both the quarter and the year primarily reflects the progress of Myovant’s Phase 3 clinical studies of relugolix, as well as additional personnel-related expenses and increased MVT-602 clinical trial expenses.
General and administrative (G&A) expenses for the quarter ended March 31, 2019, were $12.5 million compared to $7.3 million for the comparable period in 2018. G&A expenses for the fiscal year ended March 31, 2019, were $42.2 million, compared to $24.2 million for the prior fiscal year. The increase in G&A expenses for both the quarter and the year primarily reflects increases in personnel-related expenses, professional service fees, share-based compensation expense, and other administrative expenses to support Myovant’s headcount growth and expanding operations.
Interest expense for the quarter ended March 31, 2019, was $3.9 million compared to $1.1 million in the comparable prior year period. Interest expense for the fiscal year ended March 31, 2019, was $8.8 million, compared to $2.0 million for the prior fiscal year. This consists of interest expense related to financing agreements with NovaQuest and Hercules Capital, Inc., as well as the associated non-cash amortization of debt discount and issuance costs. The increase for both the quarter and the year was primarily the result of higher outstanding debt balances under the financing agreements as compared to the prior year periods.
Interest income for the quarter and year ended March 31, 2019, was $0.8 million and $0.9 million, respectively. There was no interest income for the quarter and year ended March 31, 2018. During the year ended March 31, 2019, a portion of Myovant’s cash was invested in a combination of money market funds and commercial paper. There were no such investments during the prior year periods.

Net loss for the quarter ended March 31, 2019, was $75.0 million, compared to $48.3 million for the comparable period in 2018. Net loss for the fiscal year ended March 31, 2019, was $273.6 million, compared to $143.3 million for the prior fiscal year. On a per common share basis, net loss was $1.07 and $0.81 for the quarters ended March 31, 2019, and 2018, respectively, and $4.09 and $2.41 for the fiscal years ended March 31, 2019 and 2018, respectively. The increases in the net loss and net loss per common share for both the quarter and the year were driven primarily by the increase in costs outlined above.
Capital resources: Cash and cash equivalents totaled $156.1 million as of March 31, 2019. Currently, an additional $10.4 million of capacity remains available under the "at-the-market" equity offering program that Myovant initiated in April 2018.
About Relugolix
Relugolix is a once daily oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces ovarian estradiol production, a hormone known to stimulate the growth of uterine fibroids. Myovant is studying relugolix combination therapy (relugolix 40 mg plus 1.0 mg estradiol with 0.5 mg norethindrone acetate) in two Phase 3 clinical studies (LIBERTY 1 and LIBERTY 2) in women with uterine fibroids and heavy menstrual bleeding, and in two Phase 3 clinical studies (SPIRIT 1 and SPIRIT 2) in women with endometriosis-associated pain. Myovant is studying whether the combination optimizes estradiol levels to maximize the benefit of relugolix on symptoms of uterine fibroids and endometriosis, while maintaining bone health and mitigating other side effects from a low-estrogen state such as vasomotor symptoms. Relugolix monotherapy, 120 mg once daily, is also being evaluated in the Phase 3 HERO study in men with advanced prostate cancer.

About MVT-602

MVT-602 is an oligopeptide kisspeptin-1 receptor agonist. Kisspeptin, the ligand, is a naturally-occurring peptide that stimulates GnRH release and is required for puberty and maintenance of normal reproductive function, including production of sperm, follicular maturation and ovulation, and production of estrogen and progesterone in women and testosterone in men. A Phase 2a clinical study in healthy female volunteers to characterize the dose-response curve in the controlled ovarian stimulation setting has been completed. This study is intended to provide information for dose selection for a study of MVT-602 in infertile women seeking pregnancy.