On November 7, 2019 VistaGen Therapeutics (NASDAQ: VTGN), a clinical-stage biopharmaceutical company developing new generation medicines for central nervous system (CNS) diseases and disorders with high unmet need, reported financial results for its fiscal year 2020 second quarter ended September 30, 2019 (Press release, VistaGen Therapeutics, NOV 7, 2019, View Source [SID1234550704]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During the quarter, we achieved several important milestones intended to advance development of each of our three differentiated CNS drug candidates," stated Shawn Singh, Chief Executive Officer of VistaGen. "We recently achieved target enrollment and completed patient dosing in our randomized, double-blind, placebo-controlled Phase 2 ELEVATE study of AV-101, our novel oral NMDA receptor antagonist, in major depressive disorder. In addition, for development of AV-101 in suicidal ideation, with emphasis on U.S. Military Veterans, our collaborators at Baylor University recently completed dosing in their Phase 1b target engagement study in healthy volunteer Veterans, a study funded by the U.S. Department of Veteran’s Affairs. We are also encouraged by post-Phase 2 feedback from the FDA earlier this year regarding our Phase 3 development plan for PH94B, our first-in-class, rapid-acting neuroactive nasal spray, in social anxiety disorder. We expect the coming months to be equally active and potentially transformative, as we look forward to topline readouts from two studies involving AV-101 before the end of 2019, and potential regulatory milestones involving PH94B before the end of fiscal 2020."

Financial Results for the Fiscal Quarter Ended September 30, 2019:

Net loss attributable to common stockholders for the fiscal quarter ended September 30, 2019 decreased to approximately $5.7 million compared to $7.7 million for the fiscal quarter ended September 30, 2018, primarily attributable to research and development activities relating to the Company’s CNS drug development programs.

Research and development expense decreased to $4.2 million for the fiscal quarter ended September 30, 2019, compared with $5.3 million for the fiscal quarter ended September 30, 2018. Expense for the quarter ended September 30, 2018 included $2.25 million noncash expense associated with the Company’s acquisition of its exclusive worldwide license to develop and commercialize PH94B and an option to acquire an exclusive worldwide license to develop and commercialize PH10, the Company’s first-in-class, rapid-onset neuroactive nasal spray in Phase 2 development for major depressive disorder, which option was subsequently exercised. Offsetting this noncash expense in the quarter ended September 30, 2019 are increased expenses for the ELEVATE study and various nonclinical activities across the Company’s CNS pipeline.

General and administrative expense decreased to approximately $1.1 million in the fiscal quarter ended September 30, 2019, compared to approximately $2.2 million in the fiscal quarter ended September 30, 2018. Noncash expense of $272,000 in the quarter ended September 30, 2019, decreased from $792,000 in the quarter ended September 30, 2018, primarily due to decreases in stock-based compensation and other expenses.

At September 30, 2019, VistaGen had cash and cash equivalents of $4.1 million, compared to $13.1 million at March 31, 2019.

As of November 6, 2019, there were 43,222,965 shares of common stock outstanding.

VistaGen’s CNS Pipeline

VistaGen is developing three new generation clinical-stage CNS drug candidates, AV-101, PH10 and PH94B, each with a differentiated mechanism of action, an exceptional safety profile in all clinical studies to date, and therapeutic potential in multiple CNS markets where current treatments are inadequate to meet high unmet patient needs.

AV-101 belongs to a new generation of investigational medicines in neuropsychiatry and neurology known as NMDA (N-methyl-D-aspartate) receptor modulators. The NMDA receptor is a pivotal receptor in the brain and abnormal NMDA function is associated with numerous CNS diseases and disorders. AV-101 is an oral prodrug of 7-Cl-KYNA, a potent and selective full antagonist of the glycine co-agonist site of the NMDA receptor. Based on several positive preclinical studies and its exceptional safety profile in all preclinical and clinical studies to date, AV-101 has potential to be a new at-home, non-sedating treatment for multiple large market CNS indications, including major depressive disorder, neuropathic pain, suicidal ideation, epilepsy and dyskinesia associated with levodopa therapy for Parkinson’s disease. The FDA has granted Fast Track designation for development of AV-101 as both a novel potential adjunctive treatment for MDD and a non-opioid treatment for neuropathic pain.

PH10 is a first-in-class, odorless, rapid-onset CNS neuroactive nasal spray in development for treatment of major depressive disorder. Administered in microgram doses, PH10 activates nasal chemosensory receptors that, in turn, engage neural circuits that lead to rapid antidepressant effects without psychological side effects, systemic exposure or safety concerns often associated with current oral antidepressants and ketamine-based therapies (intravenous ketamine or esketamine nasal spray). In an exploratory (n=30) randomized, double-blind, placebo-controlled Phase 2a clinical study in major depressive disorder, at microgram doses, rapid-onset antidepressant effects were observed and sustained for 8 weeks, without psychological side effects or systemic exposure. VistaGen is preparing for planned Phase 2b clinical development of PH10 for major depressive disorder.

PH94B is a first-in-class, odorless, rapid-onset (approximately 10 to 15 minutes) CNS neuroactive nasal spray with the potential to be the first FDA-approved, as-needed, on-demand treatment for millions of Americans who suffer from social anxiety disorder, with additional potential in peripartum anxiety, pre/postoperative anxiety, post-traumatic stress disorder, panic disorder and generalized anxiety disorder. Administered at microgram doses, PH94B activates nasal chemosensory receptors that trigger neural circuits in the brain that suppress fear and anxiety associated with everyday social and work or performance situations. Following successful Phase 2 development, VistaGen is preparing for Phase 3 clinical development of PH94B for social anxiety disorder.

On November 7, 2019 Athenex, Inc. (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported its financial results and business highlights for the third quarter of 2019 (Press release, Athenex, NOV 7, 2019, View Source [SID1234550720]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to make strong progress across the board, bringing us closer to our goal of becoming a fully integrated global pharma company," stated Dr. Johnson Lau, Chief Executive Officer and Chairman of Athenex. "We have several major catalysts upcoming, including two anticipated NDA submissions. We are also scheduled to deliver an oral presentation to discuss the Phase III data for Oral Paclitaxel in metastatic breast cancer at the San Antonio Breast Cancer Symposium in December. We have been strategic in building out and integrating our clinical and manufacturing operations in order to maximize the commercial opportunities of our rapidly advancing pipeline. We believe we are in a strong position to develop multiple, potentially successful therapies in the future."

Mr. Jeffrey Yordon, Chief Operating Officer of Athenex, commented, "We are continually optimizing our commercial infrastructure, developing the market, and building awareness of our Athenex Oncology brand in anticipation of commercial launch of Oral Paclitaxel. We have finalized our staffing and organizational plan, and intend to make additional key hires next year, including medical science liaisons and regional sales leaders, with the full sales team expected to come on board in the months leading up to potential approval. In addition to late-stage pipeline progress, we once again achieved strong revenue growth for our existing commercial business. We plan to continue launching additional products in the remainder of 2019 and into next year."

Third Quarter 2019 and Recent Business Highlights:

Clinical Programs:

Phase III Study of Oral Paclitaxel and Encequidar for Metastatic Breast Cancer

Study met primary endpoint showing statistically significant improvement in overall response rate for oral paclitaxel and encequidar ("Oral Paclitaxel") compared to IV paclitaxel.

Strong trend in progression-free survival (PFS) and overall survival (OS) of Oral Paclitaxel compared to IV paclitaxel.

Proportion of confirmed responders with duration of response >150 days was 2.5 times higher for Oral Paclitaxel than IV paclitaxel.

Neuropathy was less frequent with Oral Paclitaxel compared to IV paclitaxel.

Planning to meet with the FDA and present data at SABCS.

Other Oral Paclitaxel Developments

European Commission granted orphan designations for paclitaxel and for encequidar for the treatment of soft tissue sarcoma.

Presented three posters at European Society for Medical Oncology Congress 2019 demonstrating the results from clinical studies of Oral Paclitaxel for patients with a number of advanced solid tumor types as well as in combination with ramucirumab, an anti-VEGFR2 antibody therapy.

Tirbanibulin Ointment for Actinic Keratosis (AK)

Partner Almirall announced a progress update on the program, including AK recurrence rates in those patients who had complete clearance at the primary evaluation endpoint on day 57 and who were followed quarterly in the 12-month extension period.

Athenex completed pre-NDA consultation with the FDA.

Partner Almirall reiterated its expectations for launch of tirbanibulin ointment in the US and Europe in Q1 2021 and Q2 2021, respectively.

Phase I Clinical Study of KX2-361 Oral

Partner Xiangxue Pharmaceutical initiated a Phase I study in China. KX2-361 is the second compound derived from Athenex’s Src kinase inhibition platform, for the treatment of glioblastoma multiforme.

Corporate Announcements:

Appointed Daniel Lang, MD, as President of Axis Therapeutics Limited (Axis) and Senior Director of Corporate Development at Athenex.

Completed construction of new API (active pharmaceutical ingredients) facility in Chongqing, China.

The 440,000-square-foot facility is expected to commence operations in the first half of 2020.

The construction of the facility is part of Athenex’s strategy for vertical integration to capture value across the supply chain.

Commercial Business:

Athenex Pharmaceutical Division (APD) currently markets a total of 31 products with 59 SKUs.

Athenex Pharma Solutions (APS) currently markets 5 products with 13 SKUs.

Goal is to launch 3-5 products in the remainder of 2019.

Financial Results for the Quarter Ended September 30, 2019

Product sales for the three months ended September 30, 2019 were $19.2 million, compared with $13.3 million for the three months ended September 30, 2018, an increase of $5.9 million or 45%. This increase was primarily attributable to an increase in specialty product revenue and 503B revenue of $4.7 million and $2.5 million, respectively. The licensing fees and consulting revenue recorded in the three months ended September 30, 2018 primarily related to our tirbanibulin license agreement with Almirall.

Cost of sales for the three months ended September 30, 2019 totaled $17.1 million, an increase of $5.1 million, or 43%, as compared to $12.0 million for the three months ended September 30, 2018. The increase in cost of sales was in line with the increase in product sales.

Research and development expenses for the three months ended September 30, 2019 were $19.6 million as compared to $51.2 million for the three months ended September 30, 2018. This was primarily due to a decrease in licensing fees, product development, clinical operations, and R&D related compensation. The licensing fee decrease mainly resulted from a $29.5 million non-cash license fee related to the license of TCR-T technology in connection with the establishment of Axis, recorded in the third quarter of 2018 and which did not recur. The decrease in R&D expenses was offset primarily by an increase in preclinical development costs related to the Arginine Deprivation Therapy and TCR-T Immunotherapy platforms.

Selling, general and administrative expenses for the three months ended September 30, 2019 totaled $16.3 million, compared to $11.5 million for the three months ended September 30, 2018. This was primarily due to an increase in costs of preparing to commercialize our proprietary drugs, if approved, and an increase in general administrative expenses including legal fees and other professional service fees. Administrative-related compensation expense remained consistent with the prior year.

Net loss attributable to Athenex for the three months ended September 30, 2019 was $34.8 million, or ($0.45) per diluted share, compared to a net loss of $46.2 million, or ($0.70) per diluted share, in the same period last year.

The Company received a $20 million milestone payment from Almirall during the second quarter of 2019 in connection with the partnership on tirbanibulin and expects this payment to be recorded as revenue in the fourth quarter of 2019.

At September 30, 2019, the Company had cash, cash equivalents, restricted cash and short-term investments of $129.2 million, compared to $107.4 million at December 31, 2018. Based on the current operating plan, we expect that our cash, cash equivalents, and restricted cash as of September 30, 2019, together with cash to be generated from our operating activities, will enable us to fund our operations into the third quarter of 2020.

Financial Results for the Nine Months Ended September 30, 2019

Product sales increased to $66.4 million for the nine months ended September 30, 2019, from $37.4 million for the nine months ended September 30, 2018.

Total revenue for the nine months ended September 30, 2019 decreased by $1.0 million, to $66.9 million, as compared to $67.8 million for the nine months ended September 30, 2018. The decrease was primarily due to $30.0 million related to license milestone revenue earned during 2018, and $2.3 million decrease in medical device product sales and contract manufacturing revenue, offset by a $15.2 million increase in specialty product sales, a $13.4 million increase in 503B sales, and a $2.7 million increase in sales of API. Revenue from 503B and API sales is expected to decline for the remainder of the year as we ceased sales of vasopressin in August 2019 and suspended production of API in the second quarter of 2019.

Cost of sales for the nine months ended September 30, 2019 totaled $53.9 million, an increase of $21.2 million, or 65%, as compared to $32.7 million for the nine months ended September 30, 2018. This was primarily due to the increase of $16.9 million in cost of sales from the sale of specialty products and $4.3 million in cost of sales from 503B and API products. The increase in cost of sales was lower than that in product sales, primarily as a result of changes in our product portfolio.

Research and development expenses for the nine months ended September 30, 2019 totaled $62.6 million, as compared to $99.1 million for the nine months ended September 30, 2018. This was primarily due to a decrease in licensing fees, as well as expenses in relation to clinical operations and product development, partially offset by an increase in preclinical development costs related to the Arginine Deprivation Therapy and TCR-T Immunotherapy platforms, and an increase of R&D related compensation expense.

Selling, general and administrative expenses for the nine months ended September 30, 2019 totaled $48.6 million, as compared to $37.4 million for the nine months ended September 30, 2018. This was primarily due to an increase related to the costs of preparing to commercialize our proprietary drugs, if approved, and an increase in general administrative expenses including legal fees and other professional service fees, partially offset by a decrease of in administrative related compensation expense.

Net loss attributable to Athenex for the nine months ended September 30, 2019 was $102.0 million, or ($1.41) per diluted share, compared to a net loss of $90.3 million, or ($1.42) per diluted share, in the same period last year.

Outlook and Upcoming Milestones:

Oral presentation of Phase III results for Oral Paclitaxel at the San Antonio Breast Cancer Symposium (December 13, 2019)

Expect to submit an NDA for tirbanibulin ointment in actinic keratosis (Q1 2020)

Expect to submit an NDA for Oral Paclitaxel in metastatic breast cancer (Q1 2020)

Raising Financial Guidance:

Athenex provides revenue guidance for product sales only. The Company is raising its product sales guidance for the full year 2019 to an increase of 35% to 40% year-over-year from $56.4 million in 2018, versus prior guidance of 30% to 35% year-over-year. This new revenue guidance has taken into account our discontinuation of vasopressin sales and the suspension of operations at our Taihao API plant. The revenue guidance excludes license and collaboration fees.

Conference Call and Webcast Information:

The Company will host a conference call and live audio webcast today, Thursday, November 7, 2019, at 8:00am Eastern Time to discuss the financial results and provide a business update.

To participate in the call, dial 877-407-0784 (domestic) or 201-689-8560 (international) fifteen minutes before the conference call begins and reference the conference passcode 13694941. The live conference call and replay can also be accessed via audio webcast here and on the Investor Relations section of the Company’s website, located at View Source

On November 7, 2019 Cryoport, Inc. (NASDAQ: CYRX) (NASDAQ: CYRXW) ("Cryoport"), the world’s leading temperature-controlled logistics company dedicated to the life sciences industry, reported financial results for the three and nine-month periods ended September 30, 2019 (Press release, Cryoport, NOV 7, 2019, View Source [SID1234550785]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Cryoport reported record revenue of $9.6 million for the three-month period ended September 30, 2019, an increase of 81% compared with the same period in the prior year," stated Jerrell Shelton, Chief Executive Officer of Cryoport. "This was primarily driven by record revenue from our commercial agreements supporting Gilead’s YESCARTA and Novartis’ KYMRIAH which contributed $2.6 million in the Third Quarter of 2019, representing an increase of $2.0 million compared with the Third Quarter of 2018. Notably, this also represents a 39% sequential quarterly increase in revenue over the Second Quarter of 2019. We expect revenue from our commercial agreements to continue to grow and accelerate as the rollouts of these lifesaving therapies continue. We also anticipate that this growth will be supplemented with bluebird bio’s anticipated commercial launch of ZYNTEGLO in in early 2020 and followed by others later in the year.

"Our growth in the Third Quarter was also attributable to Bioservices revenue of $1.2 million from our Houston based operation, Cryogene. We have begun to leverage Cryogene with cross-selling opportunities that we believe will drive revenue growth and provide more comprehensive supply chain solutions to existing and prospective clients.

"Our global footprint continues to mature as our Global Logistics Centre in Amsterdam experienced higher revenue driven by strong regional demand for reliable, integrated manufacturing and distribution solutions during the quarter. To further expand our Global Supply Chain Network, we are building partnerships with multinational biopharmaceutical companies and biopharmaceutical support companies in order to advance our mission to provide cell and gene therapy developers with fully integrated supply chain solutions.

"We recently signed an agreement with, Swiss based, Lonza, one of the world’s leading and most-trusted integrated solutions providers to the pharmaceutical, biotech, and consumer health markets. This agreement makes Cryoport a Lonza preferred partner in the transport and delivery of patient materials on a global basis. Lonza has twenty-four major sites located throughout the world, including four centers of excellence in cell and gene therapy. Its flagship dedicated cell and gene therapy manufacturing facility is located in Houston, Texas. This state-of-the-art manufacturing facility produces treatments for thousands of patients suffering from rare genetic disorders or life-threatening diseases. It is the intent of Lonza and Cryoport that through their new partnership, they will drive supply chain and manufacturing efficiencies in the cell and gene therapy manufacturing processes by integrating Cryoport’s logistics and bioservices solutions with Lonza’s manufacturing services and expertise. The Lonza-Cryoport partnership is expected to provide a best in class solution for the outsourced manufacturing and logistics of cell and gene therapies.

"We also partnered with Vineti to pair their supply chain orchestration (SCO) platform with our integrated temperature-controlled supply chain capabilities and near real-time monitoring. This partnership provides end-to-end solutions for advanced therapies, supporting improved product quality and patient safety.

"Other recent announcements include a three-year agreement with Adaptimmune Therapeutics plc, to optimize Adaptimmune’s manufacturing and supply operations and reduce "vein-to-vein" time for patients in clinical trials. Forming valuable industry partnerships such as these further integrates Cryoport into the market at a time when biopharmaceutical companies are discovering new ways to harness regenerative therapies and combat illnesses.

"During the Third Quarter, we added a net total of 12 clinical trials, bringing the grand total of regenerative therapy clinical trials supported by Cryoport to a record 425, of which 54 are currently in Phase III. As more and more of these therapies approach commercialization, demand for reliable, integrated outsourced manufacturing and distribution solutions continues to increase. As the clear market leader, Cryoport is poised to capture additional market share and expand as the market grows.

"Data provided by the Alliance for Regenerative Medicine states that there are five anticipated BLA or MAA submissions in the remainder of 2019 or early 2020 for therapies supported by Cryoport. Data provided by the Alliance for Regenerative Medicine states that there are currently a total of 1,052 clinical trials in the Regenerative Medicine market, globally, with 363 trials in Phase I, 594 in Phase II, and 95 in Phase III.

"With our strong business model and approximately $94 million in cash and short-term investments, we believe we are well positioned to drive growth organically and through acquisitions as we further enmesh ourselves within the Regenerative Medicine industry and build out our global supply chain network at this pivotal time in the market’s evolution. With this strong foundation, we look forward to continuing to advance our mission to bring life changing therapies to patients while building shareholder value."

Market Highlights:

Global Logistics Solutions

Biopharma

Biopharma revenue increased by 67% to $7.5 million for the three months ended September 30, 2019 compared to $4.5 million for the same period in 2018.
A net addition of 12 new biopharma clinical trials were added during the three months ended September 30, 2019.
Cryoport is now supporting a net total of 425 clinical trials as of September 30, 2019 compared with 323 as of September 30, 2018. The number of trials in Phase III grew to 54, compared with 46 as of September 30, 2018. Of the 425 total trials Cryoport supports, 360 are in the Americas, 55 in EMEA (Europe, the Middle East and Africa) and 10 in APAC (Asia Pacific). This compares to 295 in the Americas and 28 in EMEA as of September 30, 2018.
Lonza announced Cryoport as its preferred partner in the transport and delivery of patient tissues on a global basis, with the continued goal of seamless service for Lonza’s customers and their patients.
Vineti announced a commercial partnership with Cryoport designed to extend end-to-end delivery of cell therapies and gene therapies to a growing number of patients. Pairing Cryoport’s integrated temperature-controlled capabilities and near real-time monitoring with Vineti’s supply chain orchestration (SCO) platform with will support improved drug product quality for advanced therapies and patient safety.
Cryoport announced the industry’s first Advanced Therapy Shipper in its Cryoport Express product line for the Regenerative Medicine market. The Cryoport Express Advanced Therapy Shipper guarantees each shipper has been used only for human use and provides complete traceability of all equipment, components and commodities. The result is an industry first, providing unmatched verification information and supply chain support for biopharma companies researching and commercializing cell and gene therapies.
Animal Health

Revenue from Animal Health was $0.2 million for both the three months ended September 30, 2019 and 2018. We are now in a position to grow our revenue in the Animal Health market through new dedicated resources that are focused on this market on a global basis. During the quarter we made significant increases in our Animal Health pipeline, which we anticipate translating to revenue growth in 2020.

Reproductive Medicine

Reproductive Medicine revenue increased 26% for the three months ended September 30, 2019 compared to the same period in 2018, growing both domestically as well as internationally. This growth can be attributed to increasing awareness of our CryostorkTM platform which was launched in 2018 as well as maturing commercial relationships with large clinic networks.

Global Bioservices

Bioservices revenue for the three months ended September 30, 2019 was $1.2 million, resulting from our acquisition of the Cryogene business consummated in May 2019.

Financial Highlights:

Revenue increased 81% to $9.6 million for the three-month period ended September 30, 2019, compared with the same period in the prior year.
Revenue increased 77% to $24.7 million for the nine-month period ended September 30, 2019, compared with the same period in the prior year.
Excluding the $1.2 million of revenue for the three months ended September 30, 2019 from the Cryogene acquisition, consummated in May of 2019, revenue grew 59% and 65% for the three and nine-month periods ended September 30, 2019, compared with the same period in the prior year.
Gross margin for the three and nine-months ended September 30, 2019 was 48% and 50%, respectively, compared to 52% and 53% for both the respective periods in the prior year.
Third quarter financial results include a one-time charge of $10.8 million in stock-based compensation expense (non-cash) of which $0.4 million and $10.4 million are included in cost of revenues and operating costs and expenses, respectively. This charge reflects 24 months of automatic accelerated vesting under the terms of certain outstanding stock option grants as a result of the Company meeting certain financial performance criteria defined in such grants, including reaching positive adjusted EBITDA for two consecutive quarters.
Operating costs and expenses increased by $12.1 million, and $15.4 million, for the three and nine-month periods ended September 30, 2019, respectively, compared to the same periods in the prior year, as a result of $10.4 million in one-time accelerated stock-based compensation expenses (as explained above) as well as continued investments in the build out of infrastructure, including the addition of two new Global Logistics Centers in the U.S. and Europe during the second half of 2018. Without one-time accelerated stock-based compensation expenses the operating costs and expenses increased by $1.7 million, and $5.0 million, for the three and nine-month periods ended September 30, 2019, respectively, compared to the same periods in the prior year.
Adjusted EBITDA for the three-month period ended September 30, 2019 was $1.2 million, compared with ($0.4 million) in the same three-month period in the prior year. Adjusted EBITDA for the nine-month period ended September 30, 2019, was $0.9 million, compared with ($1.8 million) in the same nine-month period in the prior year.
Net loss for the three-month period ended September 30, 2019 was $12.5 million, or $0.35 per share (Adjusted net loss was $1.7 million, or $0.05 per share, excluding the one-time charge of $10.8 million in accelerated vesting stock-based compensation expense), compared to a net loss of $2.1 million, or $0.07 per share in the same three-month period in 2018.
Net loss for the nine-month period ended September 30, 2019 was $17.4 million, or $0.54 per share (Adjusted net loss was $6.6 million, or $0.20 per share, excluding the one-time charge of $10.8 million in accelerated vesting stock-based compensation expense), compared with $7.3 million, or $0.26 per share, in the same nine-month period in 2018.
Cryoport reported $93.5 million in cash, cash equivalents and short-term investments as of September 30, 2019, compared with $47.3 million as of December 31, 2018. This increase includes net proceeds of $68.8 million received from a public offering completed in June 2019.
Subsequent to the quarter end, Cryoport’s Board of Directors unanimously authorized a share repurchase program, under which Cryoport may repurchase up to $15 million of its outstanding common stock through December 31, 2020.
Further information on Cryoport’s financial results is included on the attached condensed consolidated balance sheets and statements of operations, and additional explanations of Cryoport’s financial performance is provided in Cryoport’s quarterly report on Form 10-Q for the three-month period ended September 30, 2019, which will be filed with the Securities and Exchange Commission ("SEC") on November 12, 2019. The full report will be available on the SEC Filings section of the Investor Relations section of Cryoport’s website at www.cryoport.com.

Earnings Conference Call Information

IMPORTANT INFORMATION: A document titled "Cryoport Third Quarter 2019 in Review", which will provide a review of Cryoport’s recent financial and operational performance and a general business update, will be issued by management at 4:05 pm ET on Thursday, November 7. The document is designed to be read by investors before the questions and answers conference call and can be accessed at http://ir.cryoport.com/events-and-presentations.

Cryoport management will host a conference call at 5:00 pm ET on November 7, 2019. The conference call will be in the format of a questions and answers session and will address any queries investors have regarding Cryoport’s reported results.

Conference Call Information Conference Call Information

Date:

November 7, 2019

Time:

5:00 p.m. ET

Dial-in numbers:

+1 (800) 895-3361 (U.S.) or +1 (785) 424-1062 (International)

Confirmation code:

Request the "Cryoport Call"

Live webcast:

‘Investor Relations’ section at www.cryoport.com or at this link. Please allow 10 minutes prior to the call to visit this site to download and install any necessary audio software.

An archive of the question and answer webcast will be available approximately three hours after completion of the live event and will be accessible on the Investor Relations section of the Company’s website at www.cryoport.com for a limited time. To access the replay of the webcast, please follow this link. A dial-in replay of the call will also be available to those interested until November 14, 2019. To access the replay, dial +1 (844) 512-2921 (United States) or +1 (412) 317-6671 (International) and enter replay pin number: 136824.

On November 7, 2019 Amgen (NASDAQ:AMGN) reported that it will present at the 28th Annual Credit Suisse Healthcare Conference at 9:45 a.m. MT on Tuesday, Nov. 12, 2019, in Scottsdale, Ariz (Press release, Amgen, NOV 7, 2019, View Source [SID1234550840]). David W. Meline, executive vice president and chief financial officer at Amgen, will present at the conference . Live audio of the presentation can be accessed from the Events Calendar on Amgen’s website, www.amgen.com, under Investors. A replay of the webcast will also be available on Amgen’s website for at least 90 days following the event.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On November 7, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its results for the quarter ended September 30, 2019 (Press release, Corcept Therapeutics, NOV 7, 2019, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-third-quarter-2019-financial [SID1234552971]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Revenue of $81.5 million, a 26 percent increase from third quarter 2018
GAAP diluted net income of $0.22 per share, compared to $0.14 per share in third quarter 2018
Non-GAAP diluted net income of $0.31 per share, compared to $0.22 per share in third quarter 2018
Cash and investments of $266.9 million, compared to $225.7 million in second quarter 2019
2019 revenue guidance narrowed to $300 – $315 million
Corcept reported quarterly revenue of $81.5 million in the third quarter, compared to $64.4 million in the third quarter of 2018. Third quarter GAAP net income was $26.3 million, compared to $17.7 million in the same period last year. Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the third quarter was $37.8 million, compared to $27.9 million in the third quarter of 2018. A reconciliation of GAAP to non-GAAP net income is included below.

The company narrowed 2019 revenue guidance to $300 – $315 million. Guidance had previously been
$285 – $315 million.

Third quarter operating expenses were $48.5 million, compared to $41.5 million in the third quarter of 2018, primarily due to increased spending to recruit and compensate additional personnel and discover and develop new selective cortisol modulators, as well as increased legal expense. Cash and investments were $266.9 million at September 30, 2019, an increase of $41.2 million from June 30, 2019.

"Our Cushing’s syndrome business had an excellent quarter," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "We expect the number of patients receiving Korlym and physicians prescribing the medication to continue to increase. To reach more doctors, we are expanding our sales force. We expect the clinical specialists we are hiring now to begin contributing to our results next year.

"I am also pleased to announce an important advance in our program to treat serious metabolic disorders. In a double-blind, placebo-controlled trial in healthy subjects, our selective cortisol modulator miricorilant significantly reduced the weight gain caused by the commonly prescribed antipsychotic medication olanzapine (Eli Lily’s drug, Zyprexa). We have already initiated one of two planned Phase 2 trials to further test miricorilant’s activity in this indication."

Cushing’s Syndrome

European sites begin dosing patients in Phase 3 trial ("GRACE") of relacorilant to treat patients with Cushing’s syndrome
Double-blind, placebo-controlled, Phase 3 trial of relacorilant in patients whose Cushing’s syndrome is caused by adrenal adenomas to start in the first quarter of next year
"As of today, 42 of 62 planned sites are recruiting patients for GRACE," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "We expect to open an additional 13 sites by the end of the year. The activation pace of ex-US sites, which we expect will provide the majority of enrollments, has refined our estimate of the trial’s completion date. Our plan is to submit our NDA in the fourth quarter of 2021.

We spent substantial time in Europe in the past quarter helping clinical site activation and speaking to investigators. Most important, our investigators are highly enthusiastic about GRACE1, because of relacorilant’s positive Phase 2 efficacy and side effect profile."

Patients in relacorilant’s Phase 2 trial exhibited meaningful improvements in glucose control and hypertension – two of Cushing’s syndrome’s most common and pernicious symptoms. The trial also met a wide range of secondary endpoints, including weight loss, liver function, coagulopathy, insulin resistance, cognitive function, mood and quality of life. These results were achieved without relacorilant causing Korlym’s significant off-target effects – vaginal bleeding, endometrial thickening and low potassium.2

In addition to GRACE, Corcept plans to start a Phase 3, double-blind, placebo-controlled trial of relacorilant in patients whose Cushing’s syndrome is caused by an adrenal adenoma – a population that has not been rigorously studied. Patients with adrenal Cushing’s syndrome typically experience a slower onset of symptoms, but their ultimate health outcomes are poor. Corcept expects to enroll 130 patients at sites in the United States and Europe in the study. Most of the planned investigators and sites are also participating in GRACE.

Metabolic Disease

Positive top-line results from double-blind, placebo-controlled, Phase 1b trial of miricorilant to reduce antipsychotic-induced weight gain
Recruiting underway in double-blind, placebo-controlled, Phase 2 trial of miricorilant to reverse recent antipsychotic-induced weight gain
"Our program to develop miricorilant as a treatment for metabolic disorders is off to an excellent start," said Dr. Grauer. "Antipsychotic medications such as olanzapine are essential to the health of millions of patients, but the weight gain and other metabolic side effects they cause are life-threatening and often lead patients to discontinue treatment. At the first dose level tested in our Phase 1b trial, healthy subjects given olanzapine plus miricorilant gained less weight than subjects receiving olanzapine plus placebo (see Figure 1). In addition, markers of liver damage that often rise temporarily at the start of olanzapine therapy increased less sharply in subjects receiving miricorilant, suggesting that miricorilant may have protective effects in the liver (see Figure 2). Five subjects in the olanzapine alone group were unable to complete the study due to elevated liver enzymes, while one patient in the miricorilant group experienced this problem."

A photo accompanying this announcement is available at View Source

The Phase 1b trial’s first part enrolled 66 healthy subjects, each of whom received olanzapine (10 mg) and either miricorilant (600 mg) or placebo daily. The trial’s duration was two weeks. The second part of the trial, which is planned to start in December, will test a higher dose of miricorilant (900 mg) in 30 healthy subjects. The study’s full results will be presented at a scientific meeting in 2020.

"These preliminary results are especially encouraging given the short duration of treatment and the low dose of miricorilant. They are consistent with the effects we had previously seen in animal studies. Our plan is to confirm these findings and explore the full breadth of miricorilant’s activity," said Dr. Grauer.

In addition to the second part of its Phase 1b trial, Corcept plans to conduct two double-blind, placebo-controlled Phase 2 trials of miricorilant for the treatment of patients with antipsychotic-induced weight gain. The first trial, which is underway, will test miricorilant’s activity in reversing recent weight gain. It is expected to enroll 100 patients with schizophrenia at 20 sites in the United States. Patients will continue to receive their established antipsychotic medication and will have either miricorilant or placebo added to their regimen for 12 weeks. A second Phase 2 trial is planned to start next year. It will enroll patients with long-standing weight gain. A third Phase 2 trial, testing miricorilant’s activity in preventing antipsychotic-induced weight gain, is under consideration.

Next year, Corcept also plans to start a double-blind, placebo-controlled, Phase 2 trial of miricorilant as a treatment for patients with non-alcoholic steatohepatitis (NASH), a serious liver disorder that afflicts millions of people.

Solid Tumors

European Commission designates relacorilant orphan drug for treatment of pancreatic cancer
Phase 3 trial of relacorilant plus nab-paclitaxel in patients with metastatic pancreatic cancer to start upon completion of consultations with the U.S. Food and Drug Administration (FDA)
"We are pleased the European Commission (EC) has joined the FDA in designating relacorilant an orphan drug for the treatment of pancreatic cancer," said Dr. Grauer. "The EC based its decision on the European Medicines Agency’s finding that relacorilant has the potential to significantly benefit patients.

"We presented the clinical data reviewed by the EMA at last year’s ASCO (Free ASCO Whitepaper) meeting and it was indeed promising," said Dr. Grauer. "Seven of 25 patients with metastatic pancreatic cancer treated with relacorilant plus nab-paclitaxel (Celgene’s drug, Abraxane) achieved durable disease control, meaning their tumors either shrank or ceased growing for 16 weeks or longer. Tumor response in two patients lasted more than 50 weeks.3 All of these patients’ tumors had progressed during multiple lines of prior therapy, including treatments with nab-paclitaxel or another taxane. That any of them responded is remarkable. We have sought FDA guidance as to the optimum development path in pancreatic cancer and plan to start a Phase 3 trial promptly upon the conclusion of our discussions."

Corcept’s 180-patient, placebo-controlled Phase 2 trial of relacorilant plus nab-paclitaxel in ovarian cancer continues to enroll patients at sites in the United States and the European Union. Dosing also continues in the company’s Phase 1/2 study of exicorilant plus enzalutamide in patients with castration-resistant prostate cancer.

Conference Call

We will hold a conference call on November 7, 2019, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, dial 1-877-260-1479 from the United States or 1-334-323-0522 internationally approximately ten minutes before the start of the call (passcode 8532239). A replay will be available through November 21, 2019 at 1-888-203-1112 in the United States and 1-719-457-0820 internationally (passcode 8532239).

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.