On May 9, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the first quarter 2019 and provided an overview of recent accomplishments and clinical development progress for its innovative drug pipeline, including for selinexor, Karyopharm’s first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound (Press release, Karyopharm, MAY 9, 2019, View Source [SID1234536006]).

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"It’s been an active first quarter of 2019, which included a U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) meeting to review our selinexor New Drug Application (NDA) and the subsequent extension of the Prescription Drug User Fee Act (PDUFA) action date by three months to July 6, 2019," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "We are working closely with the FDA while they complete their Priority Review of the selinexor NDA. Looking ahead to the remainder of 2019, we are highly focused on the potential U.S. commercial launch for selinexor. We are also continuing to advance our additional selinexor programs, including in earlier line multiple myeloma, diffuse large B-cell lymphoma, liposarcoma and endometrial cancer."

First Quarter 2019 and Recent Events

Selinexor in Multiple Myeloma

FDA Extends Review Period for NDA to July 6, 2019. On Feb 26, 2019, the FDA held an ODAC meeting to review the selinexor NDA requesting accelerated approval. The committee was specifically asked to vote on whether the approval of selinexor should be delayed until the results from the ongoing, randomized Phase 3 BOSTON study are available. In a vote of 8 Yes and 5 No, the ODAC recommended that the approval decision for selinexor should be delayed until the results of the BOSTON study are available. Following the ODAC meeting, and at the FDA’s request, Karyopharm submitted additional, existing clinical information as an amendment to the NDA. On March 14, 2019, the FDA extended the PDUFA action date for the selinexor NDA by three months to July 6, 2019. The NDA is currently under Priority Review by the FDA and is seeking accelerated approval for selinexor in combination with dexamethasone as a new treatment for patients based on the results of the Phase 2b STORM study in patients with triple class refractory multiple myeloma who were previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. Although the FDA will consider the recommendation of the ODAC panel, the final decision regarding the approval of the product is made by the FDA solely, and the recommendations by the panel are non-binding.

European Medicines Agency (EMA) Validates Marketing Authorization Application (MAA). On January 8, 2019, Karyopharm submitted a MAA to the EMA requesting conditional approval for selinexor, in combination with dexamethasone, as a new treatment for patients based on the results of the Phase 2b STORM study in patients with triple class refractory multiple myeloma who were previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. As a customary part of the marketing application review process, Karyopharm received the consolidated list of questions from EMA in early May 2019 and anticipates receiving additional feedback based on routine site audits and other activities. Karyopharm plans to promptly address the questions and feedback with EMA. To provide adequate time to evaluate the application and allow Karyopharm to respond to questions and feedback, the EMA has switched from an accelerated review to a traditional review. The Company expects to receive a decision on the application by the end of 2019.

Pivotal Phase 3 BOSTON Study in Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is progressing and, in January 2019, the Company announced the completion of patient enrollment in the study. Top-line data is expected by the end of 2019 or into 2020 contingent upon the occurrence of progression-free survival (PFS) events, the primary endpoint of the study. The BOSTON study is evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (bortezomib) (once weekly) and low dose dexamethasone (SVd), compared to standard twice weekly Velcade and low dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy. Data from the BOSTON study, if positive, would be used to support regulatory submissions to the FDA and EMA requesting the use of selinexor in patients with multiple myeloma who received at least one prior therapy. Regulatory approvals based on the results from the BOSTON study would also confirm accelerated and/or conditional approvals based on data from the Phase 2b STORM study.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

NDA and MAA Expected to be Submitted in the First Half of 2020. Following the positive top-line results from the Phase 2b SADAL study which were presented at ASH (Free ASH Whitepaper) 2018, Karyopharm expects to submit an NDA to the FDA and MAA to the EMA requesting accelerated and conditional approval, respectively, for patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for stem cell transplantation including CAR-T (chimeric antigen receptor modified T cell) therapies. In addition to Orphan Drug Designation, selinexor was granted Fast Track designation by the FDA in 2018.
Selinexor in Solid Tumors

Phase 3 SIENDO Study Evaluating Selinexor as Maintenance Therapy in Endometrial Cancer Will Now Be Conducted as a Company Sponsored Study. During the first quarter of 2019, an Investigational New Drug Application (IND) was submitted and accepted by FDA for a randomized, blinded Phase 2/3 study evaluating selinexor versus placebo as a maintenance therapy in patients with advanced or recurrent endometrial cancer following one prior platinum-based treatment. There are currently no approved therapies to treat patients with advanced or recurrent endometrial cancer in the maintenance setting. The primary endpoint of the SIENDO study is PFS. This study was previously an investigator sponsored study and has subsequently transitioned to a company sponsored study led by Professor Ignace Vergote, MD, PhD, Head of the Department of Obstetrics and Gynaecology and Gynaecologic Oncology at the Catholic University of Leuven, Belgium. Karyopharm is targeting enrollment completion for SIENDO in 2020.

Ongoing Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma. Karyopharm previously reported positive results from the Phase 2 portion of the randomized, blinded Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. Enrollment is currently ongoing in the Phase 3 portion of the SEAL study and, assuming a positive outcome on the primary endpoint of PFS, the Company intends to use the data from the SEAL study to support NDA and MAA submissions requesting approval for selinexor for patients with advanced unresectable dedifferentiated liposarcoma. Top-line data from the Phase 3 portion of the SEAL study are anticipated in 2020.
Eltanexor

Phase 1/2 Eltanexor Data in Metastatic Castrate-Resistant Prostate Cancer (mCRPC) Presented at ASCO (Free ASCO Whitepaper)-GU 2019. At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Genitourinary Cancers Symposium in February, Jingsong Zhang, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute, presented preliminary results from an ongoing Phase 1/2 investigator-sponsored study investigating eltanexor (+/- abiraterone) in patients with advanced cancers. The presented results showed that amongst the 23 patients with mCRPC evaluable for efficacy, 9% achieved a partial response and 74% achieved stable disease (≥8 weeks). The median treatment duration was 145 days, with three patients remaining on treatment as of November 19, 2018. Amongst the 30 patients evaluable for safety, treatment-related adverse events (TRAEs) occurring in ≥30% of patients included fatigue (80%; 17% Grade (Gr)≥3), nausea (70%; 0% Gr≥3), decreased appetite (60%; 0% Gr≥3), diarrhea (47%; 3% Gr≥3), weight decreased (47%; 3% Gr≥3), vomiting (40%; 7% Gr≥3), anemia (40%; 13% Gr≥3), neutropenia (33%; 13% Gr≥3), dysgeusia (33%; 0% Gr≥3) and thrombocytopenia (30%; 7% Gr≥3). There were two Gr4 TRAEs (neutropenia and elevated AST). Enrollment in the mCRPC arm of the study is now complete.
Verdinexor

$1.25 Million Grant from the U.S. Department of Defense (DoD) Awarded to Explore Use of Verdinexor in Spinal Cord Injury (SCI). A $1.25 million grant from the DoD was secured by an academic collaborator, with the assistance of Karyopharm, to study verdinexor (KPT-335) in preclinical models of SCI. The goal of this research is to establish the extent to which oral administration of verdinexor promotes tissue preservation and recovery of function following spinal cord injury. Verdinexor is also being studied as a potential anti-viral agent in a variety of human viral indications and for the treatment of systemic lupus erythematosus (SLE). For SLE, a $2.0 million grant was awarded from the National Institute of Health in 2018 and research is ongoing. The purpose of this grant is to investigate oral verdinexor activity in models of B-cell generation, double-stranded DNA antibody levels, and persistence of self-reactive antibody secreting cells.
Medical Congress Activity and Presentation of Data

Two Selinexor Abstracts Selected for Presentation at ASCO (Free ASCO Whitepaper) 2019. Two selinexor abstracts have been selected for presentations at the upcoming ASCO (Free ASCO Whitepaper) 2019 Annual Meeting taking place May 31-June 4, 2019 in Chicago. The first abstract, titled "Efficacy and Safety of Selinexor in Recurrent Glioblastoma," (Lassman, et al; Abstract #2005) was selected for an oral presentation and describes results from the Phase 2 KING study evaluating single-agent selinexor in patients with recurrent glioblastoma. The second abstract, titled "Overall Survival (OS) with Oral Selinexor Plus Low Dose Dexamethasone (Sd) in Patients with Triple Class Refractory-Multiple Myeloma (TCR-MM)," (Richardson, et al; Abstract #8014; Poster Board #340) was selected for a poster presentation and highlights overall survival (OS) data from the Phase 2b STORM study evaluating selinexor and low-dose dexamethasone in patients with triple class refractory multiple myeloma who have been previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available.

Seven Preclinical Abstracts Presented at AACR (Free AACR Whitepaper) 2019. Seven posters highlighting preclinical data for selinexor were presented at the recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2019 Annual Meeting. These posters described research evaluating selinexor in various preclinical models of high unmet need cancers, including multiple myeloma, chronic lymphocytic leukemia, breast, pancreatic, lung and metastatic brain cancers. A complete list of these presentations can be accessed by visiting the AACR (Free AACR Whitepaper) 2019 website at View Source
Corporate Updates

Michael P. Mason Appointed Chief Financial Officer. Karyopharm appointed Michael P. Mason as Chief Financial Officer. Mr. Mason formerly served as Vice President of Finance and Treasurer at Alnylam Pharmaceuticals, Inc., a public biopharmaceutical company. He brings over 18 years of diversified financial experience to Karyopharm and has extensive expertise in global financial operations and controls, financing transactions, business planning and supporting pharmaceutical product launches.

Tina Clark Beamon, Esq., Appointed Chief Compliance Officer. Karyopharm appointed Tina Clark Beamon, Esq. as Chief Compliance Officer. Ms. Beamon formerly served as Executive Director of Compliance and Ethics at Alexion Pharmaceuticals. Prior to Alexion, she served as the head attorney for the Oncology Division and the Consumer Healthcare Division at Boehringer Ingelheim USA Corporation. She brings 21 years of healthcare industry experience to Karyopharm and will serve an integral role in building upon Karyopharm’s existing high operating standards and its ongoing commitment to corporate compliance and ethics.
First Quarter 2019 Financial Results

Cash, cash equivalents and investments as of March 31, 2019, including restricted cash, totaled $265.1 million, compared to $330.9 million as of December 31, 2018.

License and other revenue for the quarter ended March 31, 2019 was $0.2 million, compared to $10.0 million for the quarter ended March 31, 2018. The revenue in 2018 was from the $10.0 million upfront payment for the asset sale of KPT-350 to Biogen in the first quarter of 2018.

For the quarter ended March 31, 2019, research and development expense was $38.0 million compared to $41.3 million for the quarter ended March 31, 2018. Karyopharm expects research and development expense to decrease going forward in 2019 compared to the first quarter of 2019. For the quarter ended March 31, 2019, general and administrative expense was $27.1 million compared to $7.6 million for the quarter ended March 31, 2018. The increase in general and administrative expenses compared to the prior year period was due primarily to the hiring of the Karyopharm commercial team and related commercial launch preparation activities to support the potential U.S. commercial launch of selinexor.

Karyopharm reported a net loss of $66.2 million, or $1.09 per share, for the quarter ended March 31, 2019, compared to a net loss of $38.5 million, or $0.78 per share, for the quarter ended March 31, 2018. Net loss includes stock-based compensation expense of $3.9 million and $4.2 million for the quarters ended March 31, 2019 and March 31, 2018, respectively.

Financial Outlook

Based on its current operating plans, which assume a selinexor commercial launch by July 2019, Karyopharm expects its operating expenses, excluding stock-based compensation expense, for the full year 2019 to be in the range of $200 million to $215 million. The Company expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations into the second half of 2020, also assuming the commercial launch of selinexor by July 2019. If the FDA decides to delay its approval decision for selinexor until the BOSTON data are available, Karyopharm will re-evaluate its spending expectations for 2019. Additional key activities expected in 2019 include supporting the ongoing multiple myeloma regulatory filings for selinexor in the U.S. and Europe, progressing the pivotal Phase 3 BOSTON study in multiple myeloma and preparing for NDA and MAA submissions in the U.S. and Europe, respectively, in DLBCL.

Further Information About Potential Accelerated Approval for Selinexor in Multiple Myeloma

The FDA instituted its Accelerated Approval Program to allow for expedited approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint or an intermediate clinical endpoint thought to predict clinical benefit, like overall response rate. Accelerated approval is available only for drugs that provide a meaningful therapeutic benefit over existing treatments at the time of consideration of the application for accelerated approval, which the FDA has reiterated in its feedback to the Company. Particularly in disease areas with multiple available and potential new therapies, such as multiple myeloma, accelerated approval carries a high regulatory threshold. Consistent with its general guidance, the FDA has noted to the Company its preference for randomized studies geared toward full approval, which the Company has undertaken with the ongoing pivotal, Phase 3 BOSTON study, and has reminded the Company that accelerated approval requires patients to have exhausted all available approved therapies.

Conference Call Information

Karyopharm will host a conference call today, Thursday, May 9, 2019, at 8:30 a.m. Eastern Time, to discuss the first quarter 2019 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 9756776. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

On May 9, 2019 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP) reported financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, Adamis Pharmaceuticals, MAY 9, 2019, View Source [SID1234536046]).

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Dr. Dennis J. Carlo, President and Chief Executive Officer of Adamis Pharmaceuticals, stated, "With the launch of our SYMJEPITM product this year, I believe Adamis is transitioning from a development-stage company with product candidates in the pipeline, into a commercial-stage company with multiple sources of revenue. Currently, I anticipate revenues from three sources for our company. First, SYMJEPI sales, mainly coming from the Sandoz retail launch, which we believe will occur shortly; second, sales from US Compounding, which have been steadily increasing; and finally, upfront payment and sales from anticipated commercialization arrangements relating to our naloxone product candidate. With all of the possible developments and potential revenue streams, I believe 2019 will be a very good year for Adamis."

Product Updates

SYMJEPI (epinephrine) Injection (0.3mg and 0.15mg)

On January 16, 2019, we announced that Sandoz had launched SYMJEPI (epinephrine) 0.3 mg Injection in the U.S. market. SYMJEPI will be rolled out via a phased launch and will initially be available in the institutional setting, an established channel where Sandoz has a significant experience and knowledge, followed by anticipated introduction into the retail market. We also anticipate that Sandoz will launch the lower dose SYMJEPI (epinephrine) 0.15 mg Injection product in the U.S. markets. With the continued rollout of SYMJEPI into the retail market, the company anticipates that the revenue stream to Adamis will increase in future quarters.

APC-6000 (naloxone)

On March 14, 2019, Adamis announced that the FDA had accepted the company’s New Drug Application (NDA) for review and provided a target agency action date of October 31, 2019. Naloxone is an opioid antagonist used to treat narcotic overdoses. The company believes that its higher dose naloxone product candidate, if approved, could be an important part of the solution to this growing health crisis. The company is in discussions with several potential commercial partners for the naloxone injection product candidate.

Other Pipeline Products

In order to focus resources on the naloxone product candidate, and in an effort to reduce operating expenses, the company has slowed development of its other pipeline product candidates for the near term. This includes a delay in the continuation of the start of patient enrollment for the company’s Phase 3 study for beclomethasone HFA (APC-1000) and a hold on the company’s sublingual tadalafil (APC-8000) product candidate. The company will prioritize future development for these products based on the availability of capital to support them and its ongoing evaluation of commercial potential.

Drug Outsourcing Facility

During the first quarter of 2019, the company continued to make changes in its wholly-owned subsidiary, US Compounding (USC), including the elimination of many lower margin products, reductions in operating cost and overhead, changes to senior leadership, and investments in and improvements to manufacturing processes, with the goals of improving overall efficiency, reducing operating expenses, and improving margins.

First Quarter 2019 Financial Results

Revenues were approximately $4.9 million and $3.2 million for the three months ended March 31, 2019 and 2018, respectively. Revenues increased by approximately $1.7 million in the first quarter of 2019 compared to the comparable period of 2018. This represents an approximate 18% increase over the last quarter of 2018 and an approximate 53% increase over the first quarter of 2018. The increase in revenues reflected the continued growth in sales of the USC division and, in part, revenues from the initial launch of SYMJEPI.

Selling, general and administrative expenses ("SG&A") for the first quarter increased approximately 23% over the first quarter of 2018, which were approximately $8.0 million and $6.5 million respectively. The single largest contributor to this increase was the initial annual maintenance fee paid to the FDA for SYMJEPI and the balance was due to an increase in direct materials, supplies and obsolete inventory caused by an increase in production of SYMJEPI and at US Compounding.

Research and development expenses for the first quarter of 2019 decreased $5.6 million from the fourth quarter of 2018. The bulk of this reduction was due to the completion of two late stage development projects for the company’s naloxone injection (APC-6000) and the sublingual tadalafil (APC-8000) product candidates.

Cash and equivalents at the end of the first quarter was $9.2 million. The company’s goal for the last three quarters of 2019 is to keep net cash used in operating and investing activities in the range of approximately $9-12 million. The company currently believes that the combination of reduced spending, cash on hand, an increase in cash flows from Sandoz and USC, and anticipated licensing fees or payments if we enter into a commercialization agreement relating to the naloxone product, would provide sufficient funding for the company through the end of 2019.

Targeted Future Milestones

●Launch by Sandoz of the SYMJEPI 0.3mg and 0.15mg products for the U.S. retail market;
●FDA approval for the higher dose naloxone product candidate – target agency action date of October 31, 2019;
●Commercial agreements for the naloxone product candidate and for the SYMJEPI products outside of the U.S.; and
●US Compounding becoming net positive for Adamis in 2019.

Conference Call

Adamis will host a conference call and live webcast on Thursday, May 9, 2019 at 2:00 pm Pacific Time to discuss its financial and operating results for the first quarter 2019 as well as provide an update on business developments and activities.

US Dial-in (Toll Free): 1-800-458-4148

TOLL/International Dial-in: 1-323-794-2597

Conference ID: 1311319

Webcast: View Source

In addition, a telephone playback of the call will be available after approximately 5:00 pm PT on May 9, 2019. To listen to the replay, call toll free 1-844-512-2921 within the United States or 1-412-317-6671 when calling internationally (toll). Please use the replay PIN number 1311319.

On May 9, 2019 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company focused on the development and commercialization of precisely targeted oncology therapies, reported financial results for the first quarter ended March 31, 2019 (Press release, Corvus Pharmaceuticals, MAY 9, 2019, View Source [SID1234536064]).

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"With the initiation of patient enrollment in our Phase1/1b trial of CPI-818, Corvus now has three agents with novel mechanisms of action in clinical trials for a wide range of cancers," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "Looking forward, we have several important milestones for each of our programs in 2019."

"For the adenosine pathway, we continue to be in a leadership position with ciforadenant (CPI-444), our small molecule inhibitor of the A2A receptor, and CPI-006, our anti-CD73 antibody. We are enrolling patients in two ciforadenant Phase 1b/2 trials and we have discovered a potentially predictive genetic biomarker, the Adenosine Signature, that may provide clinicians with the ability to select patients most likely to benefit from therapy. This positions us to potentially initiate a molecularly defined, late stage study of ciforadenant in patients with renal cell cancer around the end of the year. For CPI-006, the initial clinical data from both the monotherapy and combination arms of our Phase 1/1b clinical trial will be presented in an oral presentation at the ASCO (Free ASCO Whitepaper) annual meeting in June. In addition, we expect incremental data updates on these programs at major medical meetings over the course of the year."

"Turning to CPI-818, we are very excited to be investigating it in patients with T-cell lymphomas, a patient group that often has limited treatment options and poor clinical outcomes," continued Dr. Miller. "We believe that CPI-818 represents a novel approach for these patients and the Phase 1/1b study is designed to evaluate both anti-tumor activity and its effect on normal T-cells, which could provide valuable information for future trials of CPI-818 in other types of cancer and autoimmune diseases. We currently anticipate that initial data from the study will be presented in late 2019, providing another potential catalyst for the Company."

CPI-818, an oral, covalent, selective interleukin-2-inducible kinase (ITK) inhibitor, is based on a similar targeting strategy to that of Bruton’s tyrosine kinase (BTK) inhibitors. Key members of the scientific team at Corvus led the development of the first BTK inhibitor, ibrutinib, which is approved for the treatment of several types of B-cell lymphomas. ITK, the T-cell homologue of BTK, has many biochemical and functional similarities with BTK. T-cell lymphomas are often incurable, especially after relapse. As ITK is frequently overexpressed in T-cell lymphoma, we believe the selective inhibition of ITK may represent a new treatment strategy for this type of cancer, possibly analogous to the effects of BTK inhibition with ibrutinib in B-cell lymphomas. Unlike other ITK inhibitors, the selectivity of CPI-818 has been shown in preclinical studies to shift immune responses to a T-cytotoxic type 1 (Th1) phenotype. We believe CPI-818 has the potential to display a dual mechanism of action: direct cytotoxicity to T-cell lymphoma and enhancement of the immune system by increasing the Th1 immune response.

Recent Achievements
Ciforadenant (CPI-444): A2A Receptor Antagonist of Adenosine

Continued enrolling patients with renal cell cancer (RCC) in an amended Phase 1b/2 clinical trial evaluating ciforadenant in combination with Genentech’s Tecentriq (atezolizumab), an anti-PD-L1 antibody. The RCC patients in the trial have failed treatments with anti-PD-(L)1 antibodies and tyrosine kinase inhibitors.
Continued enrollment of up to 65 patients with non-small cell lung cancer (NSCLC) in a Phase 1b/2 trial being conducted by Genentech as part of their MORPHEUS platform. The study is evaluating ciforadenant and Tecentriq in patients who have failed no more than two prior regimens.
Presented updated data on the Adenosine Gene Signature (AdenoSig) highlighting its potential to enable patient selection for treatment with ciforadenant based on a molecularly defined gene signature, that may predict which patients may be more responsive to the adenosine blockade.
CPI-006: Anti-CD73 Antibody

Continued enrollment of up to 350 patients with advanced cancer in a Phase 1/1b clinical trial evaluating CPI-006 as a single agent and in combination with either ciforadenant or pembrolizumab. The trial is currently enrolling patients in the dose escalation phase for CPI-006 administered as a monotherapy and in combination with ciforadenant.
Initial clinical data from the Phase 1/1b study will be delivered in an oral presentation at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2019. This will build upon data presented in February that demonstrated early signs of immunologic activity across multiple pathways that may be important in cancer therapy.
CPI-818: A small molecule ITK inhibitor

Initiated enrollment of CPI-818, an ITK inhibitor, in a Phase 1/1b study in patients with several types of T-cell lymphomas, including peripheral T-cell lymphoma (PTCL), cutaneous T-cell lymphoma (CTCL) and others.
Presented preclinical and biochemical studies with CPI-818 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in March highlighting the selectivity and immunologic activity of CPI-818 and its anti-tumor activity in spontaneous canine T-cell lymphoma.
Financial Results

At March 31, 2019, Corvus had cash, cash equivalents and marketable securities totaling $105.8 million, as compared to cash, cash equivalents and marketable securities of $114.6 million at December 31, 2018.

Research and development expenses for the three months ended March 31, 2019 totaled $9.4 million compared to $12.1 million for the same period in 2018. The decrease of $2.7 million was primarily due to a decrease in ciforadenant program costs.

The net loss for the three months ended March 31, 2019 was $11.6 million, compared to a net loss of $14.3 million for the same period in 2018. Total stock compensation expense for the three months ended March 31, 2019 was $2.0 million compared to $1.8 million of total stock compensation expense for the same period in 2018.

Conference Call Details
Corvus will host a conference call and webcast today, Thursday, May 9, 2019, at 4:30 p.m. ET (1:30 p.m. PT), during which time management will provide a business update and discuss the first quarter 2019 financial results. The conference call can be accessed by dialing 1-800-479-1004 (toll-free domestic) or 1-720-543-0206 (international) and using the conference ID 5606517. The live webcast may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days.

On May 9, 2019 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported financial results and provided a business update for the first quarter ended March 31, 2019 (Press release, argenx, MAY 9, 2019, View Source [SID1234536080]).

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"During the first quarter, we advanced our broad clinical development plan of efgartigimod across four autoimmune indications in both our IV and subcutaneous formulations. Enrollment of our Phase 3 ADAPT clinical trial in generalized myasthenia gravis is on track, and we intend to launch a second Phase 3 program in primary immune thrombocytopenia in the second half of 2019 that will encompass both formulations. There remains a gap in autoimmune disease innovation for therapies that are specific and well-tolerated, and it is this need that drives us in our goal to deliver our FcRn antagonist to patients quickly," commented Tim Van Hauwermeiren, chief executive officer of argenx. "We also closed on our global collaboration with Janssen this quarter, receiving $500 million in upfront payments, to evaluate cusatuzumab for acute myeloid leukemia, myelodysplastic syndromes and other potential hematological indications."

"We look forward to our upcoming R&D Day where we will showcase our formula for translating immunology breakthroughs into first-in-class medicines and unveil two new antibody assets in our wholly-owned pipeline."

FIRST QUARTER 2019 AND RECENT HIGHLIGHTS

Pipeline Updates:

Efgartigimod (ARGX-113) Program

Phase 3 ADAPT clinical trial, including one-year open-label extension study, is ongoing for treatment of generalized myasthenia gravis with topline data expected in 2020.

argenx on track to launch global development program in primary immune thrombocytopenia (ITP) in second half 2019; update expected to be provided in third quarter 2019 on development strategy and regulatory feedback, including planned registration path and plan to bridge between intravenous (IV) and subcutaneous (SC) formulations.

Orphan drug designation granted in February 2019 by U.S. Food and Drug Administration (FDA) for treatment of primary ITP.

Ongoing open-label extension study from completed Phase 2 proof-of-concept clinical trial in ITP expected to close in mid-2019 in preparation for start of Phase 3 development program.

Phase 2 proof-of-concept clinical trial ongoing for treatment of pemphigus vulgaris; data expected in 2020.

Phase 2 clinical trial for treatment of chronic inflammatory demyelinating polyneuropathy expected to start in second half of 2019.

Phase 1 clinical trial in healthy volunteers planned with ENHANZE SC formulation of efgartigimod as part of collaboration with Halozyme announced in February 2019; data expected before end of 2019.

Collaboration provides argenx access to ENHANZE subcutaneous delivery technology for up to three targets, including exclusive rights to develop therapeutic products targeting human neonatal Fc receptor FcRn.

Upfront payment of $30 million paid to Halozyme with potential future payments up to $160 million per selected target subject to achievement of specified development, regulatory and sales-based milestones.

Cusatuzumab (ARGX-110) Program

Announced closing of exclusive global collaboration and license agreement with Janssen for cusatuzumab.

Received $300 million upfront cash payment, and Johnson & Johnson Innovation made equity investment of €176.7 million ($200.0 million based on exchange rate on date of signing) in argenx.

argenx retains right to co-promote cusatuzumab in United States and share such royalties with Janssen on 50-50 basis.

Orphan drug designation granted by FDA in January 2019 for treatment of acute myeloid leukemia.
Received first clinical milestone payment of $30 million for initiation of first-in-human clinical trial with antibody product candidate ABBV-151 (ARGX-115) as part of option agreement with AbbVie.

Corporate Updates

Torsten Dreier will resign from his function as chief development officer of argenx to focus on new role as chief development officer of AgomAb, a company founded through collaboration with argenx to advance ARGX-114, an HFG-mimetic SIMPLE Antibody directed against the MET receptor. Mr. Dreier will continue to serve as a consultant to argenx.

argenx Japan KK is being established as a wholly-owned subsidiary, and Hermann Strenger was appointed as General Manager of argenx Japan. In this role, Mr. Strenger will be responsible for all aspects of early commercial planning for efgartigimod in Japan. He has served in executive positions in the pharmaceutical industry in Japan for the last 23 years.

argenx R&D Day

argenx to host its second R&D Day on Thursday, May 22, 2019, to present new pipeline programs
ARGX-117 and ARGX-118.

Details of Financial Results

Cash, cash equivalents and current financial assets totaled €961.6 million on March 31, 2019, compared to €564.6 million on December 31, 2018 and €346.6 million on March 31, 2018. The increase in the cash balance on March 31, 2019 resulted primarily from the closing of the exclusive global collaboration and license agreement for cusatuzumab with Janssen triggering a $300 million upfront payment and a $200 million equity investment in January 2019.

Operating income increased by €33.1 million for the three months ended March 31, 2019 to reach €40.0 million, compared to €6.9 million for the three months ended March 31, 2018. The increase of €30.9 million in revenue was primarily related to the recognition of a $30.0 million development milestone under the AbbVie collaboration agreement and the partial recognition of the upfront payment received under the Janssen collaboration agreement. Other operating income increased by €2.2 million, resulting mainly from an increase in payroll tax rebates for employing certain research and development personnel.

Research and development expenses increased by €19.6 million for the three months ended March 31, 2019 to €34.8 million, compared to €15.1 million for the three months ended March 31, 2018. The increase in 2019 resulted primarily from (i) an increase of €11.1 million in external research and development expenses, reflecting higher clinical trials costs and manufacturing expenses related to the development of the late-stage argenx product candidate portfolio, (ii) an increase of €4.3 million in license fee costs payable to one of argenx’ licensors following the achievement of a development milestone under the AbbVie collaboration agreement and (iii) a €2.6 million increase in share-based compensation expenses linked to the grant of stock options to its research and development employees.

Selling, general and administrative expenses totaled €11.3 million and €5.9 million for the three months ended March 31, 2019 and 2018, respectively. The increase of €5.4 million in selling, general and administrative expenses for the three months ended March 31, 2019 primarily resulted from (i) an increase of €2.1 million in share-based compensation expenses linked to the grant of stock options to its selling, general and administrative employees and board members and (ii) higher personnel expenses and consulting fees related to the preparation of potential future commercialization of the lead product candidate efgartigimod.

For the three months ended March 31, 2019, financial income amounted to €3.5 million, compared to €0.5 million for the three months ended March 31, 2018. The increase of €3.0 million in 2019 related primarily to an increase in the interest received on cash, cash equivalents and current financial assets.

Exchange gains totaled €9.5 million for the three months ended March 31, 2019, compared to the €4.0 million exchange losses incurred for the three months ended March 31, 2018. The increase was mainly attributable to unrealized exchange rate gains on the cash, cash equivalents and current financial assets position in U.S. dollars due to the favorable fluctuation of the EUR/USD exchange rate in the first three months of 2019.

The company generated a total comprehensive profit of €6.7 million for the three months ended March 31, 2019, compared to a total comprehensive loss of €17.7 million for the three months ended March 31, 2018. The total comprehensive profit in the first quarter of 2019 mainly resulted from the recognition of a $30.0 million development milestone under the AbbVie collaboration agreement and the unrealized exchange rate gains accounted during the period.

EXPECTED 2019 FINANCIAL CALENDAR:

August 1, 2019: HY 2019 business update and financial results

October 24, 2019: Q3 2019 business update and financial results

On May 9, 2019 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a late-stage therapeutics development biopharmaceutical company pioneering advancements of cell therapies in select cardiovascular and autoimmune diseases, reported financial results for the three months ended March 31, 2019 and provides highlights of progress within the development pipeline (Press release, Caladrius Biosciences, MAY 9, 2019, View Source [SID1234536130]).

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"I am pleased with the pace of our accomplishments so far in 2019 as we continue to enroll subjects in both our ESCaPE-CMD study in the U.S. for CLBS14-CMD and our CLI study in Japan for CLBS12," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "The preliminary data for both studies are very promising and we expect to report top-line results for ESCaPE-CMD by the end of 2019 and for CLBS12 in the first half of 2020. In addition, we continue to work closely with the U.S Food and Drug Administration on the protocol design for our no-option refractory disabling angina ("NORDA") program and have targeted initiation of that Phase 3 study before the end of 2019."

"We look forward to building on the momentum we have created in advancing our clinical development pipeline and reporting on a number of important, value-creating milestones throughout the balance of this year," concluded Dr. Mazzo.

First Quarter Financial Highlights

Research and development expenses for the first quarter of 2019 were $2.0 million, a 10% decrease compared with $2.3 million for the first quarter of 2018. Research and development in both periods focused on the advancement of our ischemic repair platform and related to (i) expenses associated with our ongoing Phase 2 study of CLBS12 in critical limb ischemia development program in Japan, (ii) expense associated with our ongoing Phase 1b/2a study for CLBS14-CMD in coronary microvascular dysfunction, and (iii) expenses associated with the planning of our CLBS14-NORDA program in refractory angina.

General and administrative expenses, which focus on general corporate related activities, were $2.6 million for the first quarter of 2019, a 12% decrease compared with $2.9 million for the first quarter of 2018.

The net loss for the first quarter of 2019 was $4.4 million, or $0.44 per share, compared with $5.0 million, or $0.52 per share, for the first quarter of 2018.

Balance Sheet Highlights

As of March 31, 2019, Caladrius had cash, cash equivalents and marketable securities of $38.4 million. Based on existing programs and projections, the Company remains confident that its cash balances will allow it to fund its current business plan through the second quarter of 2020.

Conference Call

Caladrius’ management will host a conference call beginning at 4:30 p.m. ET on Thursday, May 9, 2019 to discuss the financial results, provide a company update and answer questions.

Shareholders and other interested parties may participate on the conference call by dialing (866) 595-8403 (domestic) or (706) 758-9979 (international), using the conference ID number: 2576003. The conference call will also be webcast live and can be accessed from the Company’s website at www.caladrius.com/investors/news-events.

For those unable to participate in the live conference call or webcast, an audio recording will be available for replay approximately two hours after the conclusion of the call until 11:59 p.m. ET on May 16, 2019. To access the audio replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and provide conference ID number: 2576003.

A webcast replay of the conference call will remain available on the Company’s website for 90 days.