On August 9, 2018 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 that the Company will present on Tuesday, August 14th at 1:20 p.m. ET at the 2018 Wedbush PacGrow Healthcare Conference in New York (Press release, argenx, AUG 9, 2018, View Source;p=RssLanding&cat=news&id=2363190 [SID1234528607]).

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A live webcast of the presentation will be available on the Company’s website at www.argenx.com. A replay of the webcast will be available for 90 days following the presentation.

On August 6, 2018, PRA Health Sciences, Inc. (the "Company") entered into an Underwriting Agreement (the "Underwriting Agreement") by and among the Company, the selling stockholder named therein (the "Selling Stockholder"), and Morgan Stanley & Co. LLC and Goldman Sachs & Co. LLC (the "Underwriters"), relating to an underwritten offering (the "Offering") of 6,500,000 shares (the "Shares") of the Company’s common stock, par value $0.01 per share, pursuant to the Company’s Registration Statement on Form S-3 (File No. 333-209883), filed on March 2, 2016, as supplemented by the prospectus supplement dated August 6, 2018 (Filing, 8-K, PRA Health Sciences, AUG 9, 2018, View Source [SID1234528791]). All of the Shares are being sold by the Selling Stockholder. Pursuant to the Underwriting Agreement, the Underwriters purchased the Shares at a price of $101.01 per share in a transaction that was completed on August 9, 2018.

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On August 9, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported second quarter 2018 financial results (Press release, , AUG 9, 2018, View Source [SID1234528569]).

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"We made significant progress across our pipeline in the second quarter," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "In May, our NDA for larotrectinib was accepted by FDA and granted Priority Review. In June, initial clinical data from the ongoing LIBRETTO-001 Phase 1/2 study of LOXO-292 were presented at ASCO (Free ASCO Whitepaper). These data enabled a subsequent FDA meeting, which established a path forward for the program. And lastly, we completed important enabling studies and manufacturing activities for the LOXO-305 IND. In the second half of the year, we are focused on continuing to advance our pipeline, as well as aiding and advising our partners at Bayer in anticipation of a possible larotrectinib regulatory approval. Operationally, we aim to lay the groundwork for the long-term adoption of tumor genomic testing capable of detecting TRK fusions alongside other clinically actionable alterations."

LOXO-292 Regulatory Update

Loxo Oncology recently conducted a meeting with the U.S. Food and Drug Administration (FDA) for LOXO-292. Based on written minutes from FDA, Loxo Oncology expects to submit a new drug application (NDA) for LOXO-292 in late 2019, utilizing data generated from the ongoing LIBRETTO-001 clinical trial. This timeline integrates standard NDA activities which are ongoing and planned, including clinical pharmacology studies, non-clinical studies, and manufacturing. Loxo Oncology expects to file for separate potential indications for two populations of patients: those with RET fusion-positive solid tumors such as lung and thyroid cancer, and those with RET-mutant medullary thyroid cancer (MTC). In both cases, Loxo Oncology expects that patients will be required to have received systemic therapy, progressed following prior treatment and have no satisfactory alternative treatment options.

Recent Highlights

Larotrectinib

Abstract Accepted for Oral Presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress: Clinical data for larotrectinib will be presented at the ESMO (Free ESMO Whitepaper) 2018 Congress to be held October 19-23, 2018, in Munich, Germany. The presentation will provide updated clinical follow-up for the 55 patients who comprise the primary efficacy analysis population that has supported global regulatory filings. The presentation will also include new data for TRK fusion patients subsequently enrolled.
Abstract Accepted for Poster Presentation at the Molecular Analysis for Personalised Therapy 2018 Congress: On September 15, 2018, Ventana Medical Systems, Inc., a member of the Roche Group, and Loxo Oncology, will present a co-authored poster on the analytical validation of Ventana’s pan-TRK IHC assay at the Molecular Analysis for Personalised Therapy 2018 Congress, to be held September 14-15, 2018 in Paris, France.
Acceptance of NDA by FDA: On May 29, 2018, Loxo Oncology announced FDA acceptance of the larotrectinib NDA and granting of Priority Review for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion. The FDA has set a target action date of November 26, 2018, under the Prescription Drug User Fee Act (PDUFA). More information can be found here.
International Symposium on Pediatric Neuro-Oncology (ISPNO) Poster Presentation: On July 1, 2018, a poster presentation at ISPNO 2018 detailed a case report of a pediatric patient with TRK fusion high-grade glioma treated with larotrectinib on a single patient protocol. The poster can be found here.
World Congress on Gastrointestinal Cancer Oral Presentation: On June 22, 2018, an oral presentation at the ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer detailed larotrectinib data in patients with TRK fusion gastrointestinal (GI) cancers enrolled to the pivotal larotrectinib program. The presentation can be found here.
Journal of Clinical Investigation Publication: On June 19, 2018, an article was published online in the peer-reviewed Journal of Clinical Investigation detailing the occurrence of TRK fusions in hematologic malignancies, including a case report of a patient with TRK fusion acute myeloid leukemia (AML) treated with larotrectinib on a single patient protocol. The publication can be found here.
Pediatric Blood & Cancer Publication: On June 12, 2018, a case report was published in the online edition of the peer-reviewed journal Pediatric Blood & Cancer detailing a pediatric patient with metastatic TRK fusion congenital mesoblastic nephroma (CMN) treated with larotrectinib on the SCOUT trial. The publication can be found here.
LOXO-292

Interim Clinical Data Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting: In June, interim clinical data from the LOXO-292 global LIBRETTO-001 trial were presented in an oral presentation at ASCO (Free ASCO Whitepaper). The presentation included 82 patients enrolled across eight dose escalation cohorts and employed an April 2, 2018 data cut-off. The data demonstrated a 77 percent (95% CI: 61-89%) overall response rate (ORR) in RET fusion cancers and a 45 percent (95% CI: 24-68%) ORR in RET mutated MTC as evaluated by RECIST 1.1 criteria. Patients had received a median of three prior treatments with two-thirds having been treated with at least one prior multi-kinase inhibitor. All responding patients across all tumor types remained on therapy as of the data cut-off. LOXO-292 was well tolerated with most treatment-emergent adverse events Grade 1 in severity. The treatment-emergent adverse events observed in ≥10% of patients, regardless of relationship to LOXO-292, were fatigue, diarrhea, constipation, dry mouth, nausea, and dyspnea. Phase 2 cohorts are open and enrolling at the 160mg BID dose. See the presented data here.
LOXO-305

Presentation Accepted at the Society of Hematologic Oncology (SOHO) Annual Meeting: In September 2018, Loxo Oncology authors will present LOXO-305 preclinical characterization data in oral and poster presentations at the SOHO Annual Meeting taking place September 12-15 in Houston, Texas.
Upcoming Milestones

Larotrectinib (TRK)
Presentation of updated clinical data at the ESMO (Free ESMO Whitepaper) Congress
Submission of a Marketing Authorisation Application in the European Union, by Bayer, is expected in the second half of 2018
NDA PDUFA date of November 26, 2018
LOXO-195 (next-generation TRK)
Updated clinical data is now expected in the first half of 2019
LOXO-292 (RET)
Updated clinical data is expected in the second half of 2018
LOXO-305 (BTK)
Presentation of preclinical data at the SOHO Annual Meeting
Initiation of a Phase 1 clinical trial is expected in the fourth quarter of 2018
Second Quarter 2018 Financial Results

As of June 30, 2018, Loxo Oncology had aggregate cash, cash equivalents and investments of $706.4 million, compared to $626.2 million as of December 31, 2017.

Revenue from the collaboration agreement was $42.6 million for the second quarter of 2018, compared to none for the second quarter of 2017. This represents $51.2 million in revenue recognized from the $400 million upfront payment from the Bayer collaboration offset by $8.6 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs.

Revenue from the collaboration agreement was $81.0 million for the six months ended June 30, 2018, compared to none for the six months ended June 30, 2017. This represents $94.1 million in revenue recognized from the $400 million upfront payment from the Bayer collaboration offset by $13.1 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs. Loxo Oncology recognizes revenue from the upfront payment on a proportional performance basis utilizing a calculation based on quarterly research and development spending associated with larotrectinib and LOXO-195, relative to cumulative and forecasted research and development spending on larotrectinib and LOXO-195 over the course of the collaboration agreement. As a result, the quarterly revenue recognized for the upfront payment varies from quarter to quarter. A supporting schedule that shows the different components of revenue from the collaboration agreement is included with the attached financial statements.

Research and development expenses were $41.6 million for the second quarter of 2018 compared to $24.4 million for the second quarter of 2017. This increase was primarily due to expanded development expenses across our LOXO-292 and LOXO-305 programs and higher employment costs primarily due to increased headcount. These numbers are net of 50/50 cost-sharing with Bayer for larotrectinib and LOXO-195 development costs. Loxo Oncology recognized research and development-related stock-based compensation expense of $5.8 million during the second quarter of 2018 as compared to $3.5 million for the second quarter of 2017.

Research and development expenses were $73.5 million for the six months ended June 30, 2018 compared to $44.6 million for the six months ended June 30, 2017. This increase was primarily due to expanded development expenses across our LOXO-292 and LOXO-305 programs and higher employment costs primarily due to increased headcount. These numbers are net of 50/50 cost-sharing with Bayer for larotrectinib and LOXO-195 development costs. Loxo Oncology recognized research and development-related stock-based compensation expense of $10.1 million during the six months ended June 30, 2018 as compared to $5.9 million for the six months ended June 30, 2017.

General and administrative expenses were $15.7 million for the second quarter of 2018 compared to $6.5 million for the second quarter of 2017. The increase was primarily due to additional headcount and associated employment costs and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $6.5 million during the second quarter of 2018 compared to $2.0 million for the second quarter of 2017.

General and administrative expenses were $27.9 million for the six months ended June 30, 2018 compared to $11.3 million for the six months ended June 30, 2017. The increase was primarily due to additional headcount and associated employment costs and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $11.9 million during the six months ended June 30, 2018 compared to $3.5 million for the six months ended June 30, 2017.

Net loss was $11.7 million and $15.3 million for the three and six months ended June 30, 2018, respectively, compared to $30.4 million and $54.9 million for the three and six months ended June 30, 2017, respectively. This decrease in net loss is primarily driven by the revenue recognized from the $400.0 million upfront payment from the Bayer collaboration, the larotrectinib and LOXO-195 development reimbursement from the Bayer collaboration, offset by increases in operating expenses.

Non-GAAP net loss was $50.6 million and $87.4 million for the three and six months ended June 30, 2018, respectively, compared to $25.0 million and $45.5 million for the three and six months ended June 30, 2017, respectively. This non-GAAP net loss measure, more fully described below under "Non-GAAP Financial Measures," excludes the recognition of collaboration revenue related to the Bayer upfront payment and share-based compensation expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

Earnings Conference Call and Webcast Information
Loxo Oncology will host a conference call today at 8:00 a.m. ET to discuss the second quarter 2018 financial results and company updates. A live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the company’s website at www.loxooncology.com. The conference call can be accessed by dialing (877) 930-8065 (domestic) or (253) 336-8041 (international) and referring to conference ID 7291605. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event.

On August 9, 2018 SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company delivering innovative therapies for difficult-to-treat and often life-threatening infections, reported financial results for the quarter ended June 30, 2018, and provided an update on recent operational and clinical developments (Press release, Scynexis, AUG 9, 2018, View Source [SID1234528587]).

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"With the positive results from our Phase 2b DOVE study in vulvovaginal candidiasis (VVC), we achieved a meaningful milestone in our efforts to make ibrexafungerp available to patients in need and to bring to market the first new antifungal class in nearly 20 years," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "While we continue to face challenges with the development of our IV formulation, we are pleased with the advancement and further de-risking of oral ibrexafungerp, which has the potential to address unmet medical needs in a variety of severe infections and improve the lives of patients."

Ibrexafungerp (formerly SCY-078) Update

600mg Oral Dose of Ibrexafungerp Identified for Phase 3 VVC Development; Phase 3 On Track for Initiation in the Fourth Quarter of 2018, with Potential NDA Filing in 2020.
In July 2018, SCYNEXIS reported positive top-line data from the Phase 2b DOVE study evaluating several dose regimens of oral ibrexafungerp for the treatment of VVC, compared to fluconazole (FLU), the standard of care for VVC. The 600mg dose of ibrexafungerp was well-tolerated, with strong overall clinical and mycological activity and improved sustained effect compared to FLU.
Pending the End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), we believe the 600mg dose of ibrexafungerp for one day (given as two doses of 300mg 12 hours apart) is the optimal dose regimen. We intend to use this dose in the VVC Phase 3 registration program, on track for initiation in the fourth quarter of 2018, and, pending successful completion of this trial, we anticipate filing an initial NDA for the treatment of VVC in 2020. Ibrexafungerp may represent the first new oral treatment for this indication in 25 years, and we believe it may provide significant benefits for the large number of patients not well-served by existing therapies.
Development Delay with IV Formulation of Ibrexafungerp. In January 2018, we announced encouraging pre-clinical results for the prototype liposomal IV formulation of ibrexafungerp, showing improved local tolerability profile at the infusion site in head-to-head pre-clinical evaluations with the cyclodextrin-based IV formulation. As part of our development plans, the process for the liposomal formulation was transferred for scale-up purposes at a manufacturing site intended to provide clinical supplies. Additional preclinical evaluations were performed with the scaled-up formulation, which unexpectedly revealed differences in tolerability at the injection site, delaying advancement of the IV product into human trials. As it is generally recognized that changes to manufacturing processes and/or scale-up can impact the characteristics of drug products, particularly for more technically complex formulations such as liposomal products, we are currently working with our vendors and CMC experts to enable us to resume the pre-IND pre-clinical activities for the IV formulation of ibrexafungerp.
Continued Progress on Company’s Strategy to Expand the Use of Ibrexafungerp in Indications Where the Oral Formulation is a Viable Treatment Option to Address Significant Unmet Needs.
Initiation of Phase 2 Combination Study Testing Oral Ibrexafungerp in Invasive Aspergillosis on Track for this Quarter. Encouraged by the improved outcomes and survival rates seen in an animal model of pulmonary aspergillosis, we plan to start a study in patients with invasive aspergillosis (IA) in the third quarter of 2018. The study will assess the safety and efficacy of oral ibrexafungerp in combination with azole therapy, the standard of care for this indication.
Open-label FURI and CARES Studies Ongoing with Preliminary Data Review Planned for the Fourth Quarter of 2018. These studies are designed to enroll patients with a wide range of Candida spp. infections with limited or no treatment options and are designed for potential expedited regulatory approval via the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD).
SCY-078 Granted "Ibrexafungerp" as Non-proprietary Name by WHO. In assigning ibrexafungerp as the generic name of SCY-078, the World Health Organization (WHO) incorporated a novel stem (-fungerp), recognizing ibrexafungerp as the first member of a new triterpenoid drug class. With the last new antifungal class approved in 2001, ibrexafungerp has the potential to impact the rapidly rising resistance rates observed in many fungal species to today’s standards of care.
Oral Ibrexafungerp Granted QIDP and Fast Track Designations for the Treatment of VVC and the Prevention of VVC Recurrence. The Qualified Infectious Disease Product (QIDP) designation allows for priority review and an additional five years of market exclusivity in the U.S. The FDA’s Fast Track Drug Development Program is designed to facilitate the development and expeditious review of drugs to treat serious conditions and fill unmet medical needs.
Presentations at Medical Meetings and Peer-reviewed Publications. SCYNEXIS presented ibrexafungerp data at medical meetings and published data in a peer-reviewed journal. The data demonstrate the potent antifungal activity of ibrexafungerp against a broad range of fungal species, including echinocandin-resistant strains, as well as highlight numerous unique attributes, including lack of teratogenicity, synergistic activity with azoles and low risk of interactions with agents metabolized by CYP enzymes. Collectively, the data support the investigation of ibrexafungerp across a spectrum of antifungal indications, including VVC, invasive candidiasis (IC), IA, resistant fungal infections and prophylaxis indications.
In July 2018, at the 20th Congress of the International Society for Human and Animal Mycology, SCYNEXIS presented pre-clinical data demonstrating that ibrexafungerp has fungicidal activity against C. auris.
In June 2018, at the Teratology Society 58th Annual Meeting, SCYNEXIS presented pre-clinical data demonstrating that ibrexafungerp shows no impact on fertility or early embryo/fetal activity, potentially clinically relevant for women who are pregnant or may become pregnant.
In June 2018, at the 20th International Symposium on Infections in the Immunocompromised Host, SCYNEXIS presented pre-clinical data demonstrating that ibrexafungerp has activity alone, as well as synergistic activity with azole agents, against Aspergillus spp.
In June 2018, at the American Society for Microbiology Microbe 2018, SCYNEXIS presented data demonstrating that ibrexafungerp has potent in vitro activity against echinocandin-resistant Candida, as well as exhibits synergistic activity with isavuconazole against Aspergillus spp.
In June 2018, in The Journal of Clinical Pharmacology, SCYNEXIS published Phase 1 data demonstrating that ibrexafungerp has a low risk of interactions with drugs metabolized by CYP enzymes, potentially clinically relevant for patients with Type 2 diabetes.
In April 2018, at the 28th European Congress of Clinical Microbiology and Infectious Diseases, SCYNEXIS presented data demonstrating the in vitro activity of ibrexafungerp against Aspergillus spp., as well as the in vivo activity of ibrexafungerp against Pneumocystis pneumonia, supporting potential use for prophylaxis indications.
Second Quarter 2018 Financial Results

Cash, cash equivalents and short-term investments totaled $55.2 million as of June 30, 2018, with net working capital of $43.4 million. SCYNEXIS expects its cash, cash equivalents and short-term investments at June 30, 2018 will be sufficient to fund operations into 2020.

Research and development, net expenses increased to $5.6 million in the second quarter of 2018, compared to $4.4 million in the second quarter of 2017. The increase of $1.2 million, or 26%, for the three months ended June 30, 2018, was primarily driven by an increase of $0.8 million in pre-clinical development expense and a $1.0 million increase in clinical development expense; offset by a decrease in regulatory expense of $0.2 million and a decrease of $0.4 million in consulting expense.

Selling, general and administrative expenses decreased to $2.1 million in the second quarter of 2018, compared to $2.4 million in the second quarter of 2017. The decrease of $0.2 million, or 10%, for the three months ended June 30, 2018, was primarily driven by a decrease in business development related expenses and legal fees incurred during the three months ended June 30, 2018.

Total other expense decreased to $3.1 million in the second quarter of 2018 due to a $2.9 million non-cash loss recorded on the fair value adjustment of the warrant liabilities.

Net loss for the second quarter of 2018 was $10.8 million, or $0.23 per share. This compares with a net loss for the second quarter of 2017 of $4.2 million, or $0.16 per share.

About Ibrexafungerp (formerly SCY-078)

Ibrexafungerp [pronounced eye-BREX-ah-FUN-jerp] is an investigational antifungal agent and the first representative of a novel class of structurally-distinct glucan synthase inhibitors, triterpenoids. This agent combines the well-established activity of glucan synthase inhibitors with the potential flexibility of having oral and IV formulations. Ibrexafungerp is currently in development for the treatment of fungal infections caused primarily by Candida (including C. auris) and Aspergillus species. It has demonstrated broad spectrum antifungal activity, in vitro and in vivo, against multidrug-resistant pathogens, including azole- and echinocandin-resistant strains. The FDA has granted QIDP and Fast Track designations for the formulations of ibrexafungerp for the indications of IC (including candidemia), IA and VVC, and has granted Orphan Drug Designation for the IC and IA indications.

On August 9, 2018 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the second quarter ended June 30, 2018 (Press release, Fortress Biotech, AUG 9, 2018, View Source [SID1234528835]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "During the second quarter, our Fortress Company subsidiaries reported significant value-driving milestones, including positive Phase 3 data from Avenue Therapeutics’ IV tramadol, which, if approved, would be the only Schedule IV intravenous opioid in the U.S. and could replace highly addictive Schedule II narcotics in many patients with moderate to moderately severe postoperative pain. Additionally, Cyprium Therapeutics was granted FDA Fast Track Designation for its CUTX-101 Copper Histidinate injection in patients with Menkes disease, a rare pediatric disease with no FDA-approved treatments. Also during the quarter, Mustang Bio expanded its infrastructure with the launch of a proprietary 27,000 sq. ft. CAR T cell manufacturing facility that will enable us to oversee product safety from needle-to-needle and help improve supply chain efficiencies from clinical development into commercialization."

Dr. Rosenwald continued, "We believe in the value proposition represented by our company and strive to protect the best interests of our shareholders and those of our subsidiaries. As we continue to build long-term value, our novel and efficient business model provides benefits for all stakeholders and offers unique synergies not typical of traditional biopharma companies."

Financial Results:

·As of June 30, 2018, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), cash deposits with clearing organizations and restricted cash totaled $151.8 million, compared to $168.3 million as of December 31, 2017, a decrease of $16.5 million year-to-date.
·Net revenue totaled $63.8 million for the second quarter of 2018, compared to $50.7 million for the second quarter of 2017. Total revenue as of June 30, 2018, includes $6.8 million of Fortress revenue, primarily from the sale of Journey Medical Corporation products, and $57.0 million of revenue from National Holdings Corporation1 ("National Holdings"). Total revenue as of June 30, 2017, included $4.4 million of Fortress revenue and $46.3 million of revenue from National Holdings.
·Research and development expenses were $17.5 million for the second quarter of 2018, of which $15.1 million was related to Fortress Companies. This compares to $11.7 million for the second quarter of 2017, of which $9.5 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in research and development were $0.8 million for the second quarter of 2018, compared to $2.4 million for the second quarter of 2017.
Research and development expenses from license acquisitions were nominal for the second quarter of 2018, compared to $1.8 million for the second quarter of 2017.
·General and administrative expenses were $13.1 million for the second quarter of 2018, of which $7.7 million was related to Fortress Companies. This compares to $11.1 million for the second quarter of 2017, of which $6.6 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.4 million for the second quarter of 2018, compared to $2.2 million for the second quarter of 2017.
·National Holdings’ operating expenses totaled $56.2 million for the second quarter of 2018, compared to $48.4 million for the second quarter of 2017.
·Net loss attributable to common stockholders was $21.6 million, or $0.50 per share, for the second quarter of 2018, compared to a net loss attributable to common stockholders of $17.4 million, or $0.43 per share, for the second quarter of 2017. For the first six months of 2018, net loss was $42.6 million or $0.99 per share, compared to $29.3 million or $0.73 per share in the first six months of 2017.

1Fortress acquired approximately 56 percent of National Holdings in September 2016.

Recent Fortress and Fortress Company Highlights:

Fortress Biotech, Inc.

·In June 2018, data from a Phase 1 trial evaluating Fortress’ CNDO-109-activated allogeneic natural killer (NK) cells in acute myeloid leukemia (AML) patients were published in the journal Biology of Blood and Marrow Transplantation. The data demonstrated that CNDO-109-activated NK cells are safe, well tolerated and may be capable of extending complete remissions in high-risk AML patients.

Aevitas Therapeutics, Inc.

·In August 2018, Aevitas announced that it entered a sponsored research agreement with the laboratory of Wenchao Song, Ph.D., at the University of Pennsylvania to evaluate Aevitas’ adeno-associated virus ("AAV") gene therapy technology in Penn’s proprietary animal models of complement-mediated diseases.

Avenue Therapeutics, Inc.

·In May 2018, Avenue announced that its first pivotal Phase 3 trial of IV tramadol achieved the primary endpoint of a statistically significant improvement in Sum of Pain Intensity Difference over 48 hours (SPID48) compared to placebo in patients with moderate to moderately severe postoperative pain following bunionectomy surgery. In addition, the trial met its key secondary endpoints and demonstrated a clear dose response. Avenue plans to initiate a second pivotal Phase 3 trial of IV tramadol in patients following abdominoplasty surgery in the second half of 2018.

Caelum Biosciences, Inc.

·In June 2018, Caelum announced a complete analysis of cardiac data from a Phase 1b trial of CAEL-101 (mAb 11-1F4) for the treatment of relapsed or refractory amyloid light chain ("AL") amyloidosis demonstrating CAEL-101’s potential to improve myocardial function as assessed by global longitudinal strain and generate a sustained decrease in N-terminal pro-brain natriuretic peptide levels in AL amyloidosis patients experiencing cardiac involvement. The data were presented by Columbia University at the American Society of Echocardiography 29th Annual Scientific Sessions.

Checkpoint Therapeutics, Inc.

·In April 2018, preclinical data was presented on Checkpoint’s BET inhibitor, CK-103, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. CK-103 demonstrated combinatorial effects in an in vivo model with anti-PD-1 antibodies, which may support the development of CK-103 as an anti-cancer agent alone and in combination with Checkpoint’s anti-PD-L1 antibody CK-301.

Cyprium Therapeutics, Inc.

·In July 2018, Cyprium announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to CUTX-101, a product candidate for patients diagnosed with classic Menkes disease who have not demonstrated significant clinical progression.

Mustang Bio, Inc.

·In May 2018, Mustang announced the publication of preclinical data in JCI Insight demonstrating that glioblastoma-targeted CD4+ CAR T cells mediate superior antitumor activity over CD8+ CAR T cells. The data, published by research partner City of Hope, will be applied in the ongoing Phase 1 trial of Mustang’s IL13Rα2-specific CAR T MB-101 in glioblastoma.
·In June 2018, Mustang opened a proprietary CAR T cell therapy manufacturing facility at UMass Medicine Science Park in Worcester, Mass. The facility will support the clinical development and commercialization of Mustang’s CAR T product candidates and enable proprietary cell therapy research.
·Also in June 2018, Mustang was added to the Russell 2000, 3000 and Microcap Indexes.
·In July 2018, Mustang completed a pre-Investigational New Drug (pre-IND) meeting with the FDA for MB-102 (CD123 CAR T). Based on the meeting, Mustang expects to file an IND in the fourth quarter of 2018 to support a Phase 1/2 trial of MB-102 in AML, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome.