On March 13, 2020, Aclaris Therapeutics, Inc. (the "Company") reported tha it has entered into an Open Market Sale Agreement (the "Agreement") with Jefferies LLC ("Jefferies") under which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock, par value $0.00001 per share (the "Common Stock"), having an aggregate offering price of up to $25,000,000 through Jefferies as its sales agent (Filing, 8-K, Aclaris Therapeutics, MAR 13, 2020, View Source [SID1234555548]). The issuance and sale, if any, of Common Stock by the Company under the Agreement is subject to the effectiveness of the Company’s registration statement on Form S-3, to be filed with the Securities and Exchange Commission on March 13, 2020. The Company makes no assurances as to if or whether the registration statement will become effective or, if it does become effective, as to the continued effectiveness of the registration statement.

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Jefferies may sell the Common Stock by any method permitted by law deemed to be an "at the market offering" as defined in Rule 415 of the Securities Act of 1933, as amended. Jefferies will use commercially reasonable efforts to sell the Common Stock from time to time, based upon instructions from the Company (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company will pay Jefferies a commission equal to three percent (3.0%) of the gross sales proceeds of any Common Stock sold through Jefferies under the Agreement. The Company has provided customary representations, warranties and covenants and the parties have agreed to customary indemnification rights.

The Company is not obligated to make any sales of Common Stock under the Agreement. The offering of shares of Common Stock pursuant to the Agreement will terminate upon the earlier of (i) the sale of all Common Stock subject to the Agreement or (ii) termination of the Agreement in accordance with its terms.

The foregoing description of the Agreement is not complete and is qualified in its entirety by reference to the full text of the Agreement, a copy of which is filed herewith as Exhibit 10.1 to this Current Report on Form 8-K and is incorporated herein by reference.

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any offer, solicitation, or sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.

On March 13, 2020 Moleculin Biotech Presented the Corporate Presentation (Presentation, Moleculin, MAR 13, 2020, View Source [SID1234555607]).

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On March 13, 2020 X4 Pharmaceuticals, Inc., a Delaware corporation ("X4") and its qualified subsidiaries, including without limitation X4 Therapeutics, Inc. (together with X4, the "Borrower") entered into a First Amendment to the Amended and Restated Loan and Security Agreement dated June 27, 2019 (collectively the "Amended Loan Agreement") with Hercules Capital, Inc., and Hercules Capital Funding Trust 2019-1 (collectively the "Lender" or "Hercules"), which provides for aggregate maximum borrowings of up to $50.0 million (Filing, 8-K, X4 Pharmaceuticals, MAR 13, 2020, View Source [SID1234555647]).

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The Amended Loan Agreement consists of (i) a term loan of $25.0 million (including the $20.0 million previously outstanding under the Amended and Restated Loan and Security Agreement dated June 27, 2019) and an additional $5.0 million drawn at the closing of the first amendment on March 13, 2020 (the "Closing Date") (the "Tranche 1 Term Loan Advance"), (ii) subject to the achievement of certain performance milestones and other conditions, a right of the Borrower to request that the Lender make additional term loan advances in an aggregate amount of up to $7.5 million through June 30, 2021 the ("Tranche 2 Term Loan Advance"), (iii) subject to the achievement of certain performance milestones and conditions, a right of the Borrower to request that the Lender make additional term loan advances in an aggregate amount of up to $7.5 million through June 30, 2022 ( "Tranche 3 Term Loan Advance") and (iv) subject to the Lender’s investment committee’s sole discretion, a right of the Borrower to request that the Lender make additional term loan advances in an aggregate amount of up to $10.0 million through December 31, 2022 ("Tranche 4 Term Loan Advance").

Borrowings under the Amended Loan Agreement bear interest at a variable rate equal to a per annum rate of interest equal to the greater of either (i) 3.75% plus the prime rate as reported in The Wall Street Journal, and (ii) 8.75%. In an event of default, as defined in the Amended Loan Agreement, and until such event is no longer continuing, the interest rate applicable to borrowings under the Amended Loan Agreement would be increased by 4.0%.

Borrowings under the Amended Loan Agreement are repayable in monthly interest-only payments through January 1, 2022, and in equal monthly payments of principal and accrued interest from February 1, 2022 until the maturity date of the loan, which is July 1, 2023. X4 may prepay all, but not less than all, of the outstanding borrowings, subject to a prepayment premium of up to 2.0%, 1.0% or 0.5% of the principal amount outstanding as of the date of repayment, in each case depending on when such repayment is made. In addition, the Amended Loan Agreement provides for payments by the Borrower to Hercules of (i) $795,000 payable upon the earlier of November 1, 2021 or the repayment in full of all obligations under the Amended Loan Agreement, and (ii) 4.0% of the aggregate principal amount of all Term Loan Advances drawn under the Amended Loan Agreement (which payment amount would be $2.0 million if X4 borrowed the aggregate maximum principal amount of $50.0 million), payable upon the earlier of the maturity of the Amended Loan Agreement or the repayment in full of all obligations under the Amended Loan Agreement.

Borrowings under the Amended Loan Agreement are collateralized by substantially all of the Borrower’s personal property and other assets except for their intellectual property (but including rights to payment and proceeds from the sale, licensing or disposition of the intellectual property). Under the Amended Loan Agreement, the Borrower has agreed to affirmative and negative covenants to which the Borrower will remain subject until maturity or repayment of the loan in full. The covenants include, without limitation:

(a) Effective immediately upon the date the outstanding principal amount of the advances under the Amended Loan Agreement exceeds $25.0 million, Borrower at all times thereafter shall maintain cash in an account or accounts of Borrower in which Hercules has a first priority security interest, in an aggregate amount greater than or equal to the greater of (i) $30.0 million or (ii) 6 multiplied by a metric based on prior months’ cash expenditures ("RML"); provided, however, that from and after Borrower’s achievement of certain performance milestones, the required level shall be reduced to the greater of (x) $20.0 million, or (y) 3 multiplied by the current RML; and provided further, that subject to the achievement of certain milestones, this covenant shall be extinguished.

(b) Restrictions on the Borrowers’ ability to incur additional indebtedness, pay dividends, encumber its intellectual property, or engage in certain fundamental business transactions, such as mergers or acquisitions of other businesses, with certain exceptions.

The Borrower’s obligations under the Amended Loan Agreement are subject to acceleration upon occurrence of specified events of default, including payment default, insolvency and a material adverse change in the Borrower’s business, operations or financial or other condition.

In addition, under the Amended Loan Agreement, Hercules has the right to participate, in a cumulative amount of up to $3.0 million in the aggregate, of which $1.0 million has already been exercised as of the Closing Date, and subject to exceptions as provided in the Amended Loan Agreement, in any future offering of X4’s equity securities for cash that is solely for financing purposes and is broadly marketed to multiple investors.

The foregoing description of the Amended Loan Agreement does not purport to be complete and is qualified in its entirety by reference to the Amended Loan Agreement, which is attached hereto as Exhibit 10.1 and is incorporated herein by reference.

On March 13, 2020 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 fourth quarter ended December 31, 2019 (Press release, Stemline Therapeutics, MAR 13, 2020, View Source [SID1234555550]).

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Robert Francomano, Chief Commercial Officer of Stemline, stated, "Overall, we are very pleased with the solid demand we generated for ELZONRIS in the first year of launch and look to expand our reach in the market as we continue to build a strong commercial foundation. Importantly, new medical claims data align with our independent analyses which support our market size estimates of the BPDCN U.S. patient population. We believe there is significant growth potential ahead and are poised to capture greater market penetration. Based on market assessments, we have already started to implement a new host of tactics to better situate ELZONRIS in this dynamic and emerging market – all of which should benefit growth later this year."

Ivan Bergstein, CEO of Stemline, commented, "Our first year of launch has created a strong foundation for the future growth of the company. We are investing in multiple label expansion opportunities for ELZONRIS in such indications as CMML, MF, and AML, as well as advancing our other pipeline products including felezonexor, our XPO1 inhibitor and SL-1001, our RET kinase inhibitor, toward key inflection points over the coming year and beyond."

Fourth Quarter 2019 Financial Results Review
Net product revenue for ELZONRIS was $11.8 million for the quarter ended December 31, 2019. Stemline began commercial sales of ELZONRIS within the United States in January 2019.

Stemline ended the fourth quarter with $164.4 million in cash, cash equivalents and short-term investments. For the fourth quarter, Stemline reported a net loss of $17.7 million, with net cash expenditures of $10.1 million.

Research and development expenses were $10.5 million for the fourth quarter of 2019, which reflects decrease of $1.6 million compared with $12.1 million for the fourth quarter of 2018. The lower expenses are primarily attributable to higher costs incurred during 4Q18 related to the ELZONRIS BLA filing and manufacturing of ELZONRIS prior to FDA approval.

Selling, general and administrative expenses were $16.5 million for the fourth quarter of 2019, which reflects an increase of $1.6 million compared with $14.9 million for the fourth quarter of 2018. The increase in costs were primarily attributable to ongoing U.S. launch expenses for ELZONRIS and pre-launch ELZONRIS-related costs in support of a potential regulatory approval and launch in the EU.

Corporate Highlights and Key Commercial and Clinical Milestones

BPDCN

·$43.2 million in net revenues for ELZONRIS in 2019

·IQVIA medical claims data identified approximately 534 unique patients in the U.S. in 2018 with at least one claim of Blastic NK-Cell Lymphoma, a former name of BPDCN

·Marketing Authorization Application (MAA) under review by European Medicines Agency (EMA) for potential approval in the EU

·Phase 1/2 trial of ELZONRIS in patients with BPDCN in the maintenance setting, post-stem cell transplant (SCT), open for enrollment

Chronic Myelomonocytic Leukemia (CMML)

·The CMML expansion cohort, Stage 3a, is open for enrollment of two patient populations: relapsed/refractory patients, and first-line, poor prognosis patients not expected to benefit from first line cytoreductive treatment

·Results from Stage 3a are expected to inform the design of the subsequent Stage 3b confirmatory cohort for potential registration

·We expect to provide updates from this trial in ~4Q20/1Q21

Myelofibrosis (MF)

·At ASH (Free ASH Whitepaper) 2019, ELZONRIS data in patients with relapsed/refractory MF was the subject of an oral presentation.

·In the MF clinical trial, ELZONRIS demonstrated efficacy (spleen size reductions and total symptom score [TSS] reductions) with a predictable and manageable safety profile, including in patients with poor prognostic factors, such as thrombocytopenia, CMML-type features/monocytosis, and clonal evolution

·The MF cohort of the ongoing trial has been expanded to include 20-25 additional patients

·We are evaluating relapsed/refractory patients and specific subsets of patients, including patients with monocytosis, thrombocytopenia, and CD123 positivity.

·We expect to provide updates from this trial in ~4Q20/1Q21

Acute Myeloid Leukemia (AML)

·A Phase 1/2 trial of ELZONRIS in combination with other agents in patients with relapsed/refractory AML, treatment-naive AML unfit for chemotherapy, and high-risk myelodysplastic syndrome (MDS) is currently enrolling patients. CD123 expression levels are also being evaluated. We expect to provide updates later this year.

Conference Call Information

The company will host a conference call and webcast on Monday, March 16, 2020 at 8:00 a.m. ET. The conference call can be accessed by dialing 1-800-367-2403 (domestic) or 1-334-777-6978 (international) and referring to conference ID 7728185.

The 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, and will be available live and for replay shortly after the event.

About ELZONRISÒ

ELZONRISÒ (tagraxofusp), a targeted therapy directed to CD123, 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 CD123+ indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), acute myeloid leukemia (AML), and others are planned, including a CD123+ all-comers trial.

About BPDCN

BPDCN, formerly blastic NK-cell lymphoma, is an aggressive hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. 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. The World Health Organization (WHO) termed this disease "BPDCN" in 2008; previous names included blastic NK cell lymphoma and agranular CD4+/CD56+ hematodermic neoplasm. 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 malignancies 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 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+ cells have been detected in the tumor microenvironment of several solid tumors as well as in certain autoimmune disorders including cutaneous lupus and scleroderma.

On March 13, 2020 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Applied Therapeutics, MAR 13, 2020, View Source [SID1234555551]).

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"This past year was a transformative time for Applied Therapeutics. In addition to our transition to a public company, we completed two additional financings, and made significant progress in our clinical development programs in Diabetic Cardiomyopathy and Galactosemia – both devastating diseases with no treatment options available," said Shoshana Shendelman, Ph.D., Founder, CEO and Chair of the Board of Applied Therapeutics. "With positive results from our pivotal Phase 2 ACTION-Galactosemia study in hand, we plan to move quickly towards regulatory filing, while preparing for Galactosemia commercial launch and growing our organization. On our Diabetic Cardiomyopathy program, we remain on track to fully enroll our Phase 3 registrational study this year, as we continue to advance this potential blockbuster opportunity. Throughout 2020 we’ll continue to move additional candidates into the clinic, while expanding our pipeline – delivering on our core strategy of applying technological advances to high unmet need indications."

Recent Highlights

·Closed $143.4 Million Underwritten Public Offering. In January 2020, we completed an underwritten public offering of common stock at a price to the public of $45.50 per share, resulting in gross proceeds of approximately $143.4 million.

·Announced Positive Topline Results of Pivotal Phase 2 ACTION-Galactosemia Study. In January 2020, we announced positive topline results from the Pivotal Phase 2 portion of the ACTION-Galactosemia study of AT-007, a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI), in adult Galactosemia patients. AT-007 treatment resulted in a statistically significant and robust reduction in plasma galactitol vs placebo in adult Galactosemia patients, and AT-007 was well tolerated, with no drug-related adverse events noted. We plan to utilize recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia, and expect to file for regulatory approval in the second half of 2020. We plan to present the full data from the ACTION-Galactosemia trial at the Society for Inherited Metabolic Disorders Annual Meeting, being held April 26 – 29 in Austin, Texas.

·Presented Pre-Clinical Data Highlighting AT-001 for Treatment of Diabetic Cardiomyopathy at the World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease (WCIRDC). In December 2019, we presented pre-clinical data on AT-001, a novel, potent and selective Aldose Reductase inhibitor (ARI) in Phase 3 clinical development for Diabetic Cardiomyopathy at WCIRDC in Los Angeles, California. The data showed a head to head comparison of AT-001 vs a first-generation ARI and highlights the improvements over these prior ARIs.

· Presented Study Design and Rationale for the ARISE-HF Pivotal Study of AT-001 for Treatment of Diabetic Cardiomyopathy at the 16th Annual Global Cardiovascular Clinical Trialist’s Forum (CVCT). In December 2019, we presented the study design and rationale for our ongoing ARISE-HF study at CVCT in Washington, D.C. ARISE-HF is a Phase 3 pivotal study examining effects of AT-001 on functional capacity (as measured by peak V02) in patients with Diabetic Cardiomyopathy at high risk of progression. We expect to announce topline data from the ARISE-HF trial in 2021.

·Presented Clinical Data Highlighting AT-001 for the Treatment of Diabetic Cardiomyopathy at the American Heart Association (AHA) Scientific Sessions 2019. In November 2019, we presented clinical data on AT-001 at AHA 2019 in Philadelphia, Pennsylvania. The data presented support our clinical rationale for development of AT-001 in Diabetic Cardiomyopathy by showing a reduction in NTproBNP, an important cardiac stress biomarker shown to correlate with long term heart failure outcomes and a strong preliminary indicator of efficacy in this patient population.

Financial Results

·Cash and cash equivalents and short-term investments totaled $38.9 million as of December 31, 2019, compared with $18.8 million at December 31, 2018. This does not include approximately $143.4 million in gross process we received from an underwritten public offering of common stock in January 2020.

·Research and development expenses for the year-ended December 31, 2019 were $32.4 million, compared to $11.5 million for the year ended December 31, 2018. The increase of approximately $20.9 million was primarily related to increased activity on our clinical trials, including an increase in clinical and pre-clinical expenses of $12.4 million and drug manufacturing and formulation expenses of $4.1 million, an increase in personnel expenses of $4.6 million that is allocated to research and development, offset by a decrease in regulatory and other expenses of $0.2 million.

·General and administrative expenses were $13.2 million for the year ended December 31, 2019, compared to $2.0 million for the year ended December 31, 2018. The increase of approximately $11.2 million was primarily related to the increase of personnel expenses of $4.4 million due to the portion of the chief executive officer’s salary that is allocated to general and administrative and the hiring of other personnel, including the chief financial officer, and an increase in professional and legal fees of $3.5 million due to the closing of multiple financings and increased IP work, and an increase in other expenses of $3.2 million, primarily due to recruiting efforts for the chief medical officer and rent.

·Net loss for year ended December 31, 2019 was $45.5 million, or $3.55 per basic and diluted common share, compared to a net loss of $16.5 million, or $3.01 per basic and diluted common share, for the year ended December 31, 2018.