On December 6, 2018 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported additional data on patterns of clinical relapses (including organ or site of recurrence), as well as results from a preplanned secondary efficacy analysis across various predefined subgroups from the prospective, randomized, single-blinded, controlled Phase 2b independent investigator-sponsored clinical trial of the combination of trastuzumab (Herceptin) +/- nelipepimut-S (NeuVax, NPS) targeting HER2 low-expressing breast cancer patient cohorts at the 41st San Antonio Breast Cancer Symposium (SABCS) in San Antonio, TX (Press release, Sellas Life Sciences, DEC 6, 2018, View Source [SID1234531928]).

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These new data show a decrease in the total number of clinically detectable relapses with the combination of NPS + trastuzumab (7.5%) vs. trastuzumab alone (27.3%), p-value 0.004, which represents a 72.5% relative reduction in risk of relapse across time with a median follow up of 26.1 months in favor of the combination arm. Results from a planned analysis (log-rank) of the difference in disease free survival (DFS) outcomes between the two arms of the study in prespecified TNBC patient subgroups (patients who received neoadjuvant chemotherapy, expressed lower HER2, were 51 years or older, or had AJCC 7th Edition stage I/II TNBC), showed a clinically meaningful and statistically significant effect (p-value range: 0.004 – 0.014) in these subgroups in favor of the NPS plus trastuzumab combination arm. There was an average decrease of 84.2% in the relative risk of relapse or death at 24 months across these four subgroups of TNBC patients treated with NPS plus trastuzumab vs trastuzumab alone. The full data are summarized in the table below:

Comparison between NPS + trastuzumab vs. trastuzumab arms
Patient subgroups within the TNBC cohort Hazard Ratio (HR) HR 95% Confidence Interval P-value
(difference in favor of NPS + trastuzumab) Decrease in relative risk of relapse or death at 24 mos. (in favor of NPS + trastuzumab)
Received Neoadjuvant Chemotherapy 0.226 0.063 – 0.815 0.013 78.1%
Harbored BC with HER2 IHC 1+ expression level 0.178 0.038 – 0.837 0.014 81.3%
Aged ≥ 51 years 0.144 0.031 – 0.656 0.004 77.4%
AJCC 7 Stage I/II at diagnosis Incalculable* N/A 0.006 100%
*no DFS events occurred in AJCC 7 stage I/II TNBC patients treated with NPS plus trastuzumab.

"These new data provide insights on the pattern of clinically detectable relapses across various sites/organs, as well as add to our knowledge of the specific potential benefit distribution within the TNBC cohort. The results in four predefined TNBC subgroups inform us of the types of TNBC patients with residual disease after neoadjuvant chemotherapy who may potentially benefit when treated with NPS plus trastuzumab in the adjuvant setting. As previously announced, we are on track to meet with the U.S. Food and Drug Administration this month on the most expeditious and appropriate development path for NPS in TNBC," said Nicholas J. Sarlis, MD, PhD, Executive Vice President and Chief Medical Officer of SELLAS.

"We are very pleased with the results of these new analyses which indicate that the NPS plus trastuzumab combination – when given in the adjuvant setting after frontline therapy – could potentially improve outcomes across specific predefined subgroups of patients with early-stage TNBC, an aggressive subtype of breast cancer. The clinically meaningful and statistically significant decrease in the frequency of clinically detectable relapses – with a median follow-up of over 26 months – indicates a high degree of internal consistency," commented Elizabeth A. Mittendorf, MD, PhD, Rob and Karen Hale Distinguished Chair in Surgical Oncology, Director of Research, Breast Surgical Oncology Brigham and Women’s Hospital, Director, Breast Immuno-Oncology Program Dana-Farber/Brigham and Women’s Cancer Center, and the Principal Investigator of the Phase 2b study. "The data presented today are consistent with the previously reported beneficial effect seen in the TNBC cohort at large and are consistent with the immunobiological mechanism of action of nelipepimut-S."

Herceptin is a registered trademark of Genentech, Inc. and is not a trademark of SELLAS. The manufacturer of this brand is not affiliated with and does not endorse SELLAS or its products.

SABCS Presentation Information

Date and Time: Thursday, December 6, 2018; 8:00 – 10:00 am ET
Poster Session 2: Treatment: Immunotherapy (clinical)
Poster Hall Location: Hall 1
Abstract ID: P2-09-01
Title: Subgroups analysis of a multicenter, prospective, randomized, blinded phase 2b trial of trastuzumab + nelipeptimut-S (NeuVax) vs. trastuzumab for prevention of recurrence in breast cancer patients

About SABCS

The mission of the SABC Symposium (SABCS) is to provide state-of-the-art information on breast cancer research. Since 2007, the SABCS has been jointly sponsored by the Cancer Therapy & Research Center (CTRC) at the University of Texas Health Science Center – San Antonio, the Baylor College of Medicine and the American Association for Cancer Research (AACR) (Free AACR Whitepaper).

On December 6, 2018 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported it has filed a request with the U.S. Food and Drug Administration ("FDA") for Orphan Drug Status for its drug candidate WP1066 (Press release, Moleculin, DEC 6, 2018, View Source [SID1234531927]).

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"Clinical progress with WP1066 has been encouraging," commented Walter Klemp, Moleculin’s Chairman and CEO. "Given its potential to address rare and difficult to treat cancers, including glioblastoma, we believe WP1066 is well positioned to qualify for Orphan Drug Status."

The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

On December 6, 2018 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, reported that data from the Phase 3 RECOVER clinical study was presented during the 2018 San Antonio Breast Cancer Symposium (SABCS) (Press release, Spectrum Pharmaceuticals, DEC 6, 2018, View Source [SID1234531926]). These data confirm the efficacy and safety of ROLONTIS (eflapegrastim) in reducing the Duration of Severe Neutropenia (DSN) in breast cancer patients treated with chemotherapy. ROLONTIS is a novel, long-acting granulocyte colony-stimulating factor (G-CSF) being studied as a treatment for neutropenia in patients undergoing treatment with myelosuppressive cytotoxic chemotherapy.

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"Despite available treatments, neutropenia remains a critical issue for patients undergoing chemotherapy that puts them at risk for developing life threatening infections," said Lee Schwartzberg, MD, FACP, lead investigator, professor of medicine and division chief, hematology/oncology, University of Tennessee Health Science Center, and executive director, UT/West Cancer Center. "The robust data from both Phase 3 studies demonstrate that ROLONTIS has the potential to be a valuable option in the management of neutropenia for patients undergoing treatment with chemotherapeutic agents."

The data released yesterday in a poster presentation from the ROLONTIS Phase 3 RECOVER study (n=237) showed that in Cycle 1, the mean DSN±SD was 0.31±0.688 days for ROLONTIS and 0.39±0.949 days for pegfilgrastim, demonstrating non-inferiority (p<0.0001). The non-inferiority of ROLONTIS for DSN was maintained across all four treatment cycles (all (p<0.0001)). Incidence of severe neutropenia was 20 percent versus 24 percent in the eflapegrastim and pegfilgrastim arms respectively, with a relative risk reduction of 14 percent in favor of eflapegrastim. There were no statistically significant differences on secondary endpoints such as time to absolute neutrophil count (ANC) recovery, depth of ANC nadir, and incidence of febrile neutropenia between treatment arms across all cycles. None of the ≥Grade 3 study drug related adverse events (AE) occurred in >2% of the patients and included hematologic and bone pain related AEs in both arms.

The RECOVER trial is the second ROLONTIS Phase 3 study to meet the primary efficacy endpoint of non-inferiority in mean DSN. Results from ADVANCE, the first ROLONTIS Phase 3 study, were announced at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and presented at the Multinational Association in Supportive Care in Cancer Annual Meeting (MASCC) earlier this year.

"The RECOVER study is the second Phase 3 study to confirm non-inferiority data and comparable safety profile between ROLONTIS and the current standard of care," said Joe Turgeon, chief executive officer of Spectrum Pharmaceuticals. "Data from both studies, which enrolled 643 patients combined, will be used to support the BLA filing which is expected to be submitted by the end of the year. If approved, we look forward to having a unique opportunity to launch and compete in this large market with the first novel G-CSF in more than 15 years."

About RECOVER

The RECOVER study is a Phase 3, randomized, open-label, active-controlled, multicenter study that enrolled 237 breast cancer patients who received docetaxel and cyclophosphamide chemotherapy every 21 days. Patients were randomized in a 1:1 ratio to receive either ROLONTIS (n=118) or pegfilgrastim (n=119). The primary study endpoint was the DSN in Cycle 1 of chemotherapy (absolute neutrophil count [ANC] <0.5×109/L), based on central laboratory assessment of ANC over a 21 day cycle. There were a total of four cycles evaluated in this study. Secondary endpoints included, the DSN in Cycles 2, 3, and 4, time to ANC recovery, depth of ANC nadir and incidence of febrile neutropenia at cycle one. Patients with stage I to stage IIIA breast cancer were treated on day one of each of the four cycles with adjuvant/neo-adjuvant docetaxel and cyclophosphamide. On day two of each cycle, patients received a single subcutaneous dose of either eflapegrastim 13.2 mg/0.6 mL (equivalent to 3.6 mg G-CSF) or pegfilgrastim (6 mg).

On December 6, 2018 Molecular Partners AG (SIX: MOLN), a clinical-stage biopharmaceutical company pioneering the use of DARPin therapeutics* to treat serious diseases, reported the continued progress of its pipeline of proprietary therapeutic product candidates in oncology and immuno-oncology, as well as in ophthalmology together with its partner Allergan (Press release, Molecular Partners, DEC 6, 2018, View Source [SID1234531925]).

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On its R&D Day in New York, entitled "Building Tomorrow’s Breakthroughs," the company elaborates on its refined development plans for its most advanced proprietary candidate MP0250 in multiple myeloma (MM). In particular, the company announces its intention to initiate a second phase 2 trial for MP0250 in MM. This complementary trial will recruit patients with relapsed or refractory MM who have failed at least two lines of therapy including a proteasome inhibitor and an immunomodulatory drug (IMiD) and in whom the most recent therapy was IMiD-based. Patients will be treated with MP0250 in combination with Pomalidomide and dexamethasone. The design of the initial phase 2 trial for MP0250 in MM will be updated to recruit patients with a proteasome inhibitor (PI) based regimen as the most recent therapy. Those patients continue to be dosed with MP0250 in combination with Velcade and dexamethasone.

Molecular Partners also presents additional preclinical data on MP0310, its most advanced multi-specific (FAP x 4-1BB) DARPin immuno-oncology compound, which is expected to move into the clinic in 2019. In addition, data on FAP x CD40, a second multi-specific preclinical DARPin therapeutic in immuno-oncology, will be presented.

"I am pleased to report on our progress in oncology and our efforts to build a sustainable pipeline of DARPin therapeutics," said Patrick Amstutz, Chief Executive Officer of Molecular Partners.

Pamela A. Trail, Chief Scientific Officer of the company, added: "Our unique DARPin designs have the potential to translate into novel therapeutics across the landscape of oncology. Going forward, we will focus on several key areas of therapeutic intervention which are in the sweet spot of our DARPin technology."

In addition to an overview of the Molecular Partners clinical and preclinical pipeline, the R&D Day will feature presentations by the following experts:

Dr. Robert Orlowski, Chairman, Ad Interim, Director of Myeloma, Professor of Medicine Departments of Lymphoma/Myeloma and Experimental Therapeutics, MD Anderson Cancer Center
Dr. Yehia Hashad, Vice President and Global Head of Clinical Development, Ophthalmology, Allergan
Logistics
The R&D Day for institutional investors, sell-side analysts, investment bankers, and business development professionals will take place at The Yale Club, 50 Vanderbilt Avenue, New York City, from 11:30 am – 2:30 pm EST. To RSVP email Susan A. Noonan at [email protected].

Attendees are invited to check at 11:30 am EST. The presentations will begin at 12:00 pm, followed by a Q&A session. Lunch will be served.

Audio webcast
A respective audio webcast will be accessible, both live and as a replay, on the investors section of the company’s website, along with the accompanying presentation slides.

Financial Calendar
November 1, 2018 – Q3 2018 Management Statement
December 6, 2018 – R&D Day in New York
February 7, 2019 – Publication of Full-year Results 2018 (unaudited)
March 15, 2019 – Expected Publication of Annual Report 2018
April 16, 2019 – Annual General Meeting
May 9, 2019 – Interim Management Statement Q1 2019
View Source

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage more than 5 targets at once, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics.
The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their good safety profile, low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapeutics have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology, and is advancing a proprietary pipeline of DARPin drug candidates in oncology and immuno-oncology. The most advanced global product candidate is abicipar, a molecule currently in phase 3, in partnership with Allergan. Several DARPin molecules for various ophthalmic indications are also in development. The most advanced DARPin therapeutic candidate wholly owned by Molecular Partners, MP0250, is in phase 2 clinical development for the treatment of solid tumors and hematological tumors. MP0274, the second-most advanced DARPin drug candidate owned by Molecular Partners, has broad anti-HER activity; it inhibits HER1, HER2 and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. MP0274 is currently in phase 1. Molecular Partners is also advancing a growing preclinical pipeline that features several immuno-oncological development programs. DARPin is a registered trademark owned by Molecular Partners AG

On December 6, 2018 Stemline Therapeutics, Inc. (Nasdaq: STML), a biopharmaceutical company focused on the development and potential commercialization of novel oncology therapeutics, reported that updated data from multiple ELZONRISTM (tagraxofusp; SL-401) clinical trials at the 2018 ASH (Free ASH Whitepaper) Annual Meeting (Press release, Stemline Therapeutics, DEC 6, 2018, View Source [SID1234531924]). Presentations are now available on the Stemline website, www.stemline.com, under the Scientific Presentations tab.

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Key Outcomes from Pivotal Trial of ELZONRIS in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Across all 3 stages, 42 patients received ELZONRIS (12 ug/kg/day)
• In first-line patients with BPDCN, ELZONRIS (12 ug/kg/day)
– 90% (26/29) overall response rate (ORR)
– 72% (21/29) rate of CR + CRc (complete response + clinical CR [CR with residual skin abnormality])
– 45% (13/29) of patients were bridged to stem cell transplant (SCT)
• In relapsed/refractory patients with BPDCN, ELZONRIS (12 ug/kg/day)
– 69% (9/13) ORR
– 38% (5/13) CR + CRc + CRi rate
– 1 patient was bridged to SCT
• Median overall survival (OS) was not yet reached in first-line patients treated with ELZONRIS at 12 ug/kg/day across all stages. Median follow up time is 23.0 months (range of 0.2 – 41+ months)
• Most common treatment-related adverse events (TRAEs) in all patients treated across all trials at 12 ug/kg/day dose (N=148) were: alanine aminotransferase increase (43.9%), aspartate aminotransferase increase (43.9%), hypoalbuminemia (43.9%), and thrombocytopenia (26.4%). TRAEs included capillary leak syndrome (CLS) (16.9%) which was grade 5 in 1.5% (3/202) of patients across all trials and doses and 0.6% (1/166) of patients across all trials at 12 ug/kg/day; a myocardial infarction, grade 5, occurred in a patient (12 ug/kg/day) with grade 4 CLS, as previously reported.

Stage 3 pivotal cohort (13 first-line BPDCN patients treated with SL-401 at 12 ug/kg/day):
• Met its primary endpoint with a 54% (7/13) CR + CRc rate (95% CI: 25.1, 80.8)
• 77% (10/13) ORR
• 46% (6/13) of patients were bridged to SCT
In the U.S., the ELZONRIS biologics license application (BLA) is under Priority Review, with a February 21, 2019 PDUFA action date

In the E.U., the European Medicines Agency (EMA) has granted accelerated assessment to the planned Marketing Authorization Application (MAA), which is expected to be submitted in the first quarter of 2019
Key Outcomes from Ongoing Clinical Trial of ELZONRIS in Patients with Relapsed/Refractory Chronic Myelomonocytic Leukemia (CMML)

ELZONRIS demonstrated efficacy (spleen and bone marrow responses), with a manageable safety profile, in patients with relapsed/refractory CMML, an area of unmet medical need

100% of evaluable patients had reduction in baseline splenomegaly
– 80% had reduction by ≥50%
– 67% with baseline spleen size ≥5cm had reduction by ≥50%

3 bone marrow complete responses (BMCRs)

1 patient bridged to stem cell transplant in remission on ELZONRIS

Most common TRAEs include hypoalbuminemia (35%), thrombocytopenia (35%), nausea (30%) and vomiting (30%). Most common TRAEs, grade 3+, include thrombocytopenia (35%) and nausea (5%). CLS was reported in 15% of patients, with no cases of grade 3 or higher observed

Splenomegaly has historically been associated with serious sequelae including early satiety and intractable pain, as well as poor transplant outcomes and a higher propensity for AML transformation

Targeting the proliferative component of CMML, namely alleviation of splenomegaly, could result in meaningful clinical benefit and address a key unmet medical need
Next Steps

Patient enrollment and follow up continues, and additional updates are planned in 2019

Based on the encouraging results seen in this trial, a registrational trial design, or pivotal cohort, is being designed
Key Outcomes from Ongoing Trial of ELZONRIS in Patients with Relapsed/Refractory Myelofibrosis

ELZONRIS monotherapy demonstrated efficacy (improvements in splenomegaly), with a manageable safety profile, in patients with relapsed/refractory MF, an area of unmet medical need; Patient enrollment and follow up continues

100% of evaluable patients with monocytosis and baseline spleen size ≥5cm, had reduction in baseline splenomegaly
– 80% had reduction by ≥29%; 40% had reduction by ≥45%
– Historically, monocytosis has been associated with poor prognosis in certain MF patients

57% of evaluable patients, with baseline spleen size ≥5cm, had reduction in baseline splenomegaly
– 43% had reduction by ≥29%; 21% had reduction by ≥45%

6 patients with spleen response had treatment duration of 6+ months; 5 patients ongoing
– 5 patients with baseline thrombocytopenia (platelet count <100K) had treatment duration of 6+ months; 4 ongoing
– 3 patients with baseline monocytosis (>1×109/L) had treatment duration of 8+ months; 2 patients ongoing

Initial quality of life (QOL) assessments appear promising; full symptom score analyses are ongoing

Most common TRAEs include headache and hypoalbuminemia (each 22%), and alanine aminotransferase increased and thrombocytopenia (each 17%). The most common TRAE, grade 3+, was thrombocytopenia (8%). There was one case of CLS, which was grade 3.
Next Steps

Patient enrollment and follow up continues, and additional updates are planned in 2019

Based on these encouraging results, next steps are being evaluated including single agent, combination, and registration-directed trials in patients with relapsed/refractory MF, including in poor-prognosis MF patients with monocytosis, an area of unmet medical need
Ivan Bergstein, M.D., CEO of Stemline, commented "The ELZONRIS clinical presentations showcased at ASH (Free ASH Whitepaper) demonstrate a broad clinical potential in a wide range of malignancies. In particular, we believe that our data in relapsed/refractory CMML and MF provides important clinical proof-of-concept in two indications with unmet medical need, and underscores just two of the many potential expansion opportunities that targeting CD123 offers. At ASH (Free ASH Whitepaper), we continued to ramp-up our disease awareness campaign for BPDCN ahead of the upcoming FDA decision on the ELZONRIS BLA. Also, our sales force is prepared and poised to launch ELZONRIS, should approval be obtained. In parallel, our clinical development team is moving forward with plans to expand efforts, including initiating one or more registration-directed trials in indications beyond BPDN, while also continuing to pursue novel entry points in CD123+ acute myeloid leukemia (AML) and other malignancies."

About BPDCN
To learn more about BPDCN, please visit the disease awareness website at www.bpdcninfo.com.