On August 9, 2018 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a development-stage biopharmaceutical company with multiple technology platforms targeting autoimmune and select cardiovascular indications, reported financial results for the three and six months ended June 30, 2018 and provides a business update (Press release, Caladrius Biosciences, AUG 9, 2018, View Source [SID1234528816]).

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Highlights of the 2018 second quarter and first six months include:

Received regenerative medicine advanced therapy ("RMAT") designation from the U.S. Food and Drug Administration ("FDA") for the Company’s late-stage CD34 cell therapy program CLBS14-RfA for the treatment of refractory angina, which is similar to breakthrough therapy designation, in that it provides increased agency meeting opportunities, the potential for accelerated approval and is reserved for therapies which treat a serious condition while showing preliminary evidence of addressing an unmet medical need;

Received SAKIGAKE designation from the Japan Ministry of Health, Labour and Welfare ("MHLW") for the proprietary CD34 cell therapy CLBS12 for the treatment of no-option critical limb ischemia ("CLI"), which reflects MHLW’s expectation of "prominent effectiveness" based on mechanism-of-action data from non-clinical and early clinical trials and provides an expedited path to potential conditional approval in Japan for products that show sufficient safety evidence and signals of efficacy in a Phase 2 study;

Sold our ownership interest in a non-core development-stage counter-flow centrifugation system to Hitachi Chemical Advanced Therapeutics Solutions for $2.5 million;

Continued enrollment in a Phase 2 clinical trial in Japan with CLBS12 for the treatment of no-option CLI;

Continued enrollment in a Phase 2 clinical trial with the CD34 cell therapy CLBS14-CMD for the treatment of coronary microvascular dysfunction ("CMD"); and

Continued follow-up analysis of The Sanford Project: T-Rex Study Phase 2 clinical trial of CLBS03 in type 1 diabetes after completing enrollment and reporting six-month results on 50% of trial subjects in the first quarter of 2018 that concluded the treatment is well-tolerated and non-futile for therapeutic effect.

Management Commentary

"During the second quarter, we continued to advance our CD34 cell therapy programs. We took a major step forward as we reactivated the Investigational New Drug Application ("IND") for CLBS14-RfA as the sponsor and now have three development programs targeting three indications for our CD34 technology. Additionally, with receipt of RMAT designation for CLBS14-RfA, we are afforded an opportunity to work with the FDA to more rapidly and efficiently advance the development of a therapeutic candidate with the potential to impact a condition with no known effective treatment options and high morbidity. We also advanced our Phase 2 clinical trial in Japan that is evaluating CLBS12 for the treatment of no-option CLI, a condition for which we were granted SAKIGAKE designation from the MHLW in early April. As a result, we now have two potential nearer-term commercial opportunities," said Dr. David J. Mazzo, President and Chief Executive Officer of Caladrius.

"I am also pleased to report that enrollment in our Phase 2 study of CLBS14-CMD for the treatment of coronary microvascular dysfunction continues to progress as expected and that we remain on track to complete patient follow-up and primary endpoint analysis of The Sanford Project: T-Rex Study with anticipated top-line results reported in early 2019," Dr. Mazzo continued. "Finally, as a result of continued fiscal discipline, augmented by $2.5 million of non-dilutive funding received from the sale to Hitachi Chemical Advanced Therapeutics Solutions in June of our rights to a counter-flow centrifugation cell processing device, our cash position remains strong."

Second Quarter Financial Highlights

Research and development expenses for the second quarter of 2018 were $2.1 million, a 50% decrease compared with $4.3 million for the second quarter of 2017. The decline was due to significantly lower costs in our CLBS03 clinical program in type 1 diabetes upon the completion of enrollment in December 2017, which was partially offset by costs related to the initiation of clinical trials in late 2017 and early 2018 for CLBS12 in critical limb ischemia and CLBS14-CMD in coronary microvascular dysfunction, respectively.

General and administrative expenses for the second quarter of 2018 were $2.1 million, compared with $3.4 million for the second quarter of 2017. The decrease was due to the sale of our counter-flow centrifugation system to Hitachi in the second quarter of 2018, which resulted in a one-time $1.4 million gain included in general and administrative expenses.

The net loss from continuing operations for the second quarter of 2018 was $4.1 million, or $0.42 per share, compared with $2.0 million, or $0.22 per share, for the second quarter of 2017.

Six Month Financial Highlights

Research and development expenses for the first six months of 2018 were $4.4 million, a 45% decrease compared with $8.0 million for the first six months of 2017. The decline was due to significantly lower costs in our CLBS03 clinical program in type 1 diabetes upon the completion of enrollment in December 2017, which was partially offset by costs related to the initiation of clinical trials in late 2017 and early 2018 for CLBS12 in critical limb ischemia and CLBS14-CMD in coronary microvascular dysfunction, respectively.

General and administrative expenses for the first six months of 2018 were $5.0 million, compared with $6.1 million for the first six months of 2017. The decrease was due to the sale of our counter-flow centrifugation system to Hitachi in the second quarter of 2018, which resulted in a one-time $1.4 million gain included in general and administrative expenses.

The net loss from continuing operations for the first six months of 2018 was $9.1 million, or $0.95 per share, compared with $8.7 million, or $0.99 per share, for the first six months of 2017.

Balance Sheet Highlights

As of June 30, 2018, Caladrius had cash, cash equivalents and marketable securities of $50.3 million, compared with $60.1 million as of December 31, 2017. Based on existing programs and projections, the Company continues to remain confident that its cash balances and additional grant funding, along with continued disciplined expense management, will allow it to fund its current business plan beyond 2019.

Conference Call

Caladrius’ management will host a conference call for the investment community today beginning at 4:30 p.m. Eastern time to review financial results, provide a Company update and answer questions.

Stockholders and other interested parties may participate in the conference call by dialing (866) 595-8403 (domestic), or (706) 758-9979 (international), and providing conference ID: 8899285. The call will also be broadcast live on the Internet via the Company’s website at www.caladrius.com/investors/news-events.

For those unable to participate on the live conference call, a replay will be available through August 15, 2018, and can be accessed by dialing (855) 859-2056 or (404) 537-3406. All listeners should provide the following replay access code: 8899285.

The webcast replay will be archived on the Company’s website for 90 days at www.caladrius.com.

On August 9, 2018 Histogenics Corporation (Histogenics) (Nasdaq: HSGX), a leader in the development of restorative cell therapies (RCTs) that may offer rapid-onset pain relief and restored function, reported its financial and operating results for the quarter ended June 30, 2018 (Press release, Histogenics, AUG 9, 2018, View Source;p=irol-newsArticle&ID=2363018 [SID1234528593]).

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"Our focus in the second quarter of 2018 was on the NeoCart Biologics License Application submission and we remain on track to announce top-line data in the third quarter of 2018. In preparation for this exciting milestone, we enhanced our management team with the addition of Lynne Kelley as Chief Medical Officer. Lynne’s experience and capabilities in medical and regulatory affairs and product development will be instrumental as we advance the preparation of the upcoming BLA for NeoCart," said Adam Gridley, President and Chief Executive Officer of Histogenics. "We also made important progress on the international expansion of the NeoCart platform alongside MEDINET, our NeoCart development and commercialization partner in Japan, as they prepare for the initiation of the Phase 3 trial in Japan in the second half of the year."

Second Quarter 2018 and Recent Highlights

NeoCart top-line Phase 3 Data Release on Track for Third Quarter of 2018: Histogenics expects to report top-line data from its 249-patient Phase 3 randomized, controlled clinical trial of NeoCart in the third quarter of 2018. The trial is designed to show superiority of NeoCart at one year after treatment as compared to microfracture, the current standard of care, and will follow patients for three years.

Expansion and Enhancement of Executive Team: In July 2018, Histogenics appointed Lynne Kelley as its Chief Medical Officer. In this role, Dr. Kelley will leverage her 20 plus years of executive management and surgical experience in medical affairs, clinical operations, regulatory affairs and product development to establish Histogenics’ medical affairs strategy and build a medical affairs team to support the potential launch of NeoCart. Dr. Kelley will also work with the executive team on the preparation of the upcoming Biologics License Application (BLA) for NeoCart and any related discussions with the United States Food and Drug Administration (FDA).

Held Inaugural Investor Day: In June 2018, Histogenics hosted its first investor day in New York City. Members of Histogenics’ management team discussed the commercialization plan for NeoCart and provided an overview of its Restorative Cell Technology platform. The team was joined by leading orthopedic surgeons who shared their overall experiences with and provided their clinical perspectives on NeoCart, as well as a NeoCart patient from the Phase 3 clinical trial who provided his thoughts on his recovery, specifically the impact NeoCart has had on his ability to return to work and sports activities. The event also included a discussion on the NeoCart mechanism of action based on work conducted as part of Histogenics’ collaboration with Cornell University. A full replay of the webcast is available via the "Investor Relations" page of Histogenics’ website, www.histogenics.com, or by clicking here.
Financial Results for the Second Quarter of 2018

Loss from operations was $(7.3) million in the second quarter of 2018, compared to $(6.4) million in the second quarter of 2017. The increase in operating expenses was due to an increase in both research and development expenses and general and administrative expenses.

Research and development expenses were $4.5 million in the second quarter of 2018, compared to $4.2 million in the second quarter of 2017. The increase was primarily due to increases in consulting, salaries and materials in connection with the potential submission of a BLA for NeoCart with the FDA and was partially offset by a reduction in patient costs related to the NeoCart Phase 3 clinical trial, for which enrollment was completed in June 2017. General and administrative expenses were $2.8 million in the second quarter of 2018, compared to $2.2 million in the second quarter of 2017. The increase was primarily due to higher salaries and consulting expenses related to increased activities to support the potential commercialization of NeoCart.

Net loss attributable to common stockholders was $(3.7) million in the second quarter of 2018, or $(0.13) per share, compared to $(5.5) million, or $(0.25) per share, in the second quarter of 2017. The decrease in net loss attributable to common stockholders is primarily due to the conversion of convertible preferred stock issued in connection with the 2016 private placement into common stock and a change in the fair value of the warrant liability which generated a gain in the second quarter of 2018, both of which were partially offset by an increase in operating expenses.

As of June 30, 2018, Histogenics had cash, cash equivalents and marketable securities of $8.8 million, compared to $8.0 million at December 31, 2017. Histogenics believes its current cash position will be sufficient to fund its operations into the fourth quarter of 2018.

Conference Call and Webcast Information

Histogenics management will host a conference call on Thursday, August 9, 2018 at 8:30 a.m. EDT. A question-and-answer session will follow Histogenics’ remarks. To participate on the live call, please dial (877) 930-8064 (domestic) or (253) 336-8040 (international) and provide the conference ID "6679509" five to ten minutes before the start of the call.

To access a live audio webcast of the presentation on the "Investor Relations" page of the Histogenics website, please click here. A replay of the webcast will be archived on Histogenics’ website for approximately 45 days following the presentation.

On August 9, 2018 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, reported financial results for the second quarter ended June 30, 2018 (Press release, Aeglea BioTherapeutics, AUG 9, 2018, View Source [SID1234528707]).

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"We made good progress in the second quarter with our clinical programs, and we completed a follow-on financing that sets the stage for an exciting second half of the year," said Anthony G. Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "We are looking forward to providing clinical updates at a number of medical conferences in the fall, including reporting the latest interim data from both our Arginase 1 Deficiency and cancer development programs. We also will provide updates on our clinical trial enrollment and our progress with one of our research enzyme programs, which we believe shows promise for treating the metabolic disorder homocystinuria."

"With the management team we now have in place, Aeglea is ready to take the next step in advancing therapies with the potential to improve the lives of patients with devastating diseases. I believe the Company is well positioned for future growth with our current pipeline programs and our expanded in-house drug-hunting capabilities," added Dr. Quinn.

Recent Highlights

In July, the Company’s Board of Directors named Anthony G. Quinn, M.B. Ch.B, Ph.D. as president and chief executive officer. Dr. Quinn has been a member of the Board of Directors since 2016 and had served as interim CEO since July 2017. Prior to joining Aeglea, Dr. Quinn served as executive vice president, head of research and development, and chief medical officer at Synageva Biopharma Corp., prior to its acquisition by Alexion Pharmaceuticals.
In July, the Company announced the appointment of Bryan Lawlis, Ph.D., as an independent director. Dr. Lawlis served as CEO of Itero Biopharmaceuticals, LLC from 2011 to 2017 and from 2007 to 2011 was co-founder and CEO of Itero Biopharmaceuticals, Inc. He is currently on the boards of Biomarin Pharmaceutical, Inc., Geron, Inc., and Coherus Biosciences, Inc.
Upcoming Events

Dr. Quinn will present a corporate update at the Wells Fargo Healthcare Conference being held September 5 – 6 in Boston, MA. Details regarding the date and time of the presentation and webcast will be announced before the conference.
Aeglea will present new interim Phase 1/2 clinical trial data demonstrating clinically relevant treatment effects in patients with Arginase 1 Deficiency at the 2018 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium being held September 4 – 7 in Athens, Greece.
Title: Improvements in Arginase 1 Deficiency-related disease manifestations following plasma arginine reduction with pegzilarginase
Poster #P-164
Presentation Date/Time: Wednesday, September 5 at 5:45 p.m. – 8:30 p.m. EET and Thursday, September 6 at 12:15 p.m. – 1:15 p.m. EET
Aeglea will deliver three poster presentations detailing clinical and preclinical data at the American Society of Human Genetics (ASHG) 2018 Annual Meeting being held October 16 – 20 in San Diego, California (schedule details to be announced):
Interim Phase 1/2 clinical trial data for the treatment of Arginase 1 Deficiency that will include additional clinical insights from recently enrolled adult and pediatric patients, as well as from longer-term dosing in previously enrolled patients
Title: Improvements in Arginase 1 Deficiency-related disease manifestations following plasma arginine reduction with pegzilarginase: early Phase 2 results
The effects of a novel homocysteine degrading enzyme in a preclinical model of homocystinuria.
Title: Improved survival and amelioration of disease-related liver pathology in a mouse model of homocystinuria with a novel homocysteine degrading enzyme
Summary of disease manifestation in Arginase 1 Deficiency case reports from scientific literature
Title: Clinical features of Arginase 1 Deficiency: Review of Literature Case Series
Aeglea will present interim Phase 1 advanced solid tumor clinical trial data on melanoma expansion cohorts at the 2018 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress being held October 19 – 23 in Munich, Germany.
Title: Initial cohort expansion results of sustained arginine depletion with pegzilarginase in melanoma patients in a Phase 1 advanced solid tumor trial
Abstract #1269P
Presentation Date/Time: Sunday, October 21 at 1:35 p.m. – 2:35 p.m. CEST
Second Quarter 2018 Financial Results

As of June 30, 2018, Aeglea had available cash, cash equivalents and marketable securities of $72.2 million, which includes $37.7 million in net proceeds from a follow-on public offering that closed in April 2018. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations to the middle of 2020.

Aeglea recognized grant revenues of $2.4 million in the second quarter of 2018, compared with $1.5 million in the second quarter of 2017. The grant revenues were the result of a $19.8 million research grant received from the Cancer Prevention and Research Institute of Texas (CPRIT). The revenue increase was primarily due to higher qualifying expenditures associated with the clinical trials for pegzilarginase in cancer patients in the second quarter of 2018 compared with the second quarter of 2017. Additionally, the grant contract ended in May 2018 with the full $19.8 million grant recognized as revenue over the life of the award. As of June 30, 2018, Aeglea had a remaining grant receivable totaling $4.3 million.

Research and development expenses totaled $9.1 million for the second quarter of 2018, compared with $5.8 million for the second quarter of 2017. The increase was primarily due to expanded clinical activity for Aeglea’s lead product candidate, pegzilarginase, as Aeglea advanced a Phase 1/2 clinical trial in patients with Arginase 1 Deficiency and initiated single-agent cohort expansions in a Phase 1 clinical trial for advanced solid tumor patients and a Phase 1/2 combination trial in patients with small cell lung cancer.

General and administrative expenses totaled $2.9 million for the second quarter of 2018, compared with $2.4 million in the second quarter of 2017. This increase was primarily due to additional employee compensation costs related to the building out of Aeglea’s management team as well as to support expanding research and development activities. Non-cash stock compensation expense accounted for $0.2 million of the increase.

Net loss totaled $9.4 million and $6.6 million for the second quarter of 2018 and 2017, respectively, with non-cash stock compensation expense of $1.0 million and $0.7 million for the second quarter of 2018 and 2017, respectively.

Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Aeglea also announced today that the Compensation Committee of its Board of Directors has granted non-qualified stock options to purchase an aggregate of 2,400 shares of Aeglea’s common stock to two new employees under Aeglea’s 2018 Equity Inducement Plan.

The 2018 Equity Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously an employee or non-employee director of Aeglea (or following a bona fide period of non-employment), as an inducement material to such individual’s entering into employment with Aeglea, pursuant to Rule 5635(c)(4) of the NASDAQ Listing Rules.

The options have an exercise price of $8.98 per share, which is equal to the closing price of Aeglea’s common stock on August 7, 2018. Each of the option awards vests as to 25% of the shares on the one-year anniversary of its grant, with the remainder of the shares vesting ratably over 36 months thereafter.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically degrades the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a debilitating urea cycle disorder caused by deficiency of a key arginine metabolizing enzyme that leads to severe and progressive hyperargininemia-related neurological abnormalities, hyperammonemia and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. The Company’s interim Phase 1/2 data demonstrated clinically relevant treatment effects and rapid and sustained lowering of plasma arginine in Arginase 1 Deficiency patients.

About Pegzilarginase in Cancer

Pegzilarginase is an enhanced human arginase that enzymatically degrades the amino acid arginine. In some cancers, tumor cells stop producing specific amino acids and must acquire them from the blood, making the tumor cells susceptible to starvation through depletion of those amino acids. Aeglea is developing pegzilarginase to exploit vulnerabilities in some cancers that lead to an increased dependency on extracellular arginine. Pegzilarginase targets these arginine dependent cancers by depleting blood arginine levels to below the normal range. Preclinical data demonstrated that the resulting arginine starvation inhibits proliferation, induces cell death, increases turnover of cell components and promotes anti-tumor immune responses. The Company’s Phase 1 data in advanced solid tumors demonstrated that pegzilarginase was well tolerated at doses that produced marked and sustained reductions in blood arginine levels below the normal range.

On August 9, 2018 IntelGenx Technologies Corp. (TSX-V:IGX) (OTCQX:IGXT) (the "Company" or "IntelGenx")reported financial results for the second quarter ended June 30, 2018 (Press release, IntelGenx, AUG 9, 2018, View Source [SID1234528595]). All dollar amounts are expressed in U.S. currency and results are reported in accordance with United States generally accepted accounting principles except where noted otherwise.

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2018 Second Quarter Financial Highlights:

Total revenue was $234,000, which reflected decreases in deferred revenues on monetization of $915,000.
Negative adjusted EBITDA was ($1.9 million), compared to negative adjusted EBITDA of ($390,000) in the same period last year.
Cash and short-term investments totalled $3.7 million as at June 30, 2018.
Recent Developments:

Received a Notice of Allowance from the Canadian Intellectual Property Office for the Company’s Canadian Patent Application Number 2,998,223 entitled "Loxapine Film Oral Dosage Form", covering the use of loxapine in an oral transmucosal film for the treatment of schizophrenia or bipolar 1 disorder. This was the Company’s first patent allowed in Canada and the first Canadian patent for its VersaFilm technology.
Announced that the European Patent Office issued a "Notice of Intention to Grant" for the Company’s European Patent Application Number 14832172.2 entitled, "Instantly Wettable Oral Film Dosage Form Without Surfactant or Polyalcohol." This was the first key patent allowed in Europe for the Company’s VersaFilm technology and covers the formulation of its Rizaport product.
Announced the publication of a peer-reviewed paper in the March 2018 issue of Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association, which highlighted one of the most significant unmet medical needs that may be addressed by IntelGenx’s Montelukast VersaFilm product candidate.
Announced the settlement of all Subxone patent litigation between the Company, Par Pharmaceutical, Inc., Indivior UK Limited and Aquestive Therapeutics, Inc.
"The advancement of our product pipeline toward commercialization continued to be a key priority in the 2018 second quarter," commented Dr. Horst G. Zerbe, President and CEO of IntelGenx. "To that end, we were pleased that two patent allowances – one in Canada and the other in Europe – imparted protection for our proprietary VersaFilm technology platform in those jurisdications for the first time, providing additional validation of the innovative work being carried out by our talented R&D team. More recently, we were also excited to learn from our Spanish marketing partner, Grupo Juste (now Exeltis Healthcare), that the Committee for Medicinal Products for Human Use has included RIZAPORT in the list of medicines within the Informative Note from its July 2018 meeting, one of the final steps needed before marketing authorization can be obtained from the Spanish Agency of Medicines and Medical Devices."

Financial Results:

Total revenues for the three-month period ended June 30, 2018 amounted to $234,000, compared to $1.1 million for the three-month period ended June 30, 2017. The decrease for the three-month period ended June 30, 2018 compared to last year’s corresponding period is mainly attributable to a decrease in deferred revenues on monetization of $915,000.

Operating costs and expenses were $2.4 million for the second quarter ended June 30, 2018, versus $1.7 million for the corresponding quarter in 2017. The increase for the three-month period ended June 30, 2018 is mainly attributable to a $203,000 increase in Research and Development expenses and a $496,000 increase in Selling, General and Administrative expenses.

For the second quarter ended June 30, 2018, the Company had an operating loss of $2.1 million, compared to an operating loss of $613,000 for the comparable period of 2017.

Net comprehensive loss was $2.4 million, or $0.04 on a basic and diluted per share basis, for the three-month period ended June 30, 2018, compared to a net comprehensive loss of $550,000, or $0.01 on a basic and diluted per share basis, for the comparable period of 2017.

As of June 30, 2018, the Company’s cash and short-term investments totalled $3.7 million.

Conference Call Details:

IntelGenx will host a conference call to discuss its second quarter 2018 financial results today, Aug 9, 2018, at 4:30 p.m. ET. The dial-in number for the conference call is (833) 231-8269 (Canada and United States) or (647) 689-4114 (International), conference ID 5888143. The call will also be webcast live and archived for twelve months at www.intelgenx.com.

On August 9, 2018 Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported its financial results for the quarter ended June 30, 2018 (Press release, Stemline Therapeutics, AUG 9, 2018, View Source [SID1234528708]). The Company also reviewed recent clinical and regulatory events, and outlined key upcoming milestones:

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ELZONRIS (SL-401, tagraxofusp) – BLA submission completed

We completed submission of a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for ELZONRIS, which has been granted breakthrough therapy designation (BTD), for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). If successful, we anticipate acceptance of our BLA within 60 days of submission (i.e. in the coming weeks) and potential U.S. marketing approval by 1Q19, or possibly sooner.

Data from our pivotal trial in patients with BPDCN was the subject of an oral presentation at the 23rdCongress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Sweden.

We anticipate feedback from the European Medicines Agency (EMA) later this year regarding a potential ELZONRIS regulatory filing in Europe.

In preparation for potential marketing approval, we continue to build out our pre-launch and commercial activities, including our disease awareness campaign targeting key stakeholders including hematologist-oncologists, dermatologists, and pathologists.
ELZONRIS – Other potential indications

ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF).

Clinical data from ongoing trials in patients with CMML and MF were selected for presentation at EHA (Free EHA Whitepaper) in June.

In relapsed/refractory CMML (n=16 patients), ELZONRIS demonstrated 100% (8/8) spleen responses in evaluable patients with baseline splenomegaly by physical exam and 2 bone marrow complete responses (BMCRs), coupled with a tolerable safety profile. Given the results observed to date, we are currently formulating registrational trial designs.

In relapsed/refractory MF (n=15 patients), ELZONRIS demonstrated 50% (6/12) spleen responses in evaluable patients with baseline splenomegaly (>5 cm palpable below the costal margin by physical exam), coupled with a tolerable safety profile. Given the results observed to date, we are currently evaluating next steps including possible registrational trial designs.
SL-801

Data from the ongoing Phase 1 trial of SL-801 in patients with advanced solid tumors were presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June. Patient enrollment and dose escalation continues.
SL-701

Data from the Phase 2 trial of SL-701 in patients with second-line glioblastoma (GBM) were presented at the 2018 ASCO (Free ASCO Whitepaper) meeting in June. Notably, there were long-term (>12 month) overall survivors in the SL-701+bevacizumab cohort which consisted primarily of patients who demonstrated an elevated immune response (i.e. potentially representing an "immunocompetent" population). Further analyses, including registration-directed designs are ongoing.
Ivan Bergstein, MD, CEO of Stemline, commented, "We have completed our rolling BLA submission and are quickly approaching the very important milestone of a potential BLA acceptance for filing by the FDA. We continue to advance closer to our ultimate goal of potentially making ELZONRIS widely available to patients with BPDCN. In parallel, ELZONRIS continues to generate very promising clinical data in additional indications including CMML and MF, two settings of unmet medical need for which we are actively evaluating registration pathways. Additionally, we continue to expand our commercial infrastructure including the build out of our sales and marketing teams ahead of our potential approval."

Second Quarter 2018 Financial Results Review
Stemline ended the second quarter of 2018 with $97.1 million in cash, cash equivalents and investments, with a cash burn of $17.6 million in the second quarter. The Company ended the second quarter of 2018 with 30.9 million shares outstanding. For the second quarter of 2018, Stemline had a net loss of $18.9 million, or $0.66 per share, compared with a net loss of $15.5 million, or $0.66 per share, for the same period in 2017.

Research and development expense was $11.2 million for the quarter ended June 30, 2018, compared with $11.5 million for the quarter ended June 30, 2017, representing a decrease of $0.3 million.

General and administrative expense was $8.6 million for the quarter ended June 30, 2018, compared with $4.5 million for the quarter ended June 30, 2017, representing an increase of $4.1 million. The increase in expense was primarily attributed to a $3.0 million increase in pre-launch expenses in support of our potential commercialization of ELZONRIS in BPDCN, if marketing approval from the FDA is received. Additionally, the higher expense was also due to an increase of non-cash stock-based compensation expense and increased headcount.