On August 6, 2019 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the second quarter of 2019 and updated 2019 financial guidance (Press release, Jazz Pharmaceuticals, AUG 6, 2019, View Source [SID1234538229]).

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"2019 has been notable for significant execution and accomplishments across all aspects of our business, including strong financial results, the U.S. launch of Sunosi and further expansion and diversification of our development pipeline through internal and acquired R&D programs," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "In the second half of the year, we are focused on continuing to deliver innovative therapies to patients and value to shareholders by preparing to file an NDA for JZP-258, our novel oxybate product candidate, advancing our R&D programs and planning for the potential approval of solriamfetol in the EU."

"We look forward to initiating multiple Vyxeos studies and to working with the Children’s Oncology Group to initiate a pivotal Phase 2/3 study this year for JZP-458, our recombinant crisantaspase product candidate for the treatment of acute lymphoblastic leukemia," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development, of Jazz Pharmaceuticals. "With the addition of the pan-RAF inhibitor program and our exosome therapeutics collaboration, we continue to grow and diversify our R&D pipeline."

Financial Highlights

Three Months Ended
June 30,

Six Months Ended
June 30,

(In thousands, except per share amounts and percentages)

2019

2018

Change

2019

2018

Change

Total revenues

$

534,133

$

500,479

7%

$

1,042,319

$

945,092

10%

GAAP net income

$

261,898

$

92,321

184%

$

347,099

$

138,312

151%

Adjusted net income

$

232,537

$

214,636

8%

$

445,710

$

397,007

12%

GAAP EPS

$

4.56

$

1.50

204%

$

6.01

$

2.26

166%

Adjusted EPS

$

4.05

$

3.49

16%

$

7.72

$

6.48

19%

GAAP net income for the second quarter of 2019 was $261.9 million, or $4.56 per diluted share, compared to $92.3 million, or $1.50 per diluted share, for the second quarter of 2018.

Non-GAAP adjusted net income for the second quarter of 2019 was $232.5 million, or $4.05 per diluted share, compared to $214.6 million, or $3.49 per diluted share, for the second quarter of 2018. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

In the second quarter of 2019, the company recorded a one-time tax benefit of $112.3 million, or $1.96 per diluted share, on a GAAP basis, resulting from an intra-entity intellectual property asset transfer. This tax benefit has been excluded from adjusted net income and the related per share measures for the three and six months ended June 30, 2019. In the second quarter of 2018, GAAP net income included an impairment charge of $42.9 million resulting from the company’s sale of its rights related to Prialt (ziconotide) intrathecal infusion.

Corporate Updates

In July 2019, the company launched Sunosi (solriamfetol) in the U.S. after receiving a schedule IV designation from the U.S. Drug Enforcement Agency (DEA). Sunosi is the first and only dual-acting dopamine and norepinephrine reuptake inhibitor approved to improve wakefulness in adult patients with excessive daytime sleepiness (EDS) associated with narcolepsy or obstructive sleep apnea (OSA).

The company reported the appointment of Neena M. Patil as General Counsel. Ms. Patil will oversee all legal matters for the company. Ms. Patil has been practicing law for nearly 20 years and was most recently Senior Vice President, General Counsel and Corporate Secretary for Abeona Therapeutics Inc. Prior to Abeona, Ms. Patil was Vice President for Legal Affairs, Associate General Counsel and Assistant Corporate Secretary at Novo Nordisk and held various positions at other global pharmaceutical companies. Ms. Patil received a JD from the University of Michigan Law School, a Masters in Health Services Administration from the University of Michigan School of Public Health and an undergraduate Bachelor of Arts degree from Georgetown University.

Key Regulatory/R&D Updates

In June 2019, the Children’s Oncology Group (COG) presented positive Phase 1/2 Vyxeos data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in children and young adults with relapsed/refractory acute myeloid leukemia (AML). Overall response rate1 was 81.1%, with 70% of patients achieving best response after cycle 1 with Vyxeos and the percent of patients who achieved minimal residual disease (MRD) negative status was 75% post-cycle 1 and 84% overall. Given the robust overall response rate, the company intends to discuss the data and its plans for regulatory submissions with health authorities.

In June 2019, Nippon Shinyaku Co., Ltd. announced that Japan’s Ministry of Health, Labour and Welfare approved the marketing authorization of Defitelio injection 200mg (defibrotide sodium) for the treatment of sinusoidal obstruction syndrome/hepatic veno-occlusive disease.

In July 2019, the company acquired Redx Pharma plc’s (Redx) pan-RAF inhibitor program for the potential treatment of RAF and RAS mutant tumors. Under the terms of the agreement, the company made an upfront payment of $3.5 million. Redx is eligible to receive up to $203 million in development, regulatory and commercial milestone payments from the company, and incremental tiered mid-single digit royalties, based on any future net sales.

In August 2019, the company announced positive results of a Phase 1 study for JZP-458, its recombinant crisantaspase product candidate, and plans to initiate a single-arm, pivotal Phase 2/3 study. JZP-458 is being evaluated as a potential treatment option for patients with acute lymphoblastic leukemia (ALL)/lymphoblastic lymphoma (LBL) who have had hypersensitivity to E. coli- based asparaginase products.

1 Comprised of complete remission + complete remission with incomplete platelet recovery + complete remission with incomplete hematologic recovery (CR+CRp+CRi).

Select 2019 Milestones

Programs

2019 Milestones*

Xyrem (sodium oxybate) oral solution

✔

Launched for the treatment of cataplexy or EDS in pediatric narcolepsy in March

JZP-258

✔

Announced positive top-line results from the Phase 3 narcolepsy study in March

✔

Received Orphan Drug Designation from FDA for the idiopathic hypersomnia indication

•

Top-line results from the Phase 3 narcolepsy study to be presented at the World Sleep Congress meeting in September

•

NDA submission as early as year-end

Sunosi (solriamfetol)

✔

Received FDA approval for EDS in narcolepsy or OSA in March

✔

Received DEA scheduling decision in June

✔

Launched in the U.S. in July

✔

Identified EDS associated with Major Depressive Disorder as a new area of interest

•

Obtain EU approval for EDS in narcolepsy or OSA as early as year-end

Vyxeos (daunorubicin and cytarabine) liposome for injection

✔

Positive data presented by COG in children and young adults with relapsed/refractory AML at ASCO (Free ASCO Whitepaper) in June

✔

Activated sites for Phase 1 attenuated dose finding study of Vyxeos in higher risk myelodysplastic syndrome (MDS) through MD Anderson collaboration

✔

Activated sites for Phase 1b study of low intensity therapy of Vyxeos in combination with venetoclax in first-line, unfit AML

✔

Activated sites for Phase 3 study in adult patients with newly diagnosed standard- and high-risk AML through the AML Study Group, a cooperative group

✔

Activated sites for Phase 2 study in patients with high-risk MDS through the European Myelodysplastic Syndromes Cooperative Group

•

Potential interim combination data results from studies conducted through MD Anderson collaboration

•

Activate sites for Phase 3 study in newly diagnosed pediatric patients with AML (COG)

•

Activate sites for Phase 2 study in newly diagnosed, fit, older adults with high-risk AML

•

Activate sites for Phase 2 study in a broader age range of adults with high-risk AML

Defitelio (defibrotide sodium) / defibrotide

✔

Positive results from DEFIFrance study presented at European Society for Blood and Marrow Transplant meeting in March

✔

Nippon Shinyaku Co., Ltd. received marketing authorization for Defitelio in Japan in June

•

Provide an update regarding the timing of the interim analysis in the prevention of hepatic veno-occlusive disease (VOD) study

•

Complete enrollment in prevention of acute graft-vs-host disease Phase 2 study

•

Activate sites for exploratory Phase 2 study in chimeric antigen receptor t-cell therapy associated neurotoxicity

•

Activate sites for Phase 2 study in transplant-associated thrombotic microangiopathy

JZP-458

•

Activate sites for single-arm, pivotal Phase 2/3 clinical study later this year in ALL/LBL

CombiPlex

•

Continue Investigational New Drug enabling activities for one solid tumor combination and progress exploratory activities for other hematology/oncology candidates

* Milestones denoted as ✔ have been completed; all other milestones are planned or expected in 2019 unless otherwise noted.

Total Revenues

Three Months Ended
June 30,

Six Months Ended
June 30,

(In thousands)

2019

2018

2019

2018

Xyrem (sodium oxybate) oral solution

$

413,212

$

356,008

$

781,529

$

672,785

Erwinaze / Erwinase (asparaginase Erwinia chrysanthemi)

27,622

58,713

88,521

109,340

Defitelio (defibrotide sodium) / defibrotide

46,055

40,498

87,555

75,559

Vyxeos (daunorubicin and cytarabine) liposome for injection

31,362

27,951

60,305

54,179

Other

5,172

12,925

8,844

25,079

Product sales, net

523,423

496,095

1,026,754

936,942

Royalties and contract revenues

10,710

4,384

15,565

8,150

Total revenues

$

534,133

$

500,479

$

1,042,319

$

945,092

Total revenues increased 7% in the second quarter of 2019 compared to the same period in 2018.

Xyrem net product sales increased 16% in the second quarter of 2019 compared to the same period in 2018.

Erwinaze/Erwinase net product sales decreased 53% in the second quarter of 2019 compared to the same period in 2018 due to ongoing quality and supply issues at the sole manufacturer resulting in minimal supply during the quarter. The company anticipates inter-quarter variability in Erwinaze net sales due to expected supply disruptions during the second half of 2019.

Defitelio/defibrotide net product sales increased 14% in the second quarter of 2019 compared to the same period in 2018 primarily due to an increase in volumes. The second quarter included a shipment to Nippon Shinyaku following the recent approval of Defitelio in Japan. The company continues to expect inter-quarter variability in Defitelio net sales.

Vyxeos net product sales increased 12% in the second quarter of 2019 compared to the same period in 2018 primarily due to the ongoing EU launch. The company continues to implement its intensive education and outreach initiatives while advancing a broad development program to support potential expanded uses of Vyxeos.

Operating Expenses

Three Months Ended
June 30,

Six Months Ended
June 30,

(In thousands, except percentages)

2019

2018

2019

2018

GAAP:

Cost of product sales

$

27,676

$

34,714

$

61,182

$

68,633

Gross margin

94.7%

93.0%

94.0%

92.7%

Selling, general and administrative

$

176,014

$

158,579

$

343,961

$

365,792

% of total revenues

33.0%

31.7%

33.0%

38.7%

Research and development

$

62,384

$

56,132

$

122,489

$

118,799

% of total revenues

11.7%

11.2%

11.8%

12.6%

Impairment charges

$

—

$

42,896

$

—

$

42,896

Acquired in-process research and development

$

2,200

$

—

$

58,200

$

—

Income tax provision (benefit)

$

(78,650)

$

36,524

$

(49,534)

$

55,670

Effective tax rate

(42.7)%

28.2%

(16.5)%

28.6%

Three Months Ended
June 30,

Six Months Ended
June 30,

(In thousands, except percentages)

2019

2018

2019

2018

Non-GAAP adjusted:

Cost of product sales

$

25,968

$

32,911

$

57,815

$

65,136

Gross margin

95.0%

93.4%

94.4%

93.0%

Selling, general and administrative

$

155,329

$

137,706

$

302,906

$

269,685

% of total revenues

29.1%

27.5%

29.1%

28.5%

Research and development

$

56,488

$

51,423

$

111,070

$

98,715

% of total revenues

10.6%

10.3%

10.7%

10.4%

Acquired in-process research and development

$

2,200

$

—

$

2,200

$

—

Income tax provision

$

52,027

$

50,336

$

104,741

$

89,029

Effective tax rate

18.2%

19.0%

19.0%

18.3%

Operating expenses changed over the prior year period primarily due to the following:

Selling, general and administrative (SG&A) expenses increased in the second quarter of 2019 compared to the same period in 2018 on a GAAP and on a non-GAAP adjusted basis primarily due to higher expenses related to the U.S. launch of Sunosi and an increase in headcount and other expenses to support expansion of the business.
Research and development (R&D) expenses increased in the second quarter of 2019 on a GAAP and on a non-GAAP adjusted basis primarily due to expenses related to the company’s pre-clinical and clinical development programs and its partner programs.
Cash Flow and Balance Sheet

As of June 30, 2019, cash, cash equivalents and investments were $882.7 million and the outstanding principal balance of the company’s long-term debt was $1.8 billion. During the six months ended June 30, 2019, the company generated $351.1 million of cash from operations, used $171.1 million to repurchase shares, made an upfront payment of $56.0 million to Codiak BioSciences, Inc. under a collaboration agreement and made milestone payments totaling $25.5 million related to Sunosi.

In the six months ended June 30, 2019, the company repurchased approximately 1.3 million ordinary shares under the company’s share repurchase program at an average cost of $131.17 per ordinary share. As of June 30, 2019, the remaining amount authorized for share repurchases was $208.0 million.

2019 Financial Guidance

Jazz Pharmaceuticals is updating its full year 2019 financial guidance as follows (in millions, except per share amounts and percentages):

Includes minimal net sales contribution from Sunosi in the U.S.

Excludes $6-$8 million of share-based compensation expense from estimated GAAP gross margin.

Excludes $82-$90 million of share-based compensation expense from estimated GAAP SG&A expenses.

Excludes $22-$27 million of share-based compensation expense and $0-$11 million of milestone payments from estimated GAAP R&D expenses.

Excludes the income tax effect of adjustments between GAAP reported and non-GAAP adjusted net income and the income tax benefit related to an intra-entity intellectual property asset transfer.

See "Non-GAAP Financial Measures" below. Reconciliations of non-GAAP adjusted guidance measures are included above and in the table titled "Reconciliation of GAAP to Non-GAAP Adjusted 2019 Net Income Guidance" at the end of this press release.

Conference Call Details

Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. EDT (9:30 p.m. IST) to provide a business and financial update and discuss its 2019 second quarter results. The live webcast may be accessed from the Investors section of the company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 5590569.

A replay of the conference call will be available through August 13, 2019 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 5590569. An archived version of the webcast will be available for at least one week in the Investors section of the company’s website at www.jazzpharmaceuticals.com.

On August 6, 2019 Supernus Pharmaceuticals, Inc. (NASDAQ: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for the second quarter of 2019 and associated Company developments (Press release, Supernus, AUG 6, 2019, View Source [SID1234538255]).

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Commercial Update

Second quarter 2019 product prescriptions for Trokendi XR and Oxtellar XR, as reported by IQVIA, totaled 209,066, a 7.4% increase over the second quarter of 2018.

Net product sales for the second quarter of 2019 were $102.4 million, a 5.5% increase over $97.0 million in the second quarter of 2018. Net product sales by product are as follows:

"Prescription growth for Trokendi XR improved by 4.8% in the second quarter of 2019 as compared to the first quarter of 2019, but not to the degree we had expected," said Jack Khattar, President and CEO of Supernus. "Following the abnormally large seasonal decline we experienced in the first quarter of 2019, reflecting the impact of high deductible managed care programs, prescription growth for Trokendi XR has been hindered by a moderate contraction in the overall topiramate market. In addition, sales deductions, particularly rebates, have not improved in the second quarter of 2019 relative to the first quarter of 2019 as we had expected, but have remained relatively flat." Mr. Khattar added, "As a result, we are revising full year 2019 guidance for net product sales, and, to a lesser extent, operating earnings."

Progress of Product Pipeline

SPN-812 – Novel non-stimulant for the treatment of ADHD

– The Company concluded its pre-New Drug Application (NDA) clinical meeting with the U.S. Food and Drug Administration (FDA) in July 2019, and continues to expect to submit an NDA for SPN-812 in the second half of 2019. Pending FDA approval, the Company continues to expect to launch SPN-812 in the second half of 2020.
– The Company has advanced manufacture of SPN-812 to support the NDA submission and in preparation of commercial launch.
– A Phase III program in adult patients is anticipated to start in the fourth quarter of 2019.

SPN-810 – Novel treatment of Impulsive Aggression in patients with ADHD

– Enrollment in the Phase III P301 trial is complete, with data expected in the fourth quarter of 2019.
– Enrollment in the Phase III P302 trial continues, with data now expected in the first quarter of 2020.
– The Company continues to expect to submit an NDA for SPN-810 in the second half of 2020, and to launch SPN-810, pending FDA approval, in the second half of 2021.
– Enrollment in the open label extension (OLE) study continues at 90% or higher. On average, a patient in the OLE study remains on SPN-810 treatment for approximately 10.7 months, which the Company believes is an encouraging sign of the tolerability and efficacy of SPN-810.
– Patient dosing continues in the Phase III trial (P503) in adolescent patients.

SPN-604 – Novel treatment of bipolar disorder

– The Company remains on track to start a pivotal Phase III program for the treatment of bipolar disorder in the fourth quarter of 2019.

Operating Expenses

Research and development (R&D) expenses in the second quarter of 2019 were $17.0 million, as compared to $20.0 million in the same quarter last year. This decrease is primarily due to the completion of the four Phase III clinical trials for SPN-812, three of which were completed in December 2018 and the fourth in March 2019. Decreased expenses were partially offset by costs to manufacture SPN-812 to support the Company’s upcoming submission of its NDA.

Selling, general and administrative expenses in the second quarter of 2019 were $41.1 million, essentially equivalent to $40.1 million in the same quarter last year.

Operating Earnings and Earnings Per Share

Operating earnings in the second quarter of 2019 were $42.6 million, a 19.3% increase from $35.7 million in the same quarter last year. Operating earnings increased faster than net product sales, which grew by 5.5%, demonstrating the Company’s ability to manage operating expenses and leverage its established infrastructure.

Net earnings (GAAP) in the second quarter of 2019 were $32.7 million, or $0.61 per diluted share, an increase from $30.7 million, or $0.57 per diluted share, in the same period last year. Growth in net earnings was driven primarily from the aforementioned increase in operating earnings, partially offset by the higher effective tax rate in the second quarter of 2019 compared to the year earlier period. The effective tax rate in the second quarter of 2018 benefited from employees exercising stock options.

Weighted-average diluted common shares outstanding were approximately 53.9 million in the second quarter of 2019, as compared to approximately 54.2 million in the prior year period.

Balance Sheet Highlights

As of June 30, 2019, the Company had $852.3 million in cash, cash equivalents, marketable securities and long term marketable securities, compared to $774.8 million at December 31, 2018. This increase primarily reflects cash generated from operations in the first six months of 2019.

Financial Guidance

The Company is revising its full year 2019 guidance for net product sales and operating earnings, and reaffirming expectations for R&D expenses and the effective tax rate as set forth below:

– Net product sales in the range of $400 million to $410 million, compared to the previously expected range of $435 million to $455 million.
– R&D expenses in the range of $70 million to $80 million.
– Operating earnings in the range of $150 million to $160 million, compared to the previously expected range of $160 million to $180 million.
– Effective tax rate of approximately 23% to 25%.

Conference Call Details

The Company will hold a conference call hosted by Jack Khattar, President and Chief Executive Officer, and Greg Patrick, Senior Vice President and Chief Financial Officer, to discuss these results at 9:00 a.m. Eastern Time, on Wednesday, August 7, 2019. An accompanying webcast also will be provided.

Please refer to the information below for conference call dial-in information and webcast registration. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference dial-in: (877) 288-1043
International dial-in: (970) 315-0267
Conference ID: 1527779
Conference Call Name: Supernus Pharmaceuticals Second Quarter 2019 Earnings Conference Call
Following the live call, a replay will be available on the Company’s website, www.supernus.com, under "Investor Relations".

On August 6, 2019 Leap Therapeutics, Inc. (NASDAQ:LPTX) reported that its anti-Dickkopf-1 (DKK1) antibody, DKN-01, in combination with Merck’s anti-PD-1 antibody, KeytrudaÒ (pembrolizumab), demonstrated higher survival and objective response outcomes in patients with advanced gastroesophageal junction and gastric cancer (GEJ/GC) whose tumors expressed high levels of DKK1 (DKK1-high) (Press release, Leap Therapeutics, AUG 6, 2019, View Source [SID1234538168]). DKN-01 plus Keytruda therapy achieved over 22 weeks median progression-free survival (PFS) and nearly 32 weeks median overall survival (OS) with a 50% overall response rate (ORR) and 80% disease control rate (DCR) in patients with DKK1-high GEJ/GC who had not received prior anti-PD-1/PD-L1 therapy. Leap will host a DKN-01 Clinical Perspectives and Program Update for the investment community through a live conference call and webcast with two clinical investigators today at 8:30 AM US Eastern Time.

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"Gastric and gastroesophageal junction cancers represent a major global cancer burden with significant unmet needs, particularly in patients with advanced disease. Outside of rare microsatellite instable tumors and EBV-associated cancers the response rates to immune checkpoint inhibitors are low and median progression free survival remains short, in the range of 6-8 weeks. Oncologists and patients are eager for new therapeutic combinations and biomarkers to help predict patients most likely to benefit from a given treatment," stated Samuel J. Klempner, MD, Assistant Professor, Massachusetts General Hospital Cancer Center and Harvard Medical School.

"The responses and early survival data seen in DKK1-high patients treated with DKN-01 plus pembrolizumab are highly encouraging," commented Dr. Klempner. "This study builds on previously reported positive monotherapy and paclitaxel combination data and importantly suggests that elevated DKK-1 expression is a potential predictive biomarker. DKN-01 warrants further study in gastroesophageal cancers in combination with immune checkpoint inhibitors and with chemotherapy."

Key Findings from KEYNOTE-731 DKN-01 plus Keytruda Combination

The esophagogastric cancer clinical trial is a multipart study of DKN-01 as a monotherapy and in combination with paclitaxel or pembrolizumab. Sixty-three patients were treated with DKN-01 plus Keytruda combination therapy across all arms and dose groups of the study. Fifty-three patients had not received prior PD-1/PD-L1 therapy, and ten patients were refractory to PD-1/PD-L1 therapy. All of the patients enrolled had tumors that were microsatellite stable or unknown. Patients in the study were heavily pretreated having had received one to five prior lines of therapy, with nearly 64% having received a prior taxane regimen, 37% having received prior ramucirumab, and 24% having received prior trastuzumab. The combination therapy was well tolerated with no new safety signals.

The combination of DKN-01 and Keytruda in GEJ/GC patients demonstrated improved outcomes in patients whose tumors are DKK1-high and who were PD-1/PD-L1 naïve. DKK1-high patients experienced

over 22 weeks median PFS and nearly 32 weeks OS, with a 50% ORR and 80% DCR in ten evaluable patients. DKK1-low patients experienced nearly 6 weeks median PFS and over 17 weeks OS, with a 20% DCR in fifteen evaluable patients.

PD-L1 Combined Positive Scores (CPS) did not predict efficacy on the combination of DKN-01 plus Keytruda. In multi-variate analysis, DKK1-high status correlated with longer PFS independent of PD-L1 CPS scores. One-third of patients in the study were DKK1-high.

Among the six GEJ/GC patients who were refractory to PD-1/PD-L1 therapy, three DKK1-high patients had a best response of stable disease, whereas the three patients with DKK1-low tumors had progressive disease.

DKN-01 Clinical Perspectives Conference Call and Webcast

Samuel J. Klempner, MD, Assistant Professor, Massachusetts General Hospital Cancer Center and Harvard Medical School, will describe his experience with treating esophagogastric cancer patients in the DKN-01 study. In addition, Rebecca C. Arend, MD, Assistant Professor and Associate Scientist, Gynecologic Oncology Clinic, UAB Comprehensive Cancer Center Experimental Therapeutics Program, will discuss her experience with endometrial cancer and carcinosarcoma patients treated with DKN-01.

To access the conference call, please dial (866) 589-0108 (US/Canada Toll-Free) or (409) 231-2048 (international) and refer to conference ID 3571417. The presentation will also be webcast live and will be available under "Events & Presentations" in the Investor section of Leap’s website, View Source A replay of the webcast will be available on Leap’s website shortly after the event and will be available for a limited time.

On August 6, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, reports its financial results for the quarter ended June 30, 2019 and provides a corporate update (Press release, BioLineRx, AUG 6, 2019, View Source [SID1234538198]).

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Highlights and achievements during the second quarter 2019 and subsequent period:

Continued to advance multiple clinical trials of its lead therapeutic candidates, BL-8040 and AGI-134, and anticipates top-line data from the triple combination arm of the COMBAT/KEYNOTE-202 pancreatic cancer trial by year-end.

Received approval from the FDA for an Investigational New Drug (IND) application for AGI-134, which will enable expansion of the ongoing Phase 1/2a study, currently being carried out in the UK and Israel, to the US by the first half of 2020.

"BL-8040, our lead drug candidate, is rapidly advancing in multiple promising programs. The triple combination arm of our ongoing COMBAT/KEYNOTE-202 Phase 2 study of BL-8040, KEYTRUDA and chemotherapy in metastatic pancreatic cancer is progressing as planned, and top-line results are expected by year end. AML consolidation also remains an important indication in our BL-8040 development plan, and we, with our partners, continue to progress our BLAST Phase 2b study towards a robust interim analysis. In addition, the GENESIS Phase 3 study in stem cell mobilization, our most advanced indication and most direct path to registration, is progressing as planned with top-line results expected in the second half of next year," stated Philip Serlin, Chief Executive Officer of BioLineRx.

"Regarding our second clinical candidate, the universal anti-cancer vaccine AGI-134, we continue to advance our phase 1/2a clinical trial toward initial safety data later this year, and with the acceptance of our IND, we look forward to the next part of the study, which will assess potential efficacy. Taken together, we believe our broad development pipeline provides multiple opportunities for long-term value creation and we are diligently working toward that goal," Mr. Serlin concluded.

Upcoming Milestones

Second half of 2019

Top-line results from COMBAT/KEYNOTE-202 Phase 2 pancreatic cancer trial

Initial safety results from part 1 of Phase 1/2a trial of AGI-134

Potential interim results from Phase 2b AML consolidation study

Initiation of monotherapy basket arm of Part 2 of Phase 1/2a trial of AGI-134

2020

Progression-free survival (PFS) and overall survival (OS) data from COMBAT/KEYNOTE-202 trial in mid-2020

Top-line results from Phase 3 GENESIS registration trial in stem-cell mobilization in second half of 2020

Financial Results for the Second Quarter Ended June 30, 2019

Research and development expenses for the three months ended June 30, 2019 were $5.3 million, an increase of $0.8 million, or 18%, compared to $4.5 million for the three months ended June 30, 2018. The increase resulted primarily from higher expenses associated with the BL-8040 GENESIS and COMBAT clinical trials. Research and development expenses for the six months ended June 30, 2019 were $9.7 million, an increase of $0.1 million, or 2%, compared to $9.6 million for the six months ended June 30, 2018. The small increase resulted primarily from higher expenses associated with the BL-8040 GENESIS and COMBAT clinical trials, offset by a decrease in expenses related to BL-1230, a project which was terminated, as well as a decrease in payroll and related expenses.

Sales and marketing expenses for the three months ended June 30, 2019 were $0.2 million, a decrease of $0.1 million, or 37%, compared to $0.3 million for the three months ended June 30, 2018. The decrease resulted primarily from a decrease in payroll and related expenses. Sales and marketing expenses for the six months ended June 30, 2019 were $0.5 million, a decrease of $0.4 million, or 43%, compared to $0.9 million for the six months ended June 30, 2018. The decrease resulted primarily from a decrease in payroll and related expenses, including a one-time compensation payment in the 2018 period.

General and administrative expenses for the three months ended June 30, 2019 were $0.9 million, similar to the comparable period in 2018. General and administrative expenses for the six months ended June 30, 2019 were $1.9 million, similar to the comparable period in 2018.

The Company’s operating loss for the three months ended June 30, 2019 was $6.5 million, compared to $5.7 million for the three months ended June 30, 2018. The Company’s operating loss for the six months ended June 30, 2019 was $12.1 million, compared to $12.4 million for the comparable period in 2018.

Non-operating income for the three and six months ended June 30, 2019 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, offset by warrant offering expenses. Non-operating income for the six months ended June 30, 2018 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, as well as a capital gain from realization of the investment in iPharma.

Net financial expenses amounted to $0.3 million for the three months ended June 30, 2019 compared to net financial income of $0.3 million for the three months ended June 30, 2018. Net financial expenses for the 2019 period primarily relate to interest paid on loans, offset by investment income earned on bank deposits. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions. Net financial expenses amounted to $0.5 million for the six months ended June 30, 2019 compared to net financial income of $0.3 million for the six months ended June 30, 2018. Net financial expenses for the 2019 period primarily relate to interest paid on loans, offset by investment income earned on bank deposits. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions.

The Company’s net loss for the three months ended June 30, 2019 amounted to $5.5 million, compared with a net loss of $4.8 million for the comparable period in 2018. The Company’s net loss for the six months ended June 30, 2019 amounted to $11.6 million, compared with a net loss of $11.0 million for the comparable period in 2018.

The Company held $35.2 million in cash, cash equivalents and short-term bank deposits as of June 30, 2019.

Net cash used in operating activities was $11.1 million for the six months ended June 30, 2019, compared with net cash used in operating activities of $13.0 million for the six months ended June 30, 2018. The $1.9 million decrease in net cash used in operating activities during the six-month period in 2019, compared to the six-month period in 2018, was primarily the result of changes in operating asset and liability items in the two periods., i.e., a decrease in prepaid expenses and other receivables in 2019 versus an increase in 2018, as well as a higher decrease in accounts payable and accruals in 2018.

Net cash used in investing activities was $3.1 million for the six months ended June 30, 2019, compared to net cash provided by investing activities of $10.8 million for the six months ended June 30, 2018. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits and the realization of the investment in iPharma in 2018.

Net cash provided by financing activities was $15.7 million for the six months ended June 30, 2019, compared to net cash provided by financing activities of $2.8 million for the six months ended June 30, 2018. The increase in cash flows from financing activities reflects the underwritten public offering completed in February 2019.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, August 6, 2019 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0644 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until August 8, 2019; please dial +1-888-295-2634 from the U.S. or +972-3-925-5904 internationally.

On August 6, 2019 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that engineers next-generation human enzymes to provide solutions for diseases with unmet medical need, reported financial results for the second quarter ended June 30, 2019 and corporate highlights (Press release, Aeglea BioTherapeutics, AUG 6, 2019, View Source [SID1234538214]).

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"We made tremendous progress this quarter with pegzilarginase, our lead product candidate for the treatment of Arginase 1 Deficiency (ARG1-D)," said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief executive officer of Aeglea. "We dosed our first patient in our global pivotal Phase 3 PEACE trial, which is a major milestone toward providing our therapy to patients. The U.S. Food and Drug Administration’s (FDA) breakthrough therapy designation highlights the clinical relevance of the emerging data from the Phase 1/2 and Phase 2 open-label extension clinical trials as well as the potential of pegzilarginase to provide meaningful clinical improvements over available therapy."

Recent Highlights

Aeglea dosed the first patient in the Company’s global pivotal Phase 3 PEACE trial. The pivotal trial is intended to further evaluate the efficacy and safety of pegzilarginase, the Company’s lead product candidate for the treatment of ARG1-D, a progressive disease presenting in early childhood that results in severe complications and early mortality. The Company expects to report topline data from the PEACE trial in the first quarter of 2021.

The FDA granted Breakthrough Therapy Designation (BTD) to the Company’s lead product candidate, pegzilarginase, for the treatment of ARG1-D. The FDA’s BTD is intended to expedite the development and review of new therapies that are aimed at treating a serious or life-threatening condition when preliminary clinical evidence demonstrates the therapy may have substantial improvement on at least one clinically significant endpoint over available therapy. The designation was based on data from the completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study. Aeglea expects to continue discussions with the FDA regarding the pegzilarginase program and the Company’s next steps in the fourth quarter of 2019.

Interim data from 35 patients in the Company’s Phase 1/2 combination trial of pegzilarginase and KEYTRUDA in extensive disease small cell lung cancer revealed to date one complete response, four partial responses and 11 patients with stable disease. The combination trial was well tolerated, and safety observations were consistent with prior studies of pegzilarginase in patients with cancer. The Company has concluded enrollment and intends on submitting the results for presentation or publication after the final dataset becomes available.
Upcoming Events

Aeglea will present at the following conferences, with details regarding the date and time of the presentations and webcasts to be announced prior to the events.

Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, September 3-6, Rotterdam, Netherlands
H.C. Wainwright & Co. 21st Annual Global Investment Conference, September 8-10, New York, NY
Second Quarter 2019 Financial Results

As of June 30, 2019, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $107.0 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $14.8 million for the second quarter of 2019, compared with $9.1 million for the second quarter of 2018. The increase was primarily due to expanded personnel-related expenses, clinical development activity, investment in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, and a ramp-up in manufacturing activities for the Company’s AEB4104 program for homocystinuria.

General and administrative expenses totaled $3.8 million for the second quarter of 2019, compared with $2.9 million for the second quarter of 2018. This increase was primarily due to additional employee headcount and compensation to support company growth.

Net loss totaled $18.0 million and $9.4 million for the second quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.2 million and $1.0 million for the second quarter of 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. Aeglea’s Phase 1/2 and Phase 2 open-label extension data evaluating pegzilarginase in patients with Arginase 1 Deficiency demonstrated clinical improvements and sustained lowering of plasma arginine. Aeglea is currently recruiting patients for its single, global pivotal Phase 3 PEACE trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.