On August 13, 2019 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines, reported second quarter 2019 financial results and provided an overview of operational progress (Press release, Rubius Therapeutics, AUG 13, 2019, View Source [SID1234538800]).

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"During the second quarter, we continued to advance our promising pipeline of Red Cell Therapeutics for the potential treatment of rare diseases, cancer and autoimmune diseases," said Pablo J. Cagnoni, M.D., chief executive officer of Rubius Therapeutics. "In addition, we have been working closely with our contract manufacturer to improve its ability to consistently produce product in 200-liter bioreactors. We expect to begin enrolling patients in the near term and remain on track to deliver initial clinical data by year-end. Finally, we continue to make great progress on the buildout of our new manufacturing facility in Smithfield, RI to provide GMP manufacturing for clinical supply."

Second Quarter Highlights and Upcoming Milestones

·Rubius is working closely with its contract manufacturer to improve its ability to consistently produce product in 200-liter bioreactors in order for Rubius to begin enrolling patients in its Phase 1b clinical trial for patients with phenylketonuria (PKU) in the near term.

·Rubius plans to report initial clinical data from the first patients enrolled in the trial by year-end, including:

·Preliminary safety;

·Longevity of RTX-134 cells in circulation; and

· Proof-of-mechanism as measured by production of trans-cinnamic acid, the metabolite of phenylalanine when degraded by phenylalanine ammonia lyase, or PAL.

·The Company continued to strengthen its leadership by appointing Kris Elverum as senior vice president of business development and strategy to oversee corporate strategy and create and execute potential new business development opportunities.

·On June 5, 2019, Rubius announced the issuance of three patents from the U.S. Patent and Trademark Office related to its rare disease and oncology programs and platform.

·Two of the issued patents cover composition of matter and methods of use for the Company’s lead investigational cellular therapy, RTX-134, for the treatment of patients with PKU.

·The third patent, the Company’s first issued composition of matter patent in immuno-oncology, relates to human cells having a checkpoint inhibitor linked to the cell surface.

·Today, in total, Rubius Therapeutics has 30 patent families and more than 130 pending patent applications worldwide.

Second Quarter Financial Results

Net loss for the second quarter of 2019 was $39.4 million or $0.50 per common share, compared to $21.2 million or $2.43 per common share in the second quarter of 2018.

In the second quarter of 2019, Rubius invested $27.5 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $11.4 million in the second quarter of 2018. This year-over-year increase was driven primarily by $7.6 million in incremental R&D program spending in preparation for the Company’s Phase 1b clinical trial for RTX-134 and towards preclinical activities for Rubius’ lead oncology programs, including RTX-240. In addition, $7.4 million in incremental R&D spending was driven by increased R&D headcount, including technical operations, a move into larger facilities and purchasing lab supplies to support Rubius’ goal of delivering four to five INDs across 2019 and 2020. R&D stock-based compensation also increased by $1.9 million.

G&A expenses were $13.8 million during the second quarter of 2019, as compared to $9.0 million for the second quarter of 2018. The higher costs were primarily driven by a $1.5 million increase in personnel and facility costs due to increased headcount in the general and administrative function, as well as increases in professional fees and infrastructure costs to support the Company’s growth. G&A stock-based compensation also increased by $2.8 million.

Six Month Financial Results

Net loss for the first six months of 2019 was $72.0 million or $0.92 per common share, compared to $35.7 million or $4.17 per common share in the first six months of 2018.

In the six months ended June 30, 2019, Rubius invested $48.4 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $20.9 million in the first six months of 2018. This year-over-year increase was largely due to an additional $14.9 million in R&D personnel, including technical operations, facilities and lab supplies to support the Company’s pipeline expansion in 2019 and 2020 and $11.2 million in R&D program spending, including preparing for the RTX-134 Phase 1b clinical trial and preclinical activities for Rubius’ lead oncology programs, including RTX-240. R&D stock-based compensation also increased by $3.6 million.

G&A expenses were $27.3 million during the first six months of 2019, as compared to $14.1 million for the same period in 2018. The higher costs were primarily driven by a $7.9 million increase in stock-based compensation and a $3.7 million increase in personnel costs and facility costs due to increased headcount in our general and administrative function, as well as increases in professional fees and infrastructure costs to support the Company’s growth.

Cash Position

As of June 30, 2019, cash, cash equivalents and investments were $362.3 million as compared to $404.1 million as of December 31, 2018, providing Rubius with a cash runway into 2021. During the year, the Company used $52.7 million of cash to fund operations and $17.4 million to fund capital expenditures, including work related to the buildout of Rubius’ manufacturing facility. In addition, during the quarter, the Company drew down a second tranche of $25.0 million from its $75.0 million loan agreement with Solar Capital, which leaves a third tranche of $25.0 million that can be drawn through June 2020, subject to the satisfaction of certain financial covenants.

On August 13, 2019 Myriad Genetics, Inc. (NASDAQ: MYGN, "Myriad" or the "Company"), a global leader in molecular diagnostics and precision medicine, reported financial results for its fiscal fourth-quarter and full-year 2019, reported an update on recent business highlights and provided fiscal year and first-quarter 2020 financial guidance (Press release, Myriad Genetics, AUG 13, 2019, View Source [SID1234538763]).

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"Fiscal year 2019 revenue increased 14 percent with earnings up 18 percent. Unfortunately, revenue in the fourth quarter was two percent below expectations largely due to lower reimbursement for our expanded carrier screening test," said Mark C. Capone, president and CEO, Myriad Genetics. "Looking ahead to fiscal year 2020, with stabilized pricing, growing new product volumes and recent reimbursement advances with GeneSight, we are highly optimistic about our ability to deliver an inflection in revenue and earnings as we transition through the fiscal year."

Recent Business Highlights

Hereditary Cancer

Hereditary cancer revenue returned to year-over-year growth in fiscal year 2019 representing the first time hereditary cancer revenue has grown in the last five fiscal years.

Presented data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting from the Women’s Health Initiative study that evaluated mutation rates in 2,195 post-menopausal women with breast cancer. The study found that women with post-menopausal breast cancer had a high rate of inherited mutations in a range of cancer causing genes and this mutation rate did not diminish with age. This along with other studies have shown that

current hereditary breast cancer testing guidelines miss approximately 50 percent of mutation carriers.

GeneSight

Announced coverage decision from UnitedHealth, the largest commercial payer in the United States, covering GeneSight for patients that have a diagnosis of major depressive disorder or anxiety and have failed at least one prior medication.

Announced coverage decision for GeneSight by Kroger Prescription Plans. As part of the agreement, Kroger initiated an early access program at 500 Kroger pharmacies where GeneSight testing will be facilitated by the Kroger Health pharmacist.

Presented data at the Clinical Pharmacogenomics Implementation Consortium annual meeting showing GeneSight had more than double the predictive power for drug blood levels compared to single gene pharmacogenomics tests.

Completed a Medicare Coverage Advisory Committee meeting reviewing potential expansion of the GeneSight LCD to primary care physicians. During the meeting, Medicare’s selected subject matter experts unanimously agreed that pharmacogenomics testing should be available to primary care physicians.

Prolaris

Published data from a large study of 1,062 men with newly diagnosed localized prostate cancer to evaluate the ability of the Prolaris test to predict risk of metastases. The study found that Prolaris was the strongest independent predictor of progression to metastatic disease, and men were approximately three times more likely to develop metastatic disease with each unit increase in the Prolaris test score (HR: 2.93; p=1.8×10-11).

EndoPredict

Published data in the journal Clinical Cancer Research demonstrating that the EndoPredict test identifies women with early-stage breast cancer who can safely forgo extended endocrine therapy five years after diagnosis.

Published the results of the first comprehensive cost-effectiveness analysis of the EndoPredict test compared to other breast cancer assays. The study found that the EndoPredict test was more than twice as cost effective as Oncotype DX.

Companion Diagnostics

Announced that the BRACAnalysis CDx companion diagnostic test effectively identified patients with metastatic pancreatic cancer who benefitted from treatment with Lynparza

(olaparib) in the Phase III POLO study. Patients in the study with a germline mutation had a clinically-meaningful and statistically-significant improvement in progression-free survival (PFS) of 7.4 months when treated with Lynparza compared to 3.8 months for placebo (HR 0.53; p=0.004).

Received approval from the Japanese Ministry of Health, Labour, and Welfare for Myriad’s BRACAnalysis Diagnostic System as a companion for Lynparza in women with ovarian cancer.

Announced that BRACAnalysis CDx effectively identified patients with castrate resistant, metastatic prostate cancer who benefitted from treatment with Lynparza in the PROfound study. Results from the study showed a statistically-significant and clinically-meaningful improvement in radiographic progression-free survival in patients who received Lynparza vs. enzalutamide or abiraterone and had deleterious mutations in the BRCA1/2 genes.

myPath Melanoma

Received a positive final local coverage decision from Noridian Healthcare Solutions for myPath Melanoma.

German Clinic Sale

Announced planned sale of the Privatklinik Dr. Robert Schindlbeck GmbH & Co. KG (the "Clinic"), which is expected to occur around the middle of fiscal year 2020.

Fiscal Year 2020 and Fiscal First-Quarter 2020 Financial Guidance

Myriad’s fiscal year 2020 and first-quarter 2020 adjusted earnings per share guidance excludes the impact of stock based compensation expense, non-cash amortization associated with acquisitions and certain non-recurring expenses. These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The company will provide further details on its business outlook during the conference call today and discuss the fiscal fourth-quarter financial results and fiscal year 2020 financial guidance.

Conference Call and Webcast

A conference call will be held today, Tuesday, August 13, 2019, at 4:30 p.m. EDT to discuss Myriad’s financial results for the fiscal fourth-quarter, business developments and financial guidance. The dial-in number for domestic callers is 1-800-763-5615. International callers may dial 1-212-231-2936. All callers will be asked to reference reservation number 21927089. An archived replay of the call will be available for seven days by dialing (800) 633-8284 and entering the reservation number above. The conference call along with a slide presentation will also will be available through a live webcast at www.myriad.com.

On August 13, 2019 Atreca, Inc. (Atreca) (NASDAQ: BCEL), a biotechnology company focused on developing novel therapeutics based on a deep understanding of the human immune response, reported financial results for the second quarter ended June 30, 2019, and provided an overview of Atreca’s recent developments (Press release, Atreca, AUG 13, 2019, View Source [SID1234538759]).

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"The second quarter of 2019 was a highly productive period for Atreca, highlighted by the completion of our successful initial public offering, which further strengthens our ability to execute on the promise of our differentiated technology platform drawn from a deep understanding of the human immune response," said John Orwin, Chief Executive Officer. "We believe our lead product candidate, ATRC-101, represents an exciting and novel approach to cancer therapy with the potential to drive better outcomes for patients across a variety of solid tumors. We look forward to submitting the Investigational New Drug (IND) application for ATRC-101 later this year and initiating a Phase 1b clinical trial in patients with solid tumors in early 2020."

Recent Developments and Highlights

·In June 2019, Atreca successfully completed its initial public offering, raising $130.8 million in net proceeds.

·In July 2019, Atreca further strengthened its management team with the appointments of Lisa L. Decker, Ph.D., as Chief Business Officer and Courtney J. Phillips as General Counsel.

Upcoming Milestones

·Atreca anticipates filing an IND application with the U.S. Food and Drug Administration for ATRC-101 in late 2019 and initiating a Phase 1b clinical trial in patients with solid tumors in early 2020.

Second Quarter 2019 Financial Results

·As of June 30, 2019, cash and cash equivalents and short-term investments totaled $219.7 million. This includes net proceeds of approximately $130.8 million from Atreca’s June 2019 initial public offering of common stock, after deducting the underwriting discounts and commissions and expenses of the offering.

·Research and development expenses for the three months ended June 30, 2019 were $15.9 million, including non-cash share-based compensation expense of $752,000.

·General and administrative expenses for the three months ended June 30, 2019 were $3.5 million, including non-cash share-based compensation expense of $607,000.

·Atreca reported a net loss of $17.9 million, or basic and diluted net loss per share attributable to common stockholders of $3.67, for the three months ended June 30, 2019.

On August 13, 2019 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) reported its financial and operating results for the second quarter ended June 30, 2019 (Press release, AEterna Zentaris, AUG 13, 2019, View Source [SID1234538757]).

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All Amounts are in U.S. Dollars

Highlights

On June 6, 2019, the Company announced that it was reducing the size of its German workforce and operations to more closely reflect the Company’s ongoing commercial activities in Frankfurt. This restructuring affects 8 employees in Frankfurt, Germany and resulted in $773,000 of severance costs that are expected to be paid by January 31, 2020.

Net income for the second quarter of 2019 was $0.2 million as compared to a net loss of $2.6 million for the same period in 2018. Net loss for the six-months ended June 30, 2019 was $4.7 million as compared to net income of $11.8 million for the same period in 2018.

In the first six months of 2019, the Company earned $21,000 from the license of Macrilen (macimorelin) as gross sales in the U.S. continue to disappoint. Novo Nordisk has advised that Macrilen (macimorelin) has been successfully integrated into their patient and provider support hub as they work to gain broader product coverage with payers and specialty pharmacies. We continue to work with Novo Nordisk on addressing the disappointing U.S. commercial execution and results to date.

The initial phase of the macimorelin pediatric development program (the dose ranging study), continued to progress with the first one-third of subjects having completed the protocol, all in accordance with our expected timeline.
Summary of Second Quarter Results and Year-to-date Second Quarter Results

For the three-month period ended June 30, 2019, we reported a consolidated net income of $0.2 million, or $0.01 income per common share (basic), as compared with a consolidated net loss of $2.6 million, or $0.16 loss per common share, for the three-month period ended June 30, 2018. The $2.8 million improvement in net results is primarily from a gain in fair value of warrant liability of $4.1 million, offset by a loss in exchange rates of $0.7 million, an increase in operating expenses of $0.3 million and $0.2 million movement in tax recovery.

For the six-month period ended June 30, 2019, we reported a consolidated net loss of $4.7 million, or $0.28 loss per common share, as compared with a consolidated net income of $11.8 million, or $0.72 income per common share (basic), for the six-month period ended June 30, 2018. The $16.5 million decline in net results is primarily from a reduction of $24.5 million in gross income and $0.6 million decrease in net finance income, offset by a reduction of tax expense of $6.6 million and operating expenses of $1.9 million.

Condensed Interim Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the condensed interim consolidated financial statements as at June 30, 2019 and for the three-month and six-month periods ending June 30, 2019 and 2018 and management’s discussion and analysis of financial condition and results of operations for the three-month and six-month periods ending June 30, 2019, will be found at www.zentaris.com in the "Investors" section and at the Company’s profile at www.sedar.com and www.sec.gov.

The tables below set out summary consolidated financial information for the periods indicated. These tables should be read together with the condensed interim consolidated financial statements as at June 30, 2019 and for the three-month and six-month periods ending June 30, 2019 and 2018 and management’s discussion and analysis of financial condition and results of operations for the three-month and six-month periods ending June 30, 2019, which contain important information relating to the Company. The results of operations for interim periods are not necessarily indicative of the results to be expected for a full year or any future period. The information presented herein does not contain disclosures required by IFRS for consolidated financial statements and should be read in conjunction with the Company’s audited annual consolidated financial statements for the year ended December 31, 2018.

On August 13, 2019 KemPharm, Inc. (Nasdaq: KMPH), a specialty pharmaceutical company engaged in the discovery and development of proprietary prodrugs, reported its corporate and financial results for the quarter ended June 30, 2019 (Press release, KemPharm, AUG 13, 2019, View Source [SID1234538734]).

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"Our highest priority at KemPharm has been securing a licensing agreement that maximizes the value of KP415 and KP484. This licensing process has taken longer than expected as we have had several late entrants. Yet, we believe we are in the final phase of this process and remain optimistic about reaching a successful conclusion as soon as possible," said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "Looking ahead, once this partnership is finalized, we will focus our full attention in conjunction with our partner to finalize the NDA for KP415, which we believe could likely be filed prior to year-end."

"Additionally, we continue to work diligently with KVK Tech to prepare for the commercial launch of APADAZ, which remains on track for the second half of 2019," added Dr. Mickle. "Together with KVK, both the commercial outreach and manufacturing processes are moving forward in parallel. We look forward to reporting our progress in the coming months."

Q2 2019 Financial Results:

For the quarter ended June 30, 2019, KemPharm’s reported net loss was $9.3 million, or $0.33 per basic and diluted share, compared to a net loss of $10.0 million, or $0.65 per basic share and $0.91 per diluted share for the same period in 2018. Net loss for Q2 2019 was driven primarily by a loss from operations of $7.8 million, and net interest expense and other items of $1.5 million. The loss from operations for Q2 2019 decreased by $6.1 million compared to a loss from operations of $13.9 million in Q2 2018, which was primarily due to decreases of $5.7 million in research and development expenses and $0.4 million in general and administrative expenses, respectively.

As of June 30, 2019, total cash, which is comprised of cash, cash equivalents and restricted cash, was $7.8 million, which was a decrease of $6.2 million as compared to March 31, 2019.

"During the quarter, we took a number of steps to reduce our operating, general and administrative expenses, including an approximate 30% reduction in workforce compared to the end of Q1 2019, with the goal of reducing our cash burn rate while maintaining our research and development capabilities to support our ongoing work with KP415 and KP484, as well as remaining positioned to support the ultimate commercial partner once that process has been completed. Additionally, as the NDA filing approaches, we expect to see the expenditures related to KP415 R&D continue to reduce substantially," concluded Dr. Mickle.