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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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, 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products reported an update on its clinical pipeline and financial results for the first quarter ended January 31, 2020 (Press release, Advaxis, MAR 13, 2020, View Source [SID1234555547]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Key recent corporate and clinical pipeline updates:

●Presented updated clinical data from the ongoing Phase 1/2 ADXS-503 trial at the I/O 360° Conference. Data presented showed that the first two patients treated in the combination arm, who previously progressed on KEYTRUDA, achieved a partial response with substantial tumor shrinkage of nearly 60% and the other patient achieving stable disease with a 25% reduction in a target lesion.
●Presented updated survival data from the Phase 1/2 ADXS-PSA trial at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium. Data highlights include reported median overall survival (95% CI) of 16.4 months (4.0-NR) (n=11) for advanced prostate cancer patients with visceral metastases treated with ADXS-PSA in combination with KEYTRUDA compared to an estimated 11 months with current standard of care. In addition, median overall survival (95% CI) was 33.7 months (15.4-33.7) in all patients treated with ADXS-PSA in combination with KEYTRUDA (n=37).
●Data presented in 2020 suggest that both ADXS-503 and ADXS-PSA may have the potential to enhance or restore sensitivity to checkpoint inhibitors such as KEYTRUDA.
●Announced FDA allowance of its Investigational New Drug Application (IND) for ADXS-504 for the treatment of prostate cancer. ADXS-504 is the Company’s second drug product candidate from its HOT off-the-shelf neoantigen clinical program targeting hotspot mutations and other tumor-associated antigens.
●Closing of a $10.5 million equity financing with two investors.
●Announced a research agreement with Personalis to deploy ImmunoID NeXT Platform in the ADXS-503 clinical program. Personalis will conduct comprehensive tumor immunogenic profiling to enable the identification of predictive composite biomarkers and/or signatures of response, as well as the broad evaluation of potential mechanisms of therapy resistance.

Management Commentary

"We have started our fiscal year with encouraging positive data presented in our ADXS-PSA and ADXS-503 clinical programs," said Kenneth A. Berlin, President and Chief Executive Officer of Advaxis. "Importantly, data from both studies suggest that Lm immunotherapies may have the ability to synergistically enhance or restore sensitivity to checkpoint inhibitors which could be a meaningful breakthrough in improving outcomes for advanced and refractory patients. We continue to execute on our HOT off-the-shelf program in NSCLC with enrollment continuing in the combination arm of the study, Part B, and a planned initiation of Part C which will move combination therapy to a first-line setting, later this year. We are also planning to move an additional HOT construct, ADXS-504, for prostate cancer, into the clinic later this year for which the IND was allowed earlier this year."

Mr. Berlin continued, "We are currently evaluating next steps for our ADXS-PSA program based on the promising increases in median overall survival observed in combination with KEYTRUDA. With an anticipated cash runway into mid-2021, we are positioned to explore the early signals of activity in our ongoing trials while advancing additional programs that leverage these important findings."

First Quarter Ended January 31, 2020 Financial Results

During the quarter ended January 31, 2019, the Company recognized $19.4 million in revenue associated with the revenue recognition requirements surrounding the termination of the collaboration agreement with Amgen in 2019; no similar situation existed during the fiscal quarter ended January 31, 2020.

Research and development expenses for the first quarter of fiscal year 2020 were $4.9 million, compared with $6.7 million for the first quarter of fiscal year 2019. The decrease is largely attributable to the winding down of our Phase 3 AIM2CERV and Phase 1 ADXS-NEO studies as announced in June 2019 and October 2019, respectively.

General and administrative expenses for the three months ended January 31, 2020 were approximately $3.0 million compared to $2.7 million in the same three-month period in 2019 as a result of higher business development and legal fees.

As of January 31, 2020, the Company had approximately $34.2 million in cash and cash equivalents. The Company believes this is sufficient capital to fund its obligations, as they become due, in the ordinary course of business until at least mid-2021.

On March 13, 2020 Portage Biotech Inc. (PBT.U, OTC Markets: PTGEF) ("Portage" or the "Company") is reported that it has filed its third quarter financial statements and accompanying management and discussion (Press release, Portage Biotech, MAR 13, 2020, View Source [SID1234555546]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

These documents are available on SEDAR and on the Company’s website: www.portagebiotech.com.

Highlights of this quarter include:

• October 2019, Intensity therapeutics announced the dosing of its first patient with a combination of its drug INT230-6 and Merck’s Ketruda. This is part of a clinical trial collaboration agreement with Merck to study this combination

• October 2019, Sentien CEO Brian Miller presented Phase 1b clinical data of SBI-101 in acute renal injury at the 2019 Cell and Gene Meeting

• November 2019, Intensity announced that its experimental product, INT230-6 induced prolonged stable disease with abscopal effects and immune responses in multiple solid tumors. This data was selected for an oral presentation at the 34th annual Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting.

• In the period, further progress on preclinical work and progress made in furthering projects from the other subsidiary’s towards the clinic. The Company also wishes to provide an update on the status of its application (the "Revocation Application") to the Ontario Securities Commission (the "OSC") to revoke an outstanding cease trade order issued on August 2, 2019 (the "CTO").

The Company has resolved almost all comments from the OSC except for one: during the review process, the Company was informed by the OSC that it would be required to file a business acquisition report (BAR) under National Instrument 51-102 Continuous Disclosure Obligations in connection with its acquisition of SalvaRx Limited. Although there is already substantial public disclosure in respect of the acquisition available on SEDAR and the Company’s website, the presentation of financial information in respect of SalvaRx Limited was not in a format in compliance with the BAR requirements. The Company is in the process of compiling the relevant financial records for the creation of a proper BAR and is hopeful it will be able to resolve this issue in the very near future.

As indicated in the Company’s news release of January 30, 2020, the relief being sought under the Revocation Application is discretionary and, as such, no firm estimate can be provided as to-2-

when a revocation order will be issued by the OSC. The Company is confident, however, that a revocation order will be issued and, upon issuance, it is expected that the Company’s common shares will resume trading on CSE almost immediately thereafter.

In the interim, shareholders and investors are reminded that, although trading in the shares of the Company may not currently be effected through CSE, trading is permitted on OTC Markets in the United States as follows:

(a) Under the terms of the CTO, Canadian shareholders may trade the shares of the Company on OTC Markets in the United States provided they use the services of a registered dealer in Canada with access to OTC Markets;

(b) US shareholders may trade through OTC markets using the services of any dealer in the United States; and

(c) Other foreign shareholders, such as those in the United Kingdom, may trade through through the services of any dealer (other than Canadian) with access to OTC Markets.

Each dealer will have their own internal rules regarding the ability of shareholders and investors to trade the Company’s shares on OTC Markets. It is the individual responsibility of each shareholder or investor to satisfy these rules before they may trade.

The common shares of the Company trade on OTC Markets under the symbol "PTGEF".

At the time the revocation order is issued, the Company will announce a date for an annual meeting of shareholders at which the Company will bring shareholders and investors up to date on the current operations of the Company and its future plans. The Company wishes to thank shareholders for their continued patience while the Revocation Application is processed.