On May 10, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported first quarter 2019 financial results and business highlights, which will be discussed on a conference call scheduled for Tuesday, May 14 at 10:00 a.m. ET (Press release, Abeona Therapeutics, MAY 10, 2019, View Source [SID1234536157]). Interested parties are invited to participate in the call by dialing 844-369-8770 (toll-free domestic) or 862-298-0840 (International) or via webcast at View Source

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"We’ve had a great start to 2019, with continued progress across our pipeline, including the completion of CMC work in advance of initiating our Phase 3 VITAL clinical trial evaluating EB-101, our gene-corrected cell therapy for patients with recessive dystrophic epidermolysis bullosa," said João Siffert, M.D., Chief Executive Officer. "The study, which will utilize clinical material produced at our Cleveland manufacturing facility, is on track to start mid-2019 following completion of ongoing FDA review."

"We have continued to advance our lysosomal storage disease programs. Our increased efforts around patient recruitment are starting to bear fruit as we observe an uptick in patient screening for both MPS programs. In addition, we have submitted the IND for our CLN1 program." added Dr. Siffert.

First Quarter Financial Results:

Cash, cash equivalents and marketable securities as of March 31, 2019 were $68.3 million, compared to $85.0 million as of December 31, 2018. The decrease in cash of $16.7 million was driven primarily by the net cash used for operating activities of $15.1 million.

Research and development expenses for the first quarter ended March 31, 2019 were $11.7 million, compared to $8.2 million for first quarter 2018. The increase in research and development expense was primarily attributable to increased R&D headcount, related facility costs and internal manufacturing costs.

General and administrative expenses for the first quarter ended March 31, 2019 were $5.7 million, compared to $2.9 million for the first quarter 2018. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs.

Net loss was $0.39 per share for the first quarter of 2019, compared to $0.22 per share in the same period of 2018.

First Quarter and Recent Highlights:

·May 8, 2019: Abeona Therapeutics Announces Presentation of EB-101 Data at Society for Investigative Dermatology Annual Meeting
oFollow up data showed that three years after treatment with investigational product, EB-101, a majority of RDEB patients had durable wound healing and improvements in pain and itching. There were no serious treatment-related adverse events three years post-treatment and no replication competent virus present at any time point.
·May 1, 2019: Reported Preclinical Data Demonstrating Broad Therapeutic Potential of AIM Gene Therapy in Retinal Diseases at Association for Research in Vision and Ophthalmology Annual Meeting
oIntravitreal administration of the Company’s novel AIM AAV204 capsid in non-human primates led to robust transgene expression in the inner and outer retina. These preclinical data support the potential use of intravitreal administration to deliver gene therapy in an out-patient setting for a wide range of inherited and acquired retinal diseases.
·April 30, 2019: Reported New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 for Treatment of Cystic Fibrosis at American Society of Gene and Cell Therapy Annual Meeting
oABO-401, the Company’s novel gene therapy for cystic fibrosis (CF) efficiently delivered a highly-expressed, functional copy of human mini-CFTR (hCFTR) to the lung of CF mice and restored CFTR function in human CF patient nasal and bronchial epithelial cells.
·April 4, 2019: Received FDA Fast Track Designation for ABO-101 for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)
·February 11, 2019: Appointed João Siffert, M.D. Chief Executive Officer
·January 31, 2019: Presented New Supportive Data for Novel Gene Therapies at WORLDSymposium
oPlatform and poster presentations highlighted study results on biodistribution and tissue tropism of the next-generation AIM AAV vector platform in Pompe and Fabry diseases, as well as data from programs in MPS IIIA and CLN3 disease.
·January 8, 2019: Strengthened Financial Leadership with New Executive Appointments: Christine Silverstein as Chief Financial Officer and Edward Carr as Chief Accounting Officer

"We expect 2019 to be a transformative year for Abeona, as we prepare to execute on a number of milestones in the clinic, in our mission to develop gene and cell therapies that could transform the treatment of serious diseases," said Steven H. Rouhandeh, Chairman of the Board and Executive Chairman. "We are encouraged to continue advancing the proprietary technology of our AIM vector platform, especially following recent promising data presented across multiple indications, for which we continue to evaluate avenues of development internally, and with potential partners."

On May 10, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the first quarter ended March 31, 2019 and recent company developments (Press release, TG Therapeutics, MAY 10, 2019, View Source [SID1234536163]).

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "2019 has been an extremely productive year thus far with the achievement of many key milestones, the most important of which has been the significant progress made with our Marginal Zone Lymphoma program, including the release of positive top-line results and the receipt of breakthrough therapy designation and orphan drug designation. Moving forward, we believe further clarity on our potential MZL NDA filing by year-end along with the presentation of final MZL data later this year has the potential to unlock significant value for our shareholders." Mr. Weiss continued, "Between now and the middle of next year, we expect to have pivotal data readouts across three trials, including umbralisib in non-Hodgkin’s lymphoma, umbralisib plus ublituximab (U2) in chronic lymphocytic leukemia and ublituximab in multiple sclerosis. This is an extremely exciting time for us and we look forward to an impactful remainder of 2019 and 2020."

Recent Developments and Highlights

●Marginal Zone Lymphoma – Breakthrough Therapy and Orphan Drug Designations: Received breakthrough therapy designation (BTD) and orphan drug designation for umbralisib (TGR-1202), for the treatment of patients with all three types of marginal zone lymphoma (MZL): nodal, extranodal and splenic MZL.
●Positive Results from MZL Cohort of UNITY-NHL Trial: Announced positive outcome from the MZL cohort of the UNITY-NHL trial, which met the primary endpoint of Overall Response Rate (ORR). Interim safety and efficacy data from this study were presented in an oral presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in Atlanta.
●DSMB Updates: Meetings held by the independent Data Safety Monitoring Boards (DSMBs) for both the UNITY-CLL trial and the UNITY-NHL trial did not raise any safety concerns and recommended that both trials continue unmodified.
●TG-1801 (Anti-CD47/CD19): Commenced a Phase 1 first-in-human, dose-escalation study of TG-1801 in patients with relapsed or refractory B-cell lymphoma.
●Multiple Sclerosis Data: Presented long-term safety data from the open label extension (OLE) of the Phase 2 trial of ublituximab in multiple sclerosis, which demonstrated that ublituximab continues to be well tolerated.

Remaining 2019 Milestones

●Potential top-line progression free survival (PFS) results from the Phase 3 UNITY-CLL trial evaluating U2 in patients with CLL
●Present final data from the MZL cohort of the UNITY-NHL registration directed trial evaluating umbralisib in MZL
●Potential UNITY-NHL NDA filing in MZL
●Present updated data from our pipeline products and combination studies at upcoming major medical conferences

Financial Results for the First Quarter 2019

●Cash Position: Cash, cash equivalents and investment securities were $92.5 million as of March 31, 2019. Pro-forma cash, cash equivalents and investment securities as of March 31, 2019 (excluding our second quarter 2019 operations) are approximately $116.7 million, after giving effect to $24.2 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the second quarter of 2019.

●R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation) was $30.9 million for the three months ended March 31, 2019 compared to $32.2 million for the three months ended March 31, 2018. Included in other research and development expense for the three months ended March 31, 2019 was $13.8 million of clinical trial expense and $6.4 million of manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization. The current period decrease in Other R&D expenses is primarily due to full enrollment in our pivotal Phase III clinical development programs completed in the prior period. We expect the decrease in other R&D expenses to continue over the next several quarters.

●G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $1.9 million for the three months ended March 31, 2019 as compared to $2.1 million for the three months ended March 31, 2018. Other G&A expenses for the three months ended March 31, 2019 remained relatively flat compared to the first quarter of 2018, and we expect Other G&A expenses to increase modestly through the remainder of 2019.

●Net Loss: Net loss was $35.2 million for the three months ended March 31, 2019, compared to a net loss of $41.5 million for the three months ended March 31, 2018. Excluding non-cash items, the net loss for the three months ended March 31, 2019 was approximately $33.3 million.

●Financial Guidance: Net cash utilized for operating activities during the three months ended March 31, 2019 was approximately $33.5 million. The Company believes its cash, cash equivalents and investment securities on hand as of March 31, 2019, inclusive of the proceeds raised subsequent to the first quarter, will be sufficient to fund the Company’s planned operations through mid-2020.

Conference Call Information

The Company will host a conference call today, May 10, 2019, at 8:00 am ET, to discuss the Company’s first quarter 2019 financial results and provide a business outlook for the remainder of 2019.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics First Quarter 2019 Business Update Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

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On May 10, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present at the Bank of America Merrill Lynch Health Care Conference in Las Vegas on Wednesday, May 15, 2019 at 1:00 p.m. ET (Press release, BioCryst Pharmaceuticals, MAY 10, 2019, View Source [SID1234536158]).

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Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.

On May 10, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq:YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the first quarter of 2019 (Press release, Y-mAbs Therapeutics, MAY 10, 2019, View Source [SID1234536194]).

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"We believe Y-mAbs is adapting very well to its new role as a public company, and we are very pleased with our first quarter financial results, especially seen in conjunction with our clinical progress moving closer to our expected target of being able to file BLAs for both of our two lead pediatric drug candidates, naxitamab and omburtamab in 2019. These are exciting times for Y-mAbs," stated Thomas Gad, Founder, President and Head of Business Development and Strategy.

Dr. Moller, Chief Executive Officer continued, "We believe we have achieved the planned clinical progress with both the naxitamab and omburtamab trials during the first quarter of 2019 and we are confident that we can expect to complete the recruitment of patients for these pivotal trials for both compounds by mid 2019. We are excited to move these two antibody compounds forward to registration, and we have also initiated treatment of patients with our bispecific GD2xCD3 antibody. We believe this underlines our position as a leader in pediatric oncology and as a company focused on rapidly developing therapies to extend and enhance the lives of those living with rare pediatrics cancers."

First Quarter 2019 Financial Results

Y-mAbs reported a net loss of $15.9 million, or $0.47 per basic and diluted share, for the first quarter of 2019, compared to a net loss of $7.5 million, or $0.28 per basic and diluted share, for the first quarter of 2018.

Cost and Operating Expenses

Research and Development

Research and development expenses were $12.5 million for the quarter ended March 31, 2019, compared to $6.2 million for the corresponding period of 2018, an increase of $6.3 million. The increase in research and development expenses primarily reflects the following:

·$4.3 million increase in outsourced manufacturing for our lead product candidates, naxitamab and omburtamab

·$1.3 million increase in outsourced research and supplies to support expanding development activities

·$0.8 million increase in personnel costs

General and Administration

General and administrative expenses were $3.7 million for the quarter ended March 31, 2019, compared to $1.3 million for the same period of 2018, an increase of $2.4 million. Such increase in general and administrative expenses was primarily reflects the following expenses:

·$1.4 million increase in personnel costs

·$0.3 million increase in commercial infrastructure

Cash and Cash Equivalents

The Company had approximately $134.2 million in cash and cash equivalents as of March 31, 2019 compared to $147.8 million as of December 31, 2018. The decrease of $13.6 million was primarily attributable to the increased costs of operation.

On May 10, 2019 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK), an oncology company focused on biomarker-defined cancers, reported its first quarter 2019 financial results for the period ended March 31, 2019 (Press release, Merrimack, MAY 10, 2019, View Source [SID1234536169]).

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"We continue to focus our efforts on completing the review of our strategic alternatives through which we are committed to optimizing value for our stockholders, including the preservation of potential contractual milestone payments Merrimack is eligible to receive," said Richard Peters, M.D., Ph.D., President and Chief Executive Officer of Merrimack. "To that end, we have recently implemented a series of measures to scale back operations and strengthen our balance sheet."

Corporate Update

As previously announced, Merrimack has engaged external advisors to explore the Company’s strategic alternatives. In the first quarter and more recently, Merrimack has implemented a series of measures designed to preserve its current resources as part of this ongoing process:

In April 2019, Merrimack terminated its Phase 1 study of MM-310 and discontinued the program, following a comprehensive review of available safety data indicating that the drug would not achieve an optimal therapeutic index. In parallel, the Company announced it would close out all remaining clinical activities;

In April 2019, Merrimack initiated a workforce reduction in connection with the Company’s decision to close out clinical activities and in line with prior cost-cutting measures. Merrimack estimates that it will incur approximately $1.5 million to $1.7 million in one-time charges in connection with this workforce reduction, which is expected to be substantially completed by May 31, 2019;

In April 2019, Merrimack retired its outstanding debt with Hercules Capital ahead of schedule, which totaled $16.0 million with interest and fees; and

In May 2019, Merrimack monetized certain assets to strengthen its cash position. This includes the sale of its equity position in Silver Creek Pharmaceuticals, resulting in $7.8 million in cash, and the auction of laboratory equipment from the Company’s research and development operations, resulting in approximately $1.3 million in cash.

Merrimack continues to operate with a narrow research footprint, focused on two preclinical programs: MM-401, an agonistic antibody targeting a novel immuno-oncology target, TNFR2; and MM-201, a highly stabilized agonist-Fc fusion protein targeting death receptors 4 and 5. Merrimack plans to prudently advance these programs, with deference to the Company’s ongoing strategic process.

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held March 29 – April 3, 2019 in Atlanta, Georgia, Merrimack presented six posters highlighting its preclinical work, of which three featured MM-401 and two featured MM-201.

First Quarter 2019 Financial Results

The following summarizes Merrimack’s financial results for the three months ended March 31, 2019:

Research and development expenses for the three months ended March 31, 2019 were $6.4 million, compared to $13.1 million for the three months ended March 31, 2018. Research and development spending for the first quarter of 2019 was lower versus the comparable period in 2018, primarily due to phasing and close out of several clinical development programs;

General and administrative expenses for the three months ended March 31, 2019 were $3.7 million, compared to $4.3 million for the three months ended March 31, 2018. General and administrative spending for the first quarter of 2019 was less than expenditures over the comparable period in 2018, primarily due to a decrease in corporate expenses related to reduced headcount levels and stock-based compensation;

Net loss for the three months ended March 31, 2019 was $10.5 million, or $0.78 per share, compared to a net loss attributable to Merrimack’s continuing operations of $17.8 million, or $1.33 per share, for the three months ended March 31, 2018; and

As of March 31, 2019, Merrimack had 13.3 million shares of common stock, $0.01 par value per share, outstanding.

Financial Outlook

As of March 31, 2019, Merrimack had cash, cash equivalents and marketable securities of $58.5 million. Accounting for events subsequent to the quarter close, together with possible additional restructuring and cost-cutting measures that Merrimack could implement in the future, but excluding any potential contractual milestone payments, Merrimack continues to believe that its cash position has the potential to fund operations into at least the second half of 2022.

Merrimack remains eligible to receive additional milestone payments from Servier and Ipsen, resulting from Merrimack’s asset sale to Ipsen in 2017:

Merrimack is entitled to receive up to $5 million in milestones from Servier, triggered by Ipsen and Servier’s decision to progress their ongoing multi-part clinical trial evaluating ONIVYDE in small-cell lung cancer (SCLC) into the second randomized portion of the trial focused on efficacy; and

Merrimack is also entitled to receive up to an aggregate of $450 million in regulatory-based milestones from Ipsen, which Merrimack has said it expects to pass through to its stockholders, net of any taxes owed and subject to there being sufficient surplus at that time, consisting of:

$225 million upon approval by the FDA of ONIVYDE for the first-line treatment of metastatic adenocarcinoma of the pancreas, subject to certain conditions;

$150 million upon approval by the FDA of ONIVYDE for the treatment of SCLC after failure of first-line chemotherapy; and

$75 million upon approval by the FDA of ONIVYDE for an additional indication unrelated to those described above.