On July 8, 2019 MorphoSys and Vivoryon Therapeutics reported that it enter Agreement on Small Molecule Inhibitors of CD47-SIRP alpha Signaling in Immuno-Oncology (Press release, MorphoSys, JUL 8, 2019, View Source [SID1234537398]).

MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; Nasdaq: MOR) and Vivoryon Therapeutics AG (Euronext Amsterdam: VVY) reported that they have entered into an agreement under the terms of which MorphoSys has obtained an exclusive option to license Vivoryon’s small molecule QPCTL inhibitors in the field of oncology. The option covers worldwide development and commercialization for cancer of Vivoryon’s family of inhibitors of the glutaminyl-peptide cyclotransferase-like (QPCTL) protein, including its lead compound PQ912. In exchange, MorphoSys has committed to investing up to EUR 15 million in a minority stake in Vivoryon Therapeutics as part of a capital raise planned for later this year.

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While Vivoryon’s lead drug candidate PQ912 has already completed a phase 2a clinical trial in Alzheimer’s disease, recent preclinical data strongly suggest that the compound could represent a novel approach for cancer therapy. Vivoryon’s orally available compounds target the QPCTL enzyme, which has been shown to be a modulator of the CD47-SIRP alpha interaction. Left unchecked, this interaction, known as the "don’t eat me" signal, allows cancer cells to escape the body’s innate immune defense through inhibition of the phagocytic activity of macrophages. During the option period, MorphoSys will conduct preclinical validation experiments on Vivoryon’s family of QPCTL inhibitors, including an assessment of the potential benefits of combining them with MorphoSys’s proprietary program tafasitamab (MOR208), which is currently in late-stage development for the treatment of relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL).

"This deal gives us access to a unique set of drug candidates with exciting potential in cancer", said Dr. Simon Moroney, CEO of MorphoSys. "A number of studies suggests that the CD47-SIRP alpha interaction may be of central importance to the activity of some anti-cancer antibodies. In this regard, securing rights to Vivoryon’s estate of compounds in oncology makes strong strategic sense for us. In particular, we are looking forward to exploring the potential for synergy with tafasitamab (MOR208), our most advanced drug candidate. If successful, the use of these orally formulated QPCTL inhibitors may open the way to combinations with other anti-cancer antibodies aiming at boosting their cell killing activity."

"Our small molecule inhibitors represent a novel and innovative therapeutic approach to silence the critical CD47-SIRP alpha checkpoint signal in cancer immunotherapy," said Dr. Ulrich Dauer, CEO of Vivoryon Therapeutics. "As a leading company for antibody and protein technologies with a strong oncology focus, MorphoSys is the ideal partner for us. For Vivoryon this is a strategic alliance to exploit the potential of our first-in-class, highly specific and potent small molecules in combination with therapeutic antibodies for a targeted range of cancer indications. While remaining strongly committed to our development plans in Alzheimer’s disease, we are delivering on our strategy to extend the potential of our technology to immuno-oncology."

If MorphoSys chooses to exercise the option, Vivoryon Therapeutics will receive an option fee, and is eligible for milestone payments and royalties.

On July 8, 2019 Coherus BioSciences, Inc. ("Coherus" or "the Company", Nasdaq: CHRS), reported that UDENYCA preliminary unaudited net sales for the quarter that ended June 30, 2019 are expected to be in the range of $79.0 million to $84.0 million (Press release, Coherus Biosciences, JUL 8, 2019, View Source [SID1234537417]).

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Coherus will not disclose topline gross sales, discounts, or gross-to-net data until these data are reported in its Form 10-Q for the quarter ended June 30, 2019.

As announced, Coherus will provide additional financial updates and business highlights on August 1, 2019 at 4:30 p.m. Eastern Time.

On July 8, 2019 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health") reported that it will release its second-quarter 2019 financial results on Tuesday, Aug. 6, 2019 (Press release, Valeant, JUL 8, 2019, View Source [SID1234537399]). Bausch Health will host a conference call and live web cast at 8:00 a.m. EDT to discuss the results and provide a business update. All materials will be made available on the Investor Relations section of the Bausch Health website prior to the start of the call.

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Conference Call Details

Date:

Tuesday, Aug. 6, 2019

Time:

8:00 a.m. EDT

Webcast:

View Source

Participant Event Dial-in:

+1 (888) 317-6003 (United States)

+1 (412) 317-6061 (International)

+1 (866) 284-3684 (Canada)

Participant Passcode:

2275809

Replay Dial-in:

+1 (877) 344-7529 (United States)

+1 (412) 317-0088 (International)

+1 (855) 669-9658 (Canada)

Replay Passcode:

10132759 (replay available until Aug. 13, 2019)

On July 8, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported its annual shareholder meeting, highlights recent clinical and regulatory developments, and details other upcoming events (Press release, Stemline Therapeutics, JUL 8, 2019, View Source [SID1234537418]).

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ELZONRIS clinical data were presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in Chicago, Illinois and the 24thCongress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Amsterdam, Netherlands. Presentations highlighted updated results from ongoing Phase 2 clinical trials in chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), as well as final results of the pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN). In addition, ELZONRIS preclinical results in systemic sclerosis, an autoimmune disorder in which CD123+ plasmacytoid dendritic cells (pDCs) play a role in disease pathogenesis, were presented at the Annual European Congress of Rheumatology (EULAR) in Madrid, Spain.

As previously announced, an additional single-arm cohort of patients with previously-treated CMML, Stage 3, will be added to the currently enrolling trial. In the first part of Stage 3 (Stage 3a), enrichment strategies and certain efficacy endpoints (including spleen size reduction) will be assessed for inclusion in the confirmatory cohort (Stage 3b), that will aim to provide the primary evidence of efficacy to support potential registration. We expect to open enrollment of the new cohort in 4Q19.

Stemline continues to build out a European commercial infrastructure in advance of potential approval by the European Medicines Agency (EMA). A scientific advisory group meeting is planned for September, and the ELZONRIS marketing authorization application (MAA) review will proceed on a standard timeline. We expect an opinion by the Committee for Medicinal Products for Human Use (CHMP) later this year. If successful, Stemline is targeting a commercial launch in Europe in 1Q20.

In an ongoing market expansion effort, corporate and investigator-sponsored clinical trials with ELZONRIS in additional indications are ongoing in myelofibrosis (MF) and acute myeloid leukemia (AML) and others are planned to roll out later this year and next.

Stemline continues to advance its clinical-stage assets, including SL-801 (a reversible inhibitor of XPO1). XPO1 is a clinically validated target in oncology, and the FDA recently approved an XPO1 inhibitor in patients with relapsed/refractory multiple myeloma. Stemline is also developing its preclinical assets SL-1001 (RET kinase inhibitor) and SL-901 (kinase inhibitor).

Stemline will provide further details on commercial and clinical progress during its second quarter financial results teleconference in early August. Call-in details will be provided in advance of the call.
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.

On July 8, 2019 -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 that it has completed a successful Type B Pre-Biologics License Application ("Pre-BLA") meeting with the U.S. Food and Drug Administration ("FDA") regarding a potential pathway for FDA approval of naxitamab for the treatment of patients with relapsed/refractory high-risk neuroblastoma (Press release, Y-mAbs Therapeutics, JUL 8, 2019, View Source [SID1234537400]).

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At the meeting, the Company reached alignment with the FDA on an Accelerated Approval Pathway for naxitamab along with a rolling BLA submission. The Company expects to submit the Clinical/Safety portion and the non-Clinical portion of the BLA in November 2019. For the CMC portion, the Company believes it will have sufficient data from the process performance qualification ("PPQ") batches to complete the CMC portion in early 2020. However, Y-mAbs is still investigating possibilities for accelerating the submission of the CMC portion, and hope to comply with the FDA requirements at an earlier time.

Under naxitamab’s breakthrough therapy designation ("BTD"), the compound qualifies for a Rolling BLA, which enables individual modules of the application to be submitted by the Company and reviewed by the FDA on a rolling basis, rather than waiting for all sections to be completed before submission. The rolling application process will provide the Company with the opportunity for ongoing communications with the FDA, and, during this rolling process, the Company anticipates that it will be able to address any substantial matters raised by the FDA.

Based on the previously announced efficacy data from Study 12-230 in relapsed/refractory high-risk neuroblastoma patients at Memorial Sloan Kettering Cancer Center ("MSK"), the FDA determined that efficacy data from all 37 patients of the Company’s multicenter Study 201 would not be required for the BLA filing. The FDA advised the Company that the available data for the first group of patients treated outside MSK in Study 201 would be sufficient for the BLA filing. The first group consists of 24 patients, of which 11 were evaluable prior to the pre-BLA meeting and showed an overall response rate ("ORR") of 73%, including 55% complete responses ("CR"), as assessed by the investigators. The Company intends to announce the complete dataset for Study 201 once the data becomes available.

"The positive outcome of the Pre-BLA meeting will be consequential for high-risk neuroblastoma patients waiting to get access to this new outpatient treatment with encouraging data," stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer, continued, "We are pleased to see that the clinical data previously generated at MSK was able to be replicated at other sites. We believe that an ORR of 73% may place naxitamab in a strong position in the market for the treatment of high-risk neuroblastoma."