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Stemline Therapeutics Reports First Quarter 2019 Financial Results

On May 10, 2019 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 first quarter ended March 31, 2019 (Press release, Stemline Therapeutics, MAY 10, 2019, View Source [SID1234536138]).

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"Since ELZONRIS became commercially available in January, we have been very pleased with the progress we have made executing our launch plan," stated Robert Francomano, SVP and Global Head of Commercial. "Our entire organization is working hard to ensure patients with BPDCN have access to ELZONRIS. Although still early, we believe the launch is progressing extremely well and remain poised for a very successful 2019 and beyond."

Ivan Bergstein, M.D., CEO of Stemline Therapeutics, commented "We have built a solid foundation for growth, driven by our launch of ELZONRIS for patients with BPDCN. We are executing our commercial plan, including pursuing ongoing efforts to unlock additional value from ELZONRIS in other indications as well as from our entire pipeline, all with the goal of improving the lives of patients with cancer around the world."

First Quarter 2019 Financial Results Review

Net revenue for ELZONRIS was $5.05 million for the quarter ended March 31, 2019. Stemline began commercial sales of ELZONRIS within the United States in January 2019.

Stemline ended the first quarter with $124.4 million in cash, cash equivalents and investments. For the first quarter, Stemline had a net loss of $27.4 million. Cash expenditures for the first quarter of 2019 was $21.9 million.

Research and development expenses were $17.0 million for the first quarter of 2019, which reflects an increase of $4.3 million compared with $12.7 million for the first quarter of 2018. The higher cost was primarily driven by expense recorded related to repayment of research funding as a result of the first commercial sale of ELZONRIS.

Selling, general and administrative expenses were $16.0 million for the first quarter of 2019, which reflects an increase of $10.1 million compared with $5.9 million for the first quarter of 2018. The increase in costs were primarily attributable to launch expenses in support of the commercialization of ELZONRIS.

Recent Corporate Developments and Program Highlights

ELZONRIS (tagraxofusp) — Blastic plasmacytoid dendritic cell neoplasm (BPDCN)

· ELZONRIS was approved by the FDA on December 21, 2018 and commercially available for patients with BPDCN in the U.S. in January 2019.

· The New England Journal of Medicine published the pivotal trial results in its April 25th edition.

· We submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in January 2019 seeking marketing approval in Europe. The MAA was granted accelerated assessment and is currently under review.

ELZONRIS — Market Expansion Efforts

· We are conducting clinical trials to evaluate ELZONRIS in additional indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

· Based on clinical results observed in patients with CMML and MF, we are evaluating next steps, including potential registrational pathways. For CMML, we intend to provide our registration-directed plans mid-year.

·We are also evaluating additional expansion opportunities, including maintenance therapy after stem cell transplant in patients with BPDCN.

· In parallel, we plan to expand our clinical efforts later this year and next into subsets of AML patients, including those enriched for CD123+ expression.

· We expect to provide periodic updates on these programs throughout this year and next at scientific conferences.

ASCO Conference

· ELZONRIS clinical trial data in CMML and MF have been selected for two poster presentations at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) conference in June.

Other pipeline candidates

·We expect to provide periodic updates on our other product candidates, SL-701, SL-801, SL-901, and SL-1001, later this year.

Conference Call Information

Stemline will host a conference call and live webcast today at 8:00 a.m. ET to discuss first quarter 2019 financial results and recent business activities. The conference call can be accessed by dialing 1-800-667-5617 (domestic) or 1-334-323-0509 (international) and referring to conference ID 2090827.

The live 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. The webcast will be archived and made available for replay on the company’s website shortly after the event.

About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, 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 indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. 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. 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 myeloid tumors 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 certain lymphoid malignancies including 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 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

Top European VC leads 28-7’s Series A extension with $15 million and brings financing to over $80 million

On May 9, 2019 Sofinnova Partners, a leading European venture capital firm specialized in the life sciences, reported that the firm led Twentyeight-Seven Therapeutics, Inc’s [28-7] Series A extension with a $15 million investment (Press release, Twentyeight-Seven Therapeutics, MAY 9, 2019, View Source [SID1234638814]). In addition to Sofinnova Partners, Osage University Partners (OUP) participated in the Series A extension bringing the total Series A financing to $82.75 million. Sofinnova Partners and OUP join the initial 28-7 investors that include MPM Capital, Novartis Venture Fund, Johnson & Johnson Innovation – JJDC, Inc., Vertex Ventures HC, Longwood Fund and Astellas Venture Management.

"Sofinnova Partners is known for being a company-builder, with a strong focus on teams and science. With Sofinnova’s participation, we are able to work on proteins that regulate additional classes of RNA, including mRNA, as well as to leverage the programs into indications outside of oncology"

"Sofinnova Partners views 28-7 as the clear leader in capitalizing on the emerging science around RNA biology and how its dysregulation contributes to many serious diseases such as cancer," said Henrijette Richter, Ph.D., Managing Partner at Sofinnova Partners. "We were captivated by the Company with its strong team of serial entrepreneurs possessing an incredible track record of successes, an exceptional group of academic founders, and the impressive scientific data and drug discovery progress around their lead RNA-focused program. They are developing game-changing therapeutics to treat cancers with high unmet need. We are therefore thrilled to be joining 28-7 early in the Company’s progress and to be investing in their exciting pipeline." Dr. Richter will join the Company’s Board of Directors.

Since the close of the initial $65M Series A announced in September 2018, 28-7 has moved into its new facilities in Watertown, MA. Kazumi Shiosaki, Ph.D., CEO of 28-7, noted that the additional investment by Sofinnova Partners allows the Company to accelerate its current programs, build out its technology platform and expand into additional opportunities. "Sofinnova Partners is known for being a company-builder, with a strong focus on teams and science. With Sofinnova’s participation, we are able to work on proteins that regulate additional classes of RNA, including mRNA, as well as to leverage the programs into indications outside of oncology," said Dr. Shiosaki.

28-7’s technology does not directly target small-molecules to the RNA itself but rather targets RNA-modulating proteins (RMPs). The lead program focuses on regulating levels of let-7, an important micro RNA (miRNA) that suppresses the translation of major oncogenes, whose levels can be disrupted by a protein called LIN28. In certain cancers, LIN28 is re-expressed and its binding of the let-7 precursor prevents the production of mature let-7 needed to maintain oncogene suppression. The Company’s lead program is developing small molecules that prevent the binding of let-7 precursor to LIN28 and restore the beneficial levels of mature let-7.

Probiodrug AG to Publish its First Quarter 2019 Business Update on May 16, 2019

On May 9, 2019 Probiodrug AG (Euronext Amsterdam: PBD, ISIN: DE0007921835), is focusing on the discovery and development of drugs acting on enzymes which are modulating the activity of cellular signalling pathways connected to human disease, reported that it will publish its first quarter business update for the period ended March 31, 2019 on Thursday, May 16, 2019, in the form of an interim management report (Press release, Vivoryon Therapeutics, MAY 9, 2019, View Source [SID1234537419]).

For more information, please contact:

Probiodrug
Dr. Ulrich Dauer, CEO
Email: [email protected]

MC Services AG
Anne Hennecke, Susanne Kutter
Tel: +49 (0) 211 529 252 27
Email: [email protected]

EnGeneIC Announces First Patients with Advanced-Stage Pancreatic Cancer Dosed in Phase 1/2a Clinical Study of Targeted Cytotoxic Immunotherapy

On May 9, 2019 EnGeneIC Limited, a clinical-stage biopharmaceutical company advancing its proprietary EDV nanocell platform for targeted cyto-immunotherapy in cancer, reported that the first four patients have been dosed in a Phase I/IIa study using the Company’s tumor-targeting, immunogenic EDVs to deliver a cytotoxic drug payload directly to tumors of patients who have exhausted curative treatment options (Press release, EnGeneIC, MAY 9, 2019, View Source [SID1234536197]). The study is enrolling patients with advanced pancreatic cancer and other EGFR-expressing solid tumors in a second cohort, which is currently underway at Frankston Private Hospital in Victoria, Australia, with Professor Vinod Ganju, MBBS, FRACP, as the Principal Investigator.

EnGeneIC’s second-generation EDV nanocells deliver an extremely cytotoxic nemorubicin derivative (D682) directly to solid tumors via EGFR targeting on the tumor cell surface, keeping healthy tissue protected from damage. The novel therapy also includes EDVs carrying an immune-boosting adjuvant to further augment the anti-tumor immune response stimulated by the bacterially-derived EDVs and perpetuated by tumor cell destruction.

Jennifer MacDiarmid, Ph.D., and Himanshu Brahmbhatt, Ph.D., joint-CEOs and Directors of EnGeneIC, stated, "We developed our second-generation EDVs to address multi-drug resistance in patients who have failed multiple lines of chemotherapy and therefore have the highest unmet need. D682 is an extremely potent drug that is far too toxic to be delivered systemically, but has proven safe in patients when encapsulated in our EDVs. We have named the study, the Carolyn Trial, after a close friend who had end-stage pancreatic cancer and was treated in a compassionate use case study. Carolyn was the first patient in the world to receive D682, and we observed highly encouraging results. Not only was survival extended, but quality of life improved considerably for the patient. Moreover, there was significant evidence of tumor regression coincident with a decrease in a key pancreatic cancer blood marker and a robust increase in anti-tumor CD8+ T cells and other anti-tumor immune cells. We are now executing a more rigorous clinical trial, not only for pancreatic cancer patients, but also for other advanced-stage patients with drug-refractory tumors."

Professor Ganju commented "Novel therapies for these late stage patients with drug-resistant tumors are desperately needed. Four patients have been enrolled on the study and so far we have been impressed with the tolerability and safety of this therapy. We will be getting some efficacy data in coming months."

About the Phase 1/2a Study

The two-cohort study will enroll up to 40 evaluable patients per cohort: 1) patients with advanced pancreatic cancer and 2) patients with EGFR-expressing solid tumors who have failed first- and second-line therapy or for whom standard therapies are not appropriate. The test article is EnGeneIC’s second-generation EGFR-targeted, D682-carrying EDV (EEDVD682) plus EDVs carrying an immune adjuvant (EDVadj) which acts to augment the anti-tumor immunity. Study objectives include assessing the safety and tolerability of EEDVD682 plus EDVadj, assessing anti-tumor response and overall survival. Exploratory objectives include biomarker assessment for immune response such as cellular immune response (CD8+ T cells, NK cells), and activated dendritic cells. In addition to the current clinical site, the study is expected to be opened in at least one other major cancer center in Sydney, Australia. For more information visit View Source;isReview=true

Savara Reports First Quarter 2019 Financial Results and Provides Business Update

On May 9, 2019 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, reported financial results for the first quarter ending March 31, 2019 and provided a business update (Press release, Savara, MAY 9, 2019, View Source [SID1234536174]).

"We are diligently preparing for the highly anticipated readout of our Phase 3 IMPALA study in June, which we expect to be followed by the submission of a Biologics License Application in the first half of 2020 and, if approved, a commercial launch of Molgradex later in 2020 or early 2021," said Rob Neville, Chief Executive Officer, Savara. "These pivotal results could be transformational for the Company and, more importantly, for patients with aPAP, a rare and progressive lung disease. Our commitment to improving the lives of people with orphan lung diseases, and accelerating the advancement of our innovative therapies, is unwavering. With multiple catalysts expected over the coming quarters, we are well positioned for sustained growth."

Recent Developments and Upcoming Highlights

Molgradex for aPAP
Expect top line results from the IMPALA study in June 2019. IMPALA is a global, pivotal Phase 3 clinical study evaluating Molgradex, an inhaled formulation of granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of aPAP. Positive results would facilitate the submission of a Biologics License Application in the first half of 2020, with an anticipated commercial launch later in 2020 or early 2021.
Continued strong enrollment in IMPALA-X, an open-label, multicenter extension study to determine the long-term safety and utilization of Molgradex in patients with aPAP. At the end of Q1 2019, 25 out of 26 eligible patients had enrolled in the extension study.
Molgradex received Fast Track Designation by the FDA for the treatment of aPAP. A drug granted with this designation may be eligible for Priority and/or Rolling Review, if relevant criteria are met.
Molgradex for nontuberculous mycobacterial (NTM) lung infection
Initiated ENCORE, a Phase 2a clinical study of Molgradex for the treatment of NTM in people living with cystic fibrosis (CF).
Expect top line results from OPTIMA, a Phase 2a clinical study evaluating Molgradex for the treatment of NTM in non-CF patients in Q1 2020.

Molgradex manufacturing

Entered into a new commercial manufacturing and supply agreement with GEMA Biotech, the company that has produced the drug substance for all Molgradex non-clinical and clinical studies. Under the terms of the agreement, GEMA Biotech will exclusively supply the Company with GM-CSF expressed from Savara’s proprietary cell line.
AeroVanc
Enrollment continues in AVAIL, a pivotal, global Phase 3 clinical study of AeroVanc, an inhaled vancomycin hydrochloride powder for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in CF.
As of today, the study has enrolled 153 patients out of a target of 200. An approximate 50% screen failure rate with younger subjects (between 6-21 years of age) has slowed enrollment. The screen failures are largely due to exacerbations between time of screening and randomization.
Expect to complete patient enrollment in Q3 2019 with top line results in Q2 2020.
Exploratory Pipeline
The initial indication for the Phase 2-ready aerosolized amikacin/fosfomycin combination antibiotic will focus on non-CF bronchiectasis patients with chronic lung infection and frequent exacerbations. A Phase 2 study is expected to start enrolling in bronchiectasis patients with recurrent exacerbations later in 2019 or early 2020 and will evaluate amikacin/fosfomycin and Molgradex separately, and in combination, to reduce bacterial infection load.
First Quarter Financial Results (Unaudited)
Savara’s net loss attributable to common stockholders for the three months ended March 31, 2019 was $12.1 million, or $(0.34) per share, compared with a net loss attributable to common stockholders of $26.8 million, or $(0.86) per share, for the three months ended March 31, 2018.

Research and development expenses were $10.0 million for the three months ended March 31, 2019, compared with $8.5 million for the three months ended March 31, 2018. The increase was primarily due to $1.9 million in increased development costs associated with the development of Molgradex, partially offset by a slight decrease in other program costs for the three months ended March 31, 2019.

General and administrative expenses for the three months ended March 31, 2019 were $2.8 million, compared with $1.8 million for the three months ended March 31, 2018. The increase was primarily due to increased personnel costs and other legal, accounting, insurance and operating activities.

As noted in the first quarter 2018 10-Q, during the quarter ended March 31, 2018, the Company recognized a $21.7 million impairment charge to the carrying value of acquired IPR&D related to a drug candidate previously assumed by Savara. For the first quarter ended March 31, 2019, there were no costs associated with this activity as the Company was no longer supporting or pursuing the drug candidate
.
Other income, net of other expense, increased by $0.1 million to $0.8 million for the three months ended March 31, 2019 from $0.7 million for the three months ended March 31, 2018 and was primarily related to a reduction of net interest expense.

Income tax benefit decreased by $4.5 million for the three months ended March 31, 2019 from the three months ended March 31, 2018 primarily due to the reversal of a deferred tax liability resulting from the impairment of certain acquired IPR&D during the first quarter of 2018.

As of March 31, 2019, Savara had a carrying value of its debt of approximately $24.7 million and had cash, cash equivalents, and short-term investments of approximately $105.2 million.

Conference Call and Webcast
Savara will host a conference call today at 4:30 p.m. Eastern Time (ET) / 1:30 p.m. Pacific Time (PT). Shareholders and other interested parties may access the conference call by dialing (855) 239-3120 from the U.S., (855) 669-9657 from Canada, and (412) 542-4127 from elsewhere outside the U.S. and request the "Savara Inc." call. A live webcast of the conference call will be available online in the Investors section of Savara’s website at View Source

Approximately one hour after the call, a replay of the webcast will be available on Savara’s website for 30 days, and a telephone replay will be available through May 16, 2019 by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada and (412) 317-0088 from elsewhere outside the U.S. and entering the replay access code 10130971.