On March 16, 2018 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, reported financial results for the fourth quarter (Press release, Abeona Therapeutics, MAR 16, 2018, View Source;p=RssLanding&cat=news&id=2338533 [SID1234524853]). The Company will host a call to update investors on recent clinical developments and year-end financial results on Tuesday, March 27th at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-407-9210 (toll free domestic) or 201-689-8049 (International).

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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

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"The past year was marked by several defining events in the company’s history, having advanced our two lead clinical programs, EB-101 in Epidermolysis Bullosa and ABO-102 in MPS IIIA, and initiated our third clinical program, ABO-101 in MPS IIIB. The strong safety and biopotency data observed in our three active clinical trials and the strategic initiative of building an in-house commercial GMP manufacturing facility further strengthens our position in developing gene and cell therapies to treat these devastating and rare pediatric diseases," stated Timothy J. Miller, Ph.D., President and CEO.

4th Quarter and Year-end Summary Financial Results:

Cash position: Cash, cash equivalents and marketable securities as of December 31, 2017 were $137.8 million, compared to $56.5 million as of September 30, 2017. Net cash used in operating activities in the twelve months ended December 31, 2017 was $22.9 million as compared to $13.0 million in the same period in 2016, an increase of $9.9 million.

Offering: During the fourth quarter, on October 19, 2017, Abeona closed an underwritten public offering of 5,750,000 shares of common stock, at a public offering price of $16.00 per share. The gross proceeds to the Company were $92 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by the Company.

Revenues: Revenues were $215 thousand for the fourth quarter of 2017, compared to $256 thousand in the fourth quarter of 2016. Revenues for twelve months ended December 31, 2017 were $837 thousand, compared to $889 thousand in the same period in 2016. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.

Loss per share: Loss per share was $0.19 for the fourth quarter of 2017, compared to a loss per share of $0.18 in the comparable period in 2016. Loss per share was $0.66 for the twelve months ended December 31, 2017, compared to a loss per share of $0.64 in the same period in 2016.
Abeona Recent Highlights:

March 15, 2018: Abeona received FDA Rare Pediatric Disease Designation for ABO-202 gene therapy program in CLN1 disease
February 12, 2018: Received FDA Orphan Drug Designation for ABO-202 program in CLN1 disease
February 8, 2018: Reported top-line data from Phase 1/2 gene therapy trial in MPS IIIA
– ABO-102 results presented at WORLDSymposium for Lysosomal Diseases showed significant time- and dose-dependent reduction of underlying disease pathology, including decreased CSF and urine GAGs (HS fragments) and diminished liver volumes
– Evidence of cognitive benefit at six months post treatment in Cohort 2 and at one year in Cohort 1
– Company receives FDA allowance to lower enrollment age to six months
February 7, 2018: Reported on initial safety and biopotency signals in MPS IIIB gene therapy clinical trial
– ABO-101 is well tolerated and demonstrates early biopotency signals with significant disease-specific heparan sulfate (HS) reductions in cerebral spinal fluid, urine, and plasma and greater than 300-fold increase in NAGLU enzyme activity observed in first subject at 30 days post injection
January 29, 2018: Received FDA Regenerative Medicine Advanced Therapy designation for EB-101 in Epidermolysis Bullosa
December 20, 2017: Enrolled first patient in ABO-101 Phase 1/2 clinical trial for MPS IIIB
November 9, 2017: Enrolled first subject in Spain in ongoing Phase 1/2 clinical trial in MPS IIIA
October 16, 2017: Announced $13.85M grant from leading Sanfilippo syndrome foundations for clinical development of MPS III gene therapies
October 11, 2017: Enrolled first two patients in the Cohort 3 expansion of the Phase 1/2 clinical trial in MPS IIIA
October 6, 2017: Announced top-line one year data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa
October 4, 2017: Abeona broke ground on GMP commercial manufacturing facility for cell and gene therapies
"2017 was transformative for Abeona, with significant progress made towards our goal of building a strong leadership position in rare disease gene therapy development, and manufacturing technology and capability," stated Steven H. Rouhandeh, Executive Chairman. "The momentum will continue in 2018, with the continued enrollment in the high-dose cohort in our ABO-102 Phase 1/2 clinical trial in Sanfilippo syndrome Type A (MPS IIIA), alongside the clinical and regulatory progress in moving our EB program to a pivotal Phase 3 study and the additional work on our own proprietary AIM vector platform."

On March 16, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that financial results for the quarter ended December 31, 2017 (Press release, Stemline Therapeutics, MAR 16, 2018, View Source [SID1234524841]). The Company also reviewed recent clinical and regulatory events, and outlined key upcoming milestones:

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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

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SL-401 in Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

In December 2017, we presented detailed data from the pivotal trial at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, GA.
Based on these trial results and other data, we expect to complete submission of a rolling Biologics License Application (BLA) in the first half of 2018.
Also at ASH (Free ASH Whitepaper), we launched our BPDCN disease awareness campaign which is designed to build awareness of BPDCN and CD123.
Later this year, we anticipate feedback from the European Medicines Agency (EMA) regarding a potential regulatory filing.
Additional Clinical Trials

SL-401 is also being evaluated in clinical trials in additional indications including myeloproliferative neoplasms (MPN) focused on chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML), and multiple myeloma.
We presented data from the ongoing SL-401 MPN trial at ASH (Free ASH Whitepaper):

• In relapsed/refractory CMML (n=11), 71% (5/7) of patients with baseline splenomegaly had a >50% reduction in spleen size by physical examination. One relapsed/refractory CMML patient had a complete response (CR), comprised of a bone marrow complete response (BMCR) and a 100% spleen reduction (5 to 0 cm, or not palpable).

• In relapsed/refractory MF (n=12), 50% (5/10) of patients with baseline splenomegaly had spleen reductions of >25% (range: 29-100%) by physical exam, including 3 patients (30%) with spleen reductions >35%. Notably, 2 of these 3 patients had baseline thrombocytopenia prior to administration of SL-401: 1 patient with platelets <100K/microliter and 1 patient with platelets <50K/microliter.

• Most common treatment-related adverse events (TRAEs) included hypoalbuminemia (33%), thrombocytopenia (33%), and fatigue (29%). Capillary leak was reported in 24% (5/21) evaluable patients: 4 cases were grades 1-2 and 1 case was grade 3. Most common TRAEs (grade 3 or higher) included thrombocytopenia (24%) and anemia (19%).

• Patient enrollment and follow-up is ongoing in this trial. We believe SL-401’s favorable tolerability and preliminary signs of activity support continued development and evaluation of possible registration-directed trial designs. Updates relating to this trial, and further plans for these indications, are expected later this year.

SL-801: the Phase 1 trial in patients with advanced solid tumors is ongoing. Preliminary data were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress in 2017. No dose limiting toxicity or maximum tolerated dose has been reached. Dose escalation is ongoing and we are currently enrolling patients in the ninth dosing cohort.
SL-701: the Phase 2 trial in patients with second-line glioblastoma has been completed. Data were presented at the Society for Neuro-Oncology (SNO) annual meeting in November 2017. SL-701 was well-tolerated and demonstrated evidence of activity, with immunostimulants, as a single agent and in combination with bevacizumab including several major responses and long-term survivors. Data are being analyzed and we expect to provide next steps for the program later this year.
Fourth Quarter 2017 Financial Results Review
Stemline ended the fourth quarter of 2017 with $66.2 million in cash, cash equivalents and investments, as compared to $79.9 million as of September 30, 2017, which reflects cash expenditures of $13.7 million for the quarter. Subsequent to year-end 2017, Stemline completed a follow-on public offering during January 2018 raising $55.5 million in net cash proceeds bringing total cash, cash equivalents and investments as of March 16, 2018 to approximately $106.8 million.

For the fourth quarter of 2017, Stemline had a net loss of $21.7 million, or $0.93 per share, compared with a net loss of $10.0 million, or $0.56 per share, for the same period in 2016.

Research and development expenses were $16.7 million for the fourth quarter of 2017, which reflects an increase of $9.4 million compared with $7.3 million for the fourth quarter of 2016. The higher costs are primarily due to an increase in manufacturing and regulatory expenses in support of our BLA filing and potential commercialization of SL-401. In addition, the higher costs are attributable to increased headcount relating to the build out of various functions, including regulatory and commercial, in support of our BLA filing and potential launch.

General and administrative expenses were $5.2 million for the fourth quarter of 2017, which reflects an increase of $2.1 million compared with $3.1 million for the fourth quarter of 2016. The increase in costs was primarily attributable to pre-launch expenses in support of the potential commercialization of SL-401, if marketing approval from the FDA is obtained, legal expenses, and compensation costs.

On March 16, 2018 Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that financial results for the quarter ended December 31, 2017 (Press release, Stemline Therapeutics, MAR 16, 2018, View Source [SID1234524840]). The Company also reviewed recent clinical and regulatory events, and outlined key upcoming milestones:

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!

SL-401 in Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

In December 2017, we presented detailed data from the pivotal trial at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, GA.

Based on these trial results and other data, we expect to complete submission of a rolling Biologics License Application (BLA) in the first half of 2018.

· Also at ASH (Free ASH Whitepaper), we launched our BPDCN disease awareness campaign which is designed to build awareness of BPDCN and CD123.

Later this year, we anticipate feedback from the European Medicines Agency (EMA) regarding a potential regulatory filing.

Additional Clinical Trials

SL-401 is also being evaluated in clinical trials in additional indications including myeloproliferative neoplasms (MPN) focused on chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML), and multiple myeloma.

We presented data from the ongoing SL-401 MPN trial at ASH (Free ASH Whitepaper):

In relapsed/refractory CMML (n=11), 71% (5/7) of patients with baseline splenomegaly had a >50% reduction in spleen size by physical examination. One relapsed/refractory CMML patient had a complete response (CR), comprised of a bone marrow complete response (BMCR) and a 100% spleen reduction (5 to 0 cm, or not palpable).

In relapsed/refractory MF (n=12), 50% (5/10) of patients with baseline splenomegaly had spleen reductions of >25% (range: 29-100%) by physical exam, including 3 patients (30%) with spleen reductions >35%. Notably, 2 of these 3 patients had baseline thrombocytopenia prior to administration of SL-401: 1 patient with platelets <100K/microliter and 1 patient with platelets <50K/microliter.

· Most common treatment-related adverse events (TRAEs) included hypoalbuminemia (33%), thrombocytopenia (33%), and fatigue (29%). Capillary leak was reported in 24% (5/21) evaluable patients: 4 cases were grades 1-2 and 1 case was grade 3. Most common TRAEs (grade 3 or higher) included thrombocytopenia (24%) and anemia (19%).

· Patient enrollment and follow-up is ongoing in this trial. We believe SL-401’s favorable tolerability and preliminary signs of activity support continued development and evaluation of possible registration-directed trial designs. Updates relating to this trial, and further plans for these indications, are expected later this year.

· SL-801: the Phase 1 trial in patients with advanced solid tumors is ongoing. Preliminary data were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress in 2017.

No dose limiting toxicity or maximum tolerated dose has been reached. Dose escalation is ongoing and we are currently enrolling patients in the ninth dosing cohort.

· SL-701: the Phase 2 trial in patients with second-line glioblastoma has been completed. Data were presented at the Society for Neuro-Oncology (SNO) annual meeting in November 2017. SL-701 was well-tolerated and demonstrated evidence of activity, with immunostimulants, as a single agent and in combination with bevacizumab including several major responses and long-term survivors. Data are being analyzed and we expect to provide next steps for the program later this year.

Fourth Quarter 2017 Financial Results Review

Stemline ended the fourth quarter of 2017 with $66.2 million in cash, cash equivalents and investments, as compared to $79.9 million as of September 30, 2017, which reflects cash expenditures of $13.7 million for the quarter. Subsequent to year-end 2017, Stemline completed a follow-on public offering during January 2018 raising $55.5 million in net cash proceeds bringing total cash, cash equivalents and investments as of March 16, 2018 to approximately $106.8 million.

For the fourth quarter of 2017, Stemline had a net loss of $21.7 million, or $0.93 per share, compared with a net loss of $10.0 million, or $0.56 per share, for the same period in 2016.

Research and development expenses were $16.7 million for the fourth quarter of 2017, which reflects an increase of $9.4 million compared with $7.3 million for the fourth quarter of 2016. The higher costs are primarily due to an increase in manufacturing and regulatory expenses in support of our BLA filing and potential commercialization of SL-401. In addition, the higher costs are attributable to increased headcount relating to the build out of various functions, including regulatory and commercial, in support of our BLA filing and potential launch.

General and administrative expenses were $5.2 million for the fourth quarter of 2017, which reflects an increase of $2.1 million compared with $3.1 million for the fourth quarter of 2016. The increase in costs was primarily attributable to pre-launch expenses in support of the potential commercialization of SL-401, if marketing approval from the FDA is obtained, legal expenses, and compensation costs.

On March 16, 2018 OncoSec Medical Incorporated (OncoSec) (NASDAQ: ONCS), a company developing intratumoral cancer immunotherapies, reported that Daniel J. O’Connor, Chief Executive Officer of OncoSec, will present at the Oppenheimer 28th Annual Healthcare Conference being held March 20-21, 2018 at the Westin New York Grand Central in New York City (Press release, OncoSec Medical, MAR 16, 2018, View Source [SID1234524838]).

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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

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Mr. O’Connor will present OncoSec’s corporate growth strategy and its focus on developing DNA-based intratumoral immunotherapies that utilize the company’s proprietary ImmunoPulse technology, which is designed to enhance the local delivery and uptake of DNA-based immune-targeting agents. In Phase 1 and 2 clinical trials, ImmunoPulse IL-12 has demonstrated a favorable safety profile, evidence of anti-tumor activity in the treatment of various solid tumors, and the potential to reach beyond the site of local treatment to initiate a systemic immune response.

Details of OncoSec’s presentation are as follows:

Event:

Oppenheimer & Co. 28th Annual Healthcare Conference

Date:

March 20, 2018

Time:

1:20 PM (ET)

Location:

The Westin New York Grand Central, New York, NY

In addition to the presentation, management will be available to participate in one-on-one meetings with qualified members of the investor community who are registered to attend the conference.

To view the live webcast, please access the following link at the time of the presentation: https://www.veracast.com/webcasts/opco/healthcare2018/72115452478.cfm.
An archived version of the webcast will be available for 90 days on OncoSec’s website: View Source

On March 16, 2018 Kura Oncology, Inc. (Nasdaq:KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported its participation in the Oppenheimer 28th Annual Healthcare Conference (Press release, Kura Oncology, MAR 16, 2018, View Source [SID1234524837]). Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present an overview of the company on Wednesday, March 21, 2018 at 9:10 a.m. ET / 6:10 a.m. PT. The conference will be held from March 20-21, 2018 in New York City.

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

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A live audio webcast of the presentation will be available in the Investors section of Kura Oncology’s website at www.kuraoncology.com, with an archived replay available for 30 days following the eve