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BioSpecifics Technologies Corp. Reports First Quarter 2019 Financial and Operating Results

On May 10, 2019 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase-based therapies with a first in class collagenase-based product marketed as XIAFLEX in the U.S. and Xiapex in Europe, reported its financial results for the first quarter ended March 31, 2019 and provided a corporate update (Press release, BioSpecifics Technologies, MAY 10, 2019, View Source [SID1234536159]).

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"The first quarter of 2019 was an important time of internal execution as we worked through our path forward at BioSpecifics under our new leadership, with a focus on continued clinical and commercial advancements and driving long-term value. Importantly, we reported the full positive Phase 1 uterine fibroids data and are now assessing next steps for this medically necessary indication in women’s health, by working with our clinical partners as we further analyze the data to inform a Phase 2 study," said Dr. Ron Law, Ph.D., J.D., Principal Executive Officer of BioSpecifics. "We are also pleased to see such encouraging increases in XIAFLEX net sales as well as improved patient access for both of our marketed indications and we look forward to the upcoming BLA filing of CCH for the treatment of cellulite, which is expected in the second half of 2019."

First Quarter 2019 Financial Results

BioSpecifics reported net income of $4.4 million for the first quarter ended March 31, 2019, or $0.61 per basic share and $0.60 per share on a fully diluted basis, compared to net income of $4.0 million, or $0.55 per basic share and $0.54 per share on a fully diluted basis, for the same period in 2018.

Total revenue for the first quarter ended March 31, 2019 was $8.1 million, compared to $7.1 million for the same period in 2018. The increase in total revenues for the quarterly period was primarily due to royalties associated with higher net sales of XIAFLEX in Dupuytren’s contracture and Peyronie’s disease.

Research and development expenses for the first quarter ended March 31, 2019 were approximately $0.1 million compared to $0.2 million for the same period in 2018.

General and administrative expenses for the first quarter ended March 31, 2019 were $2.9 million, compared to $2.1 million for the same period in 2018.

Provision for income taxes for the first quarters ended March 31, 2019 and 2018 were $1.1 million, in each period.

As of March 31, 2019, BioSpecifics had cash and cash equivalents and investments of $88.1 million, compared to $82.0 million as of December 31, 2018.

As of March 31, 2019, BioSpecifics had 7.3 million shares of common stock outstanding.

Commercial & Pipeline Highlights and Anticipated Upcoming Milestones

BioSpecifics’s Royalty Revenues from XIAFLEX Commercial Franchise Grew by 14% Year-Over-Year. The XIAFLEX commercial franchise royalty revenue growth was primarily attributable to underlying value growth in both Peyronie’s disease and Dupuytren’s contracture. The Company’s partner Endo International plc (Endo) continues to invest in growth initiatives to support the future increase in consumer awareness for both marketed indications.
Full Phase 1 Uterine Fibroids Data Presented at 66th Annual SRI Scientific Meeting in March 2019: Data from the Phase 1 clinical trial of CCH for the treatment of uterine fibroids were presented at the 66th Annual Meeting of the Society of Reproductive Investigation (SRI) on March 14, 2019 in Paris, France. The reported data showed safety and statistically significant reductions in collagen content compared to control fibroids with a median reduction of 39 percent (p<0.05), as well as a 21 percent average reduction in density of collagen bundles. BioSpecifics and its clinical partners continue to analyze the full Phase 1 data to guide the design of a Phase 2 study of CCH for the treatment of uterine fibroids.
New Phase 3 Cellulite Data Presented at 2019 Annual Meeting of the Academy of Dermatology in March 2019: Endo reported new data from the RELEASE-1 and RELEASE-2 Phase 3 studies during the "Late-breaking Research: Procedural Dermatology’" session at the 2019 Annual Meeting of the American Academy of Dermatology on March 2, 2019 in Washington, D.C. The reported data showed that a greater percentage of women treated with CCH (CCH vs. placebo: RELEASE 1, n=210 vs n=213; RELEASE-2, n=214 vs n=206) met the primary endpoint versus placebo in both the RELEASE-1 (P=0.006) and RELEASE-2 (P=0.002) studies. In addition, statistically significant improvements with CCH vs placebo were observed in eight of eight (RELEASE-1) and seven of eight (RELEASE-2) secondary endpoints. Other patient-centric endpoints were also evaluated, including improvement in the Subject Global Aesthetic Improvement Scale (S-GAIS), a five-point scale rating global aesthetic improvement in appearance, compared to pretreatment, as judged by the subject.
BLA filing for CCH for Treatment of Cellulite Expected in 2H19: Endo reported that it expects to file its Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for CCH for the treatment of cellulite in the second half of 2019 with an expected commercial launch in the second half of 2020 subject to FDA approval.

BioCryst to Present at Bank of America Merrill Lynch Health Care Conference

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]).

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.

Abeona Therapeutics Reports First Quarter 2019 Financial Results and Business Highlights

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

"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."

Pieris Pharmaceuticals Reports First Quarter 2019 Financial Results and Provides Corporate Update

On May 10, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer and other indications, reported financial results for the first quarter of 2019 ended March 31, 2019, and provided an update on the Company’s recent and future developments (Press release, Pieris Pharmaceuticals, MAY 10, 2019, View Source [SID1234536144]).

”We are looking forward to reporting detailed data from the phase 1 single ascending dose study of PRS-060, an inhaled IL-4 receptor alpha antagonist for asthma, at the 2019 American Thoracic Society International Conference later this month,” said Stephen S. Yoder, President and CEO of Pieris. ”We are also looking forward to presenting data from the currently-enrolling phase 1 multiple ascending dose study of that candidate, including the drug’s FeNO-reducing potential versus placebo, at an upcoming medical meeting. Beyond PRS-060, we continue to advance multiple respiratory discovery programs, both proprietary and in collaboration with AstraZeneca, driving value across our expanding respiratory franchise. We also continue to make headway in our immuno-oncology programs. We plan to report comprehensive data from the phase 1 dose-escalation study of PRS-343, a 4-1BB/HER2 bispecific for HER2-positive solid tumors later this year. We also plan to report data from the phase 1 combination study of PRS-343 with atezolizumab later this year. Additionally, we expect to file an IND application for PRS-344, a PD-L1/4-1BB bispecific drug candidate we are co-developing with Servier, by year-end. We continue to work hard on execution over the last year and are pleased to be able to share the resulting fruits of our labor with our shareholders over the coming months.”

PRS-060: Pieris will present detailed data from the phase 1 single ascending dose study of PRS-060, an inhaled IL-4 receptor alpha antagonist for asthma, at the 2019 American Thoracic Society (ATS) International Conference later this month. The Company previously announced topline data from this study, reporting that PRS-060 was found to be safe and well-tolerated in healthy volunteers. Pieris also plans to present data from the multiple ascending dose phase 1 study of PRS-060 in patients with mild asthma and elevated levels of fractional exhaled nitric oxide (FeNO), a validated biomarker of lung inflammation, at an upcoming medical meeting. This study evaluates the safety, tolerability and FeNO-reducing potential of inhaled PRS-060 versus placebo. PRS-060 is the lead candidate in Pieris’ respiratory collaboration with AstraZeneca. Pieris is sponsoring the single and multiple ascending dose phase 1 studies and AstraZeneca is funding the costs. Assuming successful completion of the ongoing multiple ascending dose phase 1 study, AstraZeneca would sponsor and fund the phase 2a study. Upon completion of that study, Pieris may exercise options to co-develop and, later, co-commercialize PRS-060.
Respiratory Pipeline: Pieris continues to advance two discovery-stage programs as part of its respiratory alliance with AstraZeneca, under which AstraZeneca may initiate up to two additional programs. The Company also continues to advance the two proprietary discovery-stage respiratory programs initiated last year and intends to initiate additional proprietary respiratory programs in 2019.
PRS-343: Pieris continues to enroll and treat patients in a phase 1 dose-escalation study of PRS-343, a 4-1BB/HER2 bispecific for HER2-positive solid tumors, and intends to report comprehensive data from the study later this year. The Company also continues to enroll the dose-escalation phase 1 study of PRS-343 in combination with atezolizumab and intends to report data from this trial later this year.

Immuno-Oncology Pipeline: Pieris plans to file an IND application for PRS-344, a 4-1BB/PD-L1 bispecific the Company is developing as part of its immuno-oncology collaboration with Servier, later this year.
PRS-080: Pieris will present data from the phase 2a study of PRS-080, a half-life-optimized hepcidin antagonist for anemia, at the 24th European Hematology Association (EHA) (Free EHA Whitepaper) Congress on June 16, 2019.
First Quarter Financial Update:

Cash Position – Cash, cash equivalents and investments totaled $110.8 million as of March 31, 2019, compared to a cash, cash equivalents and investments balance of $128.1 million as of December 31, 2018. The decrease was due to operating cash expense, annual bonus payments, and timing of receivables collection from our strategic partners.

R&D Expense – R&D expenses were $14.3 million for the three months ended March 31, 2019, compared to $7.9 million for the three months ended March 31, 2018. The increase in research and development expenses reflects increases in clinical, drug supply and manufacturing, and salaries and benefit costs associated with the advancement of our clinical and preclinical programs.

G&A Expense – G&A expenses were $4.9 million for the three months ended March 31, 2019, compared to $4.4 million for the three months ended March 31, 2018. The increase in G&A expenses reflects higher personnel costs and audit and tax professional fees.

Interest Income – Interest income was $0.5 million for the three months ended March 31, 2019, compared to $0.3 million in interest income earned in the comparable 2018 period. The Company earned higher rates of interest despite having lower overall investment amounts compared to the first quarter of 2018.

Other Expense – Other expense was $0.2 million for the three months ended March 31, 2019, compared to other expense of $0.9 million for the three months ended March 31, 2018. The decrease in other expense was the result of a strengthening of the U.S. dollar against the euro, positively impacting the remeasurement of U.S. dollar denominated monetary assets held in Germany, combined with lower foreign denominated balances.

Net Loss – Net loss was $10.3 million or $(0.20) per share for the three months ended March 31, 2019, compared to a net loss of $8.7 million or $(0.17) per share for the three months ended March 31, 2018.

Conference Call:

Pieris management will host a conference call beginning at 8:00 AM Eastern Daylight Time on Friday, May 10, 2019, to provide a corporate update. Individuals can join the call by dialing +1-877-407-8920 (US & Canada) or +1-412-902-1010 (International). An archived replay of the call will be available by dialing +1-877-660-6853 (US & Canada) or +1-201-612-7415 (International) and providing the Conference ID #: 13661472.

Stemline Therapeutics Reports First Quarter 2019 Financial Results

On May 10, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported financial results and business highlights for the first quarter ended March 31, 2019 (Press release, Stemline Therapeutics, MAY 10, 2019, View Source [SID1234536138]).

"Since ELZONRIS became commercially available in January, we have been very pleased with the progress we have made executing our launch plan," stated Robert Francomano, SVP and Global Head of Commercial. "Our entire organization is working hard to ensure patients with BPDCN have access to ELZONRIS. Although still early, we believe the launch is progressing extremely well and remain poised for a very successful 2019 and beyond."

Ivan Bergstein, M.D., CEO of Stemline Therapeutics, commented "We have built a solid foundation for growth, driven by our launch of ELZONRIS for patients with BPDCN. We are executing our commercial plan, including pursuing ongoing efforts to unlock additional value from ELZONRIS in other indications as well as from our entire pipeline, all with the goal of improving the lives of patients with cancer around the world."

First Quarter 2019 Financial Results Review

Net revenue for ELZONRIS was $5.05 million for the quarter ended March 31, 2019. Stemline began commercial sales of ELZONRIS within the United States in January 2019.

Stemline ended the first quarter with $124.4 million in cash, cash equivalents and investments. For the first quarter, Stemline had a net loss of $27.4 million. Cash expenditures for the first quarter of 2019 was $21.9 million.

Research and development expenses were $17.0 million for the first quarter of 2019, which reflects an increase of $4.3 million compared with $12.7 million for the first quarter of 2018. The higher cost was primarily driven by expense recorded related to repayment of research funding as a result of the first commercial sale of ELZONRIS.

Selling, general and administrative expenses were $16.0 million for the first quarter of 2019, which reflects an increase of $10.1 million compared with $5.9 million for the first quarter of 2018. The increase in costs were primarily attributable to launch expenses in support of the commercialization of ELZONRIS.

Recent Corporate Developments and Program Highlights

ELZONRIS (tagraxofusp) — Blastic plasmacytoid dendritic cell neoplasm (BPDCN)

· ELZONRIS was approved by the FDA on December 21, 2018 and commercially available for patients with BPDCN in the U.S. in January 2019.

· The New England Journal of Medicine published the pivotal trial results in its April 25th edition.

· We submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in January 2019 seeking marketing approval in Europe. The MAA was granted accelerated assessment and is currently under review.

ELZONRIS — Market Expansion Efforts

· We are conducting clinical trials to evaluate ELZONRIS in additional indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

· Based on clinical results observed in patients with CMML and MF, we are evaluating next steps, including potential registrational pathways. For CMML, we intend to provide our registration-directed plans mid-year.

·We are also evaluating additional expansion opportunities, including maintenance therapy after stem cell transplant in patients with BPDCN.

· In parallel, we plan to expand our clinical efforts later this year and next into subsets of AML patients, including those enriched for CD123+ expression.

· We expect to provide periodic updates on these programs throughout this year and next at scientific conferences.

ASCO Conference

· ELZONRIS clinical trial data in CMML and MF have been selected for two poster presentations at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) conference in June.

Other pipeline candidates

·We expect to provide periodic updates on our other product candidates, SL-701, SL-801, SL-901, and SL-1001, later this year.

Conference Call Information

Stemline will host a conference call and live webcast today at 8:00 a.m. ET to discuss first quarter 2019 financial results and recent business activities. The conference call can be accessed by dialing 1-800-667-5617 (domestic) or 1-334-323-0509 (international) and referring to conference ID 2090827.

The live webcast can be accessed via the company’s website (www.stemline.com), at the bottom of the "Investors & Media" section in the "News & Events" page. The webcast will be archived and made available for replay on the company’s website shortly after the event.

About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123

CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple

myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.