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.

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 INSYS Therapeutics, Inc. (NASDAQ: INSY), a leader in the development, manufacture and commercialization of pharmaceutical cannabinoids and spray technology, reported financial results for its first quarter ended Mar. 31, 2019 (Press release, Insys Therapeutics, MAY 10, 2019, View Source [SID1234536195]).

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RECENT HIGHLIGHTS

Appointed Andy Long as chief executive officer; in parallel promoted Andrece Housley as chief financial officer; and Dr. Venkat Goskonda as chief scientific officer
Continued advancement of strategic alternatives for opioid-related assets
Furthered discussions on capital planning and the evaluation of strategic alternatives with Lazard (See Liquidity Update below)
Progressed R&D programs with a $10.5 million investment in the first quarter of 2019:
Submitted NDA for naloxone nasal spray
Received guidance from FDA following end of Phase 2 meeting for epinephrine nasal spray
Completed enrollment of second cohort in childhood absence epilepsy Phase 2 study
Active enrollment in the Company-sponsored CBD Prader-Willi syndrome (Phase 2) trial
Company-sponsored CBD Infantile Spasms Phase 3 study terminated; the closure of this trial is unrelated to efficacy or safety but was due to challenges related to recruitment of patientsPresented poster on proprietary epinephrine nasal spray at American Academy of Allergy, Asthma & Immunology Annual Meeting
Awarded National Institute on Drug Abuse (NIDA) grant for a series of clinical studies designed to evaluate the effects of pharmaceutical-grade cannabidiol on craving and relapse prevention in opioid use disorder
Financial Highlights

Net revenue for the first quarter of 2019 was $7.6 million, compared to $23.9 million for the first quarter of 2018, driven primarily by declines in the TIRF market and a $3.9 million reduction of inventory in the channel
Gross margin was 40.0 percent for the first quarter of 2019, compared to 90.8 percent in the same period of 2018 due to write-off of excess inventory
Sales and marketing investment was $4.1 million for the first quarter of 2019, compared to $9.1 million for the first quarter of 2018 as a result of managing commercial resources in line with market conditions
Research and development investment decreased to $10.5 million for the first quarter of 2019, compared to $12.3 million for the first quarter of 2018 due to fluctuations in the timing of clinical trials
General and administrative expense of $11.0 million for the first quarter of 2019 increased as compared to $9.6 million in the first quarter of 2018 as a result of professional advisory fees
Legal expense increased to $25.7 million for the first quarter of 2019, compared to $10.3 million in the first quarter of 2018, as a result of the company’s legal proceedings, including expenses associated with indemnification of John Kapoor in connection with his trial, which represented $18.1 million of the first quarter 2019 expense. Management is disputing the reasonableness of certain indemnification-related expenses for this quarter and prior periodsThe company accrued $73.9 million for potential contingent losses related to outstanding legal matters in the first quarter of 2019 compared to $0.7 million in the first quarter of 2018
Income tax expense of $1.2 million for the first quarter of 2019 compared to an expense of $0.2 million during the first quarter of 2018
Net loss for the first quarter of 2019 was ($123.8 million), or ($1.66) per basic and diluted share, compared to a net loss of ($20.4 million), or ($0.28) per basic and diluted share, for the first quarter of 2018. Adjusted net loss for the first quarter of 2019 was ($0.55) per basic and diluted shareAdjusted EBITDA loss for the first quarter of 2019 was ($44.1 million), compared to Adjusted EBITDA loss of ($14.9 million) in the prior-year quarter. The reconciliation of net income to Adjusted EBITDA is included at the end of this news release
The company had $87.6 million in cash, cash equivalents and short-term and long-term investments with no debt outstanding as of Mar. 31, 2019
Liquidity Update

As further discussed in our Form 10-Q for the period ended Mar. 31, 2019 (the "Form 10-Q"), while the company has no outstanding debt, available liquidity is limited to $87.6 million in cash and cash equivalents and investments as of Mar. 31, 2019, and the company expects to have continued negative cash flows from operating activities. The company has experienced recurring and increasing losses from operations over the previous 18 months due to significant declines in the TIRF market and significant legal expenses resulting from the investigation by the U.S. Department of Justice ("DOJ") and other significant litigation matters to which we are subject. As reported in the Form 10-Q, we have estimated liabilities of approximately $240.3 million as of Mar. 31, 2019 for proposed settlements of our various litigation matters, and there are other matters for which we have not been able to determine a reasonable estimated loss. Furthermore, we are uncertain if we will be able to complete a final settlement with the DOJ because of the Company’s inability to fulfill demands made by the DOJ, including the execution of a security agreement related to the assets of the company to collateralize payments under the settlement. These factors raise substantial doubt about the company’s ability to continue as a going concern within one year of the issuance date of the unaudited condensed consolidated financial statements.

If we are unable to continue as a going concern, we may have to liquidate our assets and may receive less than the value at which those assets are carried on our audited consolidated financial statements, and it is likely that investors will lose all or a part of their investment.

Management’s plans, in order to meet our operating cash flow requirements, include the pursuit of strategic alternatives related to the sale or licensing of the Company’s assets. As previously disclosed, on November 5, 2018, the company announced a process to review strategic alternatives for its portfolio of opioid-related assets, including SUBSYS, as well as formulations of buprenorphine and the combination of buprenorphine/naloxone. There are no assurances that the company will be successful in implementing a strategic plan for the sale of its assets in order to address its impending liquidity constraints. If the company cannot successfully implement its strategic plan for the sale of its assets, and/or reach an agreement with the DOJ, its liquidity, financial condition and business prospects will be materially and adversely affected. Accordingly, it may be necessary for the company to file a voluntary petition for relief under Chapter 11 of the United States Bankruptcy Code in order to implement a restructuring. Therefore, trading in our securities is highly speculative. Our Board of Directors has not made any decisions related to any strategic alternatives at this time.

On May 10, 2019 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the first quarter ended March 31, 2019 (Press release, Mustang Bio, MAY 10, 2019, View Source [SID1234536171]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The beginning of 2019 has been an exciting time for Mustang and provides great momentum for the rest of the year. Last month, we were thrilled to announce positive Phase 1/2 data published by our partner, St. Jude Children’s Research Hospital (St. Jude), in the New England Journal of Medicine, regarding the curative potential of MB-107, a lentiviral gene therapy for infants under the age of two years old with X-linked severe combined immunodeficiency, otherwise known as XSCID. Additionally, earlier this year, we partnered and entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop MB-108, oncolytic virus C134, for the treatment of glioblastoma multiforme. Most recently, Mustang announced the initiation of City of Hope’s CS1 chimeric antigen receptor (CAR) T cell therapy trial, MB-104. The Phase 1 clinical trial has begun enrolling patients and is the first autologous CAR T trial to target the CS1 protein, which is expressed by cancer cells in nearly all multiple myeloma patients."

Dr. Litchman continued, "We are also pleased to have raised a total of $69 million so far in 2019, bringing our total post-offering cash to approximately $89 million. This financial runway enables us to continue to progress the development of our eight gene and CAR T cell therapy product candidates. We anticipate achieving more exciting milestones in the coming months, including transferring the MB-107 IND from St. Jude to Mustang, filing Mustang’s first INDs for its CD123 and CS1 CAR T programs, and potentially reporting additional CAR T data in the fourth quarter."

Financial Results:

·As of March 31, 2019, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $41.1 million, compared to $34.6 million as of December 31, 2018, an increase of $6.5 million for the quarter.
·Research and development expenses were $7.0 million for the first quarter of 2019, compared to $4.3 million for the first quarter of 2018. Non-cash, stock-based compensation expenses included in research and development were $0.1 million for first quarter of 2019, compared to $1.5 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 $2.3 million for the first quarter of 2019, compared to $2.1 million for the first quarter of 2018. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.7 million for the first quarter of 2019, compared to $0.5 million for the first quarter of 2018.
·Net loss attributable to common stockholders was $9.6 million, or $0.34 per share, for the first quarter of 2019, compared to $6.3 million, or $0.24 per share, for the first quarter of 2018.

Recent Corporate Highlights:

·In February 2019, Mustang announced that it partnered and entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop MB-108, an oncolytic virus (C134), for the treatment of glioblastoma multiforme. Mustang intends to combine MB-108 with MB-101 (IL13Rα2-specific CAR) to potentially enhance efficacy in treating glioblastoma multiforme.
·In April 2019, Mustang announced that it had entered into a $20 million debt financing agreement with Horizon Technology Finance Corporation. Fifteen million of the $20 million loan was funded upon closing. The remaining $5 million may be funded upon Mustang achieving certain predetermined milestones. In connection with the debt financing, Mustang issued Horizon warrants to purchase up to 288,184 shares of its common stock at an exercise price of $3.47 per share.
·Also in April 2019, the New England Journal of Medicine published St. Jude data 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.
·In May 2019, Mustang completed an underwritten public offering, including a full over-allotment option exercise, that raised gross proceeds of $31.6 million, excluding underwriting discounts, commissions and other offering-related expenses.
·Also in May 2019, Mustang announced that City of Hope had begun enrolling patients with relapsed or treatment-resistant multiple myeloma in an innovative CS1 CAR T cell therapy (MB-104) trial.