On May 10, 2019 Sangamo Therapeutics, Inc. (NASDAQ: SGMO) reported that Sangamo will participate in the Bank of America Merrill Lynch 2019 Health Care Conference being held next week in Las Vegas (Press release, Sangamo Therapeutics, MAY 10, 2019, View Source [SID1234536175]). A "fireside chat" discussion with Sangamo CEO Sandy Macrae is scheduled for Tuesday, May 14th at 5 p.m. PT.

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The discussion will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The event will be archived on the Sangamo website for two weeks after the event.

On May 10, 2019 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on identifying, in-licensing and developing high-potential marketed and development-stage drugs and drug candidates, reported financial results and recent corporate highlights for the first quarter ended March 31, 2019 (Press release, Fortress Biotech, MAY 10, 2019, View Source [SID1234536161]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We enjoyed a strong start to 2019, with compelling data recently published in the New England Journal of Medicine pertaining to our partner company Mustang’s lentiviral gene therapy for infants under the age of two diagnosed with X-linked severe combined immunodeficiency ("XSCID"), and the closing of two promising partnership deals with Alexion Pharmaceuticals and InvaGen Pharmaceuticals (a subsidiary of Cipla Limited) in the first quarter. These significant achievements demonstrate how our business model is designed to drive value for our shareholders. We have several opportunities to increase economics and decrease risk for our shareholders with multiple possible near-term revenue streams due to our specialty pharmaceutical business; large equity stakes in our partner companies; strategic, flexible potential exits; and potential royalties on product sales."

Dr. Rosenwald continued, "We look forward to advancing our and our partner companies’ development programs and achieving numerous milestones during the remainder of 2019, including important data readouts and the potential for the first New Drug Application ("NDA") filing by a Fortress partner company later this year. Our sizable business development team continues to source and in-license high-potential marketed and development-stage programs to enhance the growing, diversified portfolio of seven commercial products and over 25 product candidates in development across our partner companies."

Financial Results:1

·As of March 31, 2019, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), and restricted cash totaled $137.5 million, compared to $99.2 million as of December 31, 2018, an increase of $38.3 million for the quarter.
·Fortress’ net revenue totaled $6.5 million for the first quarter of 2019, compared to $5.9 million for the first quarter of 2018.
·Research and development expenses were $23.3 million for the first quarter of 2019, of which $22.6 million was related to Fortress partner companies. This compares to $25.0 million for the first quarter of 2018, of which $22.8 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in research and development were $0.6 million for the first quarter of 2019, compared to $2.3 million for the first quarter of 2018.
·Research and development expenses from license acquisitions totaled $0.5 million for the first quarter of 2019, compared to $0.1 million for the first quarter of 2018.
·General and administrative expenses were $13.5 million for the first quarter of 2019, of which $8.9 million was related to Fortress partner companies. This compares to $13.5 million for the first quarter of 2018, of which $8.4 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.7 million for the first quarter of 2019, compared to $2.4 million for the first quarter of 2018.
·Net income attributable to common stockholders was $1.4 million, or $0.03 per share, for the first quarter of 2019, compared to a net loss attributable to common stockholders of $21.0 million, or $0.49 per share, for the first quarter of 2018.

Marketed Dermatology Products

·In the first quarter of 2019, our seven marketed products generated net revenue of $6.1 million, compared to $6.0 million in the fourth quarter of 2018 and $5.5 million in the first quarter of 2018.

IV Tramadol

·The stock purchase stage of the strategic transaction between InvaGen and our partner company Avenue closed in February 2019. InvaGen acquired approximately 5.8 million shares of Avenue Therapeutics’ common stock at $6.00 per share for total gross consideration of $35.0 million, representing a 33.3% stake in Avenue’s capital stock on a fully-diluted basis.
·Avenue is currently running a second pivotal Phase 3 efficacy and safety study of IV tramadol in patients with post-operative pain following abdominoplasty procedure, as well as an open-label, single-arm safety study. Both are expected to complete around mid-2019.

CAEL-101

·In January 2019, Caelum Biosciences, Inc. ("Caelum") signed an agreement with Alexion Pharmaceuticals, Inc. ("Alexion") to advance the development of CAEL-101. Under the terms of the agreement, Alexion purchased a 19.9% minority equity interest in Caelum for $30 million. Additionally, Alexion has agreed to make potential payments to Caelum upon the achievement of certain developmental milestones, in exchange for which Alexion obtained a contingent exclusive option to acquire the remaining equity in the company for pre-negotiated economics.

Triplex

·The multicenter Phase 2 study of Triplex for cytomegalovirus ("CMV") control in allogeneic stem cell transplant recipients has concluded, and its primary endpoint was met. The full dataset was presented at the 45th Annual Meeting of the European Society for Blood and Marrow Transplantation ("EBMT") in March 2019. We plan to complete an End of Phase 2 Meeting with the FDA for Triplex in the second half of 2019 and plan to initiate a Phase 3 study of Triplex in the first half of 2020.

MB-107 (XSCID gene therapy)

·In April 2019, the New England Journal of Medicine published data from St. Jude Children’s Research Hospital ("St. Jude"). The data are from a Phase 1/2 clinical trial of a lentiviral gene therapy for the treatment of newly diagnosed infants under two years old with XSCID. Data demonstrate the lentiviral gene therapy achieved normalization of T-cell numbers in all eight newly diagnosed infants with XSCID to date, and disseminated infections resolved completely in all affected infants. Seven of the eight infants treated have developed normal IgM levels to date. Four of those seven infants have discontinued monthly infusions of intravenous immunoglobulin (IVIG) therapy to date. Three of those four infants who discontinued monthly IVIG infusions have responded to vaccines to date.
·MB-107 (XSCID gene therapy) is currently in development at our partner company, Mustang Bio, Inc.

Cosibelimab (formerly referred to as CK-301, an anti-PD-L1 antibody)

·In January 2019, we announced that the ongoing multi-center clinical trial of anti-PD-L1 antibody, cosibelimab, was expanded to enroll patients in three endometrial and colorectal cohorts intended to support potential requests for accelerated approval and Biologics License Application ("BLA") submissions to the U.S. Food and Drug Administration ("FDA"). The ongoing trial is also enrolling cohorts of patients with non-small cell lung cancer ("NSCLC") and cutaneous squamous cell carcinoma.
·In May 2019, we announced positive interim results from our ongoing multicenter Phase 1 clinical trial of cosibelimab. There were >40% objective response rates observed in first-line non-small cell lung cancer and cutaneous squamous cell carcinoma and the antibody was well-tolerated.
·Cosibelimab (anti-PD-L1 antibody) is currently in development at our partner company, Checkpoint Therapeutics, Inc.

CK-101 (third-generation EGFR inhibitor)

·In March 2019, we announced two new patent issuances by the U.S. Patent and Trademark Office and the European Patent Office for CK-101. The patents cover CK-101 in the U.S. and Europe through at least August 2034, not including any potential patent term extensions.
·CK-101 (third-generation EGFR inhibitor) is currently in development at our partner company, Checkpoint Therapeutics, Inc.

MB-108 (Oncolytic Virus C134)

·In February 2019, we partnered and entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop an oncolytic virus (C134) for the treatment of glioblastoma multiforme. We intend to combine MB-108 with MB-101 (IL13Rα2-specific CAR) to potentially enhance efficacy in treating glioblastoma multiforme.
·MB-108 (oncolytic virus C134) is currently in development at our partner company, Mustang Bio, Inc.

MB-104 (CS1-specific CAR T)

·In May 2019, we announced that City of Hope began enrolling patients with relapsed or treatment-resistant multiple myeloma in an innovative CS1 chimeric antigen receptor ("CAR") T cell therapy (MB-104) trial. The Phase 1 clinical trial is the first autologous CAR T trial to target the CS1 protein, which is expressed by cancer cells in nearly all multiple myeloma patients.
·MB-104 (CS1-specific CAR T) is currently in development at our partner company, Mustang Bio, Inc.

On May 10, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that new data from clinical trials of 17 approved and investigational medicines across 27 cancer types, including hard-to-treat and rare tumours, will be presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL, United States, from 31 May– 4 June, 2019 (Press release, Hoffmann-La Roche, MAY 10, 2019, View Source [SID1234536160]). A total of 155 abstracts that include a Roche medicine will be presented at this year’s meeting.

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"At this year’s ASCO (Free ASCO Whitepaper) meeting, we are excited to present new data with targeted therapies, immunotherapy and pipeline combinations across a broad range of diseases including blood, breast and lung cancers, as well as paediatric tumours treated with our personalised cancer medicine, entrectinib," said Sandra Horning, MD, chief medical officer and head of Global Product Development. "Through pioneering science, strategic partnerships and data and analytics, we’re striving to develop transformative medicines that can help improve outcomes for each individual patient."

Further information on Roche’s contribution to the ASCO (Free ASCO Whitepaper) 2019 scientific programme, as well as the latest innovations and developments in Roche’s approach to accelerating progress in cancer care, will be featured during the Roche Media Briefing from 09:00 – 10:30 CDT on Friday 31 May at the Chicago Marriott Downtown Magnificent Mile, Chicago, IL, US. This event, independently organised by Roche, is open to journalists from outside the United States who have registered as media with the ASCO (Free ASCO Whitepaper) 2019 Annual Meeting.

To register for the Roche Media Briefing, please follow this link: http://roche.cvent.com/d/v6qcp1?ct=94fca666-18b0-4c40-b5f7-6b4b845171c7.

Keep up to date on ASCO (Free ASCO Whitepaper) meeting news and updates by following Roche on Twitter via @Roche and using the hashtag #ASCO19.

Key presentations in blood cancers
The first data from the pivotal Phase III CLL14 study will be presented at ASCO (Free ASCO Whitepaper), evaluating the 12-month, fixed-duration, chemotherapy-free combination of Venclexta/Venclyxto (venetoclax) plus Gazyva/Gazyvaro (obinutuzumab) compared to Gazyva/Gazyvaro plus chlorambucil in people with previously untreated chronic lymphocytic leukaemia (CLL) and co-existing medical conditions. The CLL14 study is being conducted in cooperation with the German CLL Study Group (GCLLSG), headed by Michael Hallek, MD, University of Cologne.

The US Food and Drug Administration (FDA) is reviewing a supplemental New Drug Application (sNDA) based on results of the CLL14 study under the FDA’s Real-Time Oncology Review and Assessment Aid pilot programmes. Venclexta is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche group, in the US and commercialised by AbbVie outside of the US.

Key presentations in paediatric cancers
The first data from the Phase I/II STARTRK-NG study of the investigational medicine entrectinib in children and adolescents with recurrent or refractory solid tumours harbouring neurotrophic tyrosine receptor kinase (NTRK), ROS1 or anaplastic lymphoma kinase (ALK)-positive tumours, including central nervous system tumours, will be presented. The study enrolled children and adolescents aged 4.9 months through 20 years (median of seven years) across several different cancer types, including some rare tumours. The STARTRK-NG data will be featured as part of ASCO (Free ASCO Whitepaper)’s official press programme on Wednesday, 15 May.

The FDA recently granted Priority Review for entrectinib for the treatment of paediatric and adult patients with NTRK fusion-positive, locally advanced or metastatic solid tumours who have either progressed following prior therapies or as initial therapy when there are no acceptable standard therapies, and for the treatment of people with metastatic, ROS1-positive non-small cell lung cancer (NSCLC). These NDAs are based on results from the integrated analysis of the pivotal Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials, and data from the STARTRK-NG study. The FDA is expected to make a decision on the approval by 18 August 2019.

Key presentations in breast cancers
Key data to be presented at ASCO (Free ASCO Whitepaper) include updates from Roche’s breast cancer programme across multiple subtypes of the disease, including the second interim analysis of overall survival (OS) results, updated safety data and patient-reported outcomes (PROs) from the Phase III IMpassion130 study of Tecentriq (atezolizumab) plus chemotherapy (Abraxane [paclitaxel protein-bound particles for injectable suspension (albumin-bound); nab-paclitaxel]) for the treatment of PD-L1-positive, metastatic triple-negative breast cancer (TNBC). This combination was recently granted accelerated approval from the FDA based on progression-free survival (PFS) for the treatment of adults with unresectable locally advanced or metastatic TNBC in people whose tumours express PD-L1, as determined by an FDA-approved test.

Additional data include an eight-year, end-of-study analysis from the Phase III CLEOPATRA study of Perjeta (pertuzumab) plus Herceptin (trastuzumab) and chemotherapy for first-line treatment of HER2-positive metastatic breast cancer.

Key presentations in lung cancers
Key data from Roche’s broad lung cancer programme will be presented across different types of the disease, including results from the Phase III IMpower150 trial of Tecentriq plus Avastin (bevacizumab) and chemotherapy (carboplatin and paclitaxel) in chemotherapy-naïve people previously untreated for metastatic NSCLC whose cancer has spread to the liver, which affects approximately 20% of people with the disease. Additionally, results from studies in partnership with Flatiron Health will be presented, including validation of the use of next-generation sequencing data on a broad scale to improve the understanding of clinical outcomes in people with metastatic lung cancer, and results that illustrate how real-world data can be used to supplement evidence from clinical trials in rare tumour types such as ROS1-positive lung cancer.

Key presentations featuring Roche medicines at ASCO (Free ASCO Whitepaper) 2019

About Roche in Oncology
Roche has been working to transform cancer care for more than 50 years, bringing the first specifically designed anti-cancer chemotherapy drug, fluorouracil, to patients in 1962. Roche’s commitment to developing innovative medicines and diagnostics for cancers remains steadfast.

The Roche Group’s portfolio of innovative cancer medicines includes: Alecensa (alectinib); Avastin (bevacizumab); Cotellic (cobimetinib); Erivedge (vismodegib); Gazyva/Gazyvaro (obinutuzumab); Herceptin (trastuzumab); Kadcyla (trastuzumab emtansine); MabThera/Rituxan (rituximab); Perjeta (pertuzumab); Tarceva (erlotinib); Tecentriq (atezolizumab); Venclexta/VenclyxtoTM (venetoclax); Xeloda (capecitabine); Zelboraf (vemurafenib). Furthermore, the Roche Group has a robust investigational oncology pipeline focusing on new therapeutic targets and novel combination strategies.

For more information on Roche’s approach to cancer, visit www.roche.com.

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.

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.