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Aclaris Therapeutics to Present at the 2018 American Hair Research Summit

On May 11, 2018 Paul Changelian, Ph.D., Vice President, Biology, Aclaris Therapeutics, Inc. (NASDAQ:ACRS), reported that it will speak at the 2018 American Hair Research Summit on "Preclinical to Clinical Translation of JAK Kinase Inhibitors" on May 15 (Press release, Aclaris Therapeutics, MAY 11, 2018, View Source [SID1234526536]). The meeting is organized by the American Hair Research Society (AHRS) and will take place May 14-16 in Orlando, Florida.

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Dr. Changelian will review the history of drug discovery targeting Janus kinase (JAK) enzymes. JAK inhibitors initially were conceived as drugs to reduce organ transplant rejection but gradually were found to have utility in autoimmune diseases and oncology. Harnessing the benefits of JAK inhibition for dermatological conditions will require minimizing the potential for over suppression of the immune system and subsequent infections, through strategies such as identifying molecules highly selective for JAK1 and JAK3 inhibition or developing topical formulations with high skin permeability while minimizing systemic absorption. Current efforts to expand the range of diseases that could be targeted with JAK inhibitors will be discussed with emphasis on hair disorders, including alopecia areata and androgenetic alopecia.

Dr. Changelian is known for his key role in the discovery and development of tofacitinib, a pan-JAK inhibitor, while working as a scientist at Pfizer. In 1993, he first heard about the National Institutes of Health’s discovery of an enzyme in the Janus family of kinases involving the immune function. Seven years after initiating the discovery program, Dr. Changelian and the Pfizer immunology team selected tofacitinib for clinical development. Tofacitinib became the first JAK inhibitor approved by the FDA for an autoimmune disease. Dr. Changelian joined Aclaris in 2017 when Aclaris acquired Confluence Life Sciences, Inc., a company founded by former Pfizer scientists.

Abeona Therapeutics Reports First Quarter 2018 Financial Results and Business Highlights

On May 11, 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 first quarter of 2018 (Press release, Abeona Therapeutics, MAY 11, 2018, View Source;p=RssLanding&cat=news&id=2348683 [SID1234526535]). The Company will host a call to update investors on recent clinical developments and quarter financial results on Monday, May 14th 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) or via webcast View Source

"Abeona has made significant progress across multiple fronts, including various regulatory achievements, additional enrollments in our MPS IIIA trial, and the reporting of encouraging initial results for our MPS IIIB trial," stated Carsten Thiel, Ph.D., Abeona’s CEO. "Notably, both our lead clinical programs, EB-101 and ABO-102, were recently granted RMAT designation by the FDA, supporting the strong safety and biopotency evidence from these programs, and their potential to address the unmet medical need for RDEB and MPS IIIA patients."

1st Quarter Summary Financial Results:

– Cash position: Cash and cash equivalents as of March 31, 2018 were $132 million, compared to $137.8 million as of December 31, 2017.
– Revenues: Revenues were $2.6 million for the first quarter of 2018, compared to $186 thousand in the first quarter of 2017. A large portion of the increased quarterly revenues consisted of the recognition of Foundation grants that were announced during the 4th quarter of 2017. A portion of the grants were received in the 4th quarter of 2017 and in the 1st quarter of 2018, and the amount recognized is matched against corresponding expenditures for drug manufacture and clinical readiness. Additional revenues consisted of royalties from marketed products, specifically MuGard. In the quarter, Abeona adopted ASC 606 pertaining to revenue recognition, and therefore there will no longer be any recognition of deferred revenues related to upfront payments from earlier license agreements.
– Loss per share: Loss per share was $0.18 for the first quarter of 2018, compared to a loss per share of $0.13 in the comparable period in 2017.

Abeona Recent Highlights:

April 23, 2018: Announced FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA
April 20, 2018: Announced EMA Grants Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease
April 2, 2018: Announced Appointment of Carsten Thiel, Ph.D., as Chief Executive Officer
March 15, 2018: Announced FDA Grants Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease
February 12, 2018: Announced FDA Grants Orphan Drug Designation for ABO-202 Gene Therapy Program in Infantile Batten 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 show 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: Announced FDA Grants Regenerative Medicine Advanced Therapy Designation for EB-101 Gene Therapy in Epidermolysis Bullosa

"2018 continues to be a year of execution for Abeona. With the expanded leadership, we look to a number of milestones in the year including the commencement of our pivotal Phase 3 trial in EB, the completion of enrollment in our Phase 1/2 trial in MPS IIIA, and significant progress on our in-house GMP manufacturing facility," stated Steven H. Rouhandeh, Abeona’s Executive Chairman. "With the team focusing their efforts and leading the charge on clinical development, we look forward to our continued dialogue with the regulatory bodies and advancing our gene therapies to rare disease patients."

RXi Pharmaceuticals Enters into Material Transfer Agreement with Iovance Biotherapeutics to Study its Self-Delivering RNAi Technology for the Advancement of Immuno-Oncology Therapeutics with Iovance’s Tumor Infiltrating Lymphocytes (TILs) for Solid Tumors

On May 11, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII) reported that it has entered into a research collaboration with Iovance Biotherapeutics to evaluate the potential synergies with RXi’s novel sd-rxRNA therapeutic compounds and Iovance’s autologous cell therapy based on tumor-infiltrating lymphocytes (TILs) for the use in the treatment of cancer (Press release, RXi Pharmaceuticals, MAY 11, 2018, View Source [SID1234526530]).

Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals, stated: "Iovance is the leading company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology. Our agreement will allow Iovance to explore their proprietary TIL technology and our proprietary sd-rxRNA technology. Under this research collaboration, we will further investigate and expand the recently published data with TIL and sd-rxRNA products, which demonstrated potentially enhanced tumor killing activity of TIL." He added that: "As such, we may be able to improve potency of the TIL product and also possibly broaden the applicability of TIL in other tumors types. We see this new research collaboration with the highly experienced Iovance team using TIL, as a major step to advance life-saving treatment approaches for solid tumors, a space where there is still a dire need for improved therapies for these patients."

Leap Therapeutics Reports First Quarter 2018 Financial Results

On May 11, 2018 Leap Therapeutics, Inc. (NASDAQ:LPTX), a biotechnology company developing targeted and immuno-oncology therapeutics, reported financial results for the first quarter ended March 31, 2018 (Press release, Leap Therapeutics, MAY 11, 2018, View Source;p=RssLanding&cat=news&id=2348662 [SID1234526528]).

"We had a strong first quarter, as we presented data from our study of DKN-01 as a monotherapy and in combination with KEYTRUDA (pembrolizumab) in patients with esophagogastric cancer, and dosed the first patients in new trials for both of our programs," commented Christopher K. Mirabelli, Ph.D, President and Chief Executive Officer of Leap Therapeutics. "We also successfully completed a public offering, strengthening our balance sheet and enabling further growth of the company."

Recent Pipeline Highlights:

DKN-01:

Completed enrollment of the dose escalation phase and presented interim data of a clinical trial evaluating DKN-01 and KEYTRUDA (pembrolizumab) in patients with advanced esophagogastric cancer. Data from the dose escalation phase indicated that the combination was well tolerated with early signals of clinical activity. In the high-dose DKN-01 cohort, one of four evaluable patients naïve to anti-PD-1/PD-L1 therapy had a partial response with a 66% reduction in target tumor volume. This patient has a tumor phenotype which is typically less responsive to anti-PD-1 therapy. The study is now enrolling two expansion cohorts in patients with esophagogastric cancer who are naïve to anti-PD-1/PD-L1 therapy (n=40) and patients who are refractory to anti-PD-1/PD-L1 therapy (n=15).
Presented data on the monotherapy activity of DKN-01 in patients with advanced esophagogastric cancer. Of 16 patients evaluable by central imaging analysis, two patients had a partial response and five patients had stable disease, representing a total disease control rate of 43.8%. One patient who had failed prior investigational immunotherapies had a partial response on DKN-01 monotherapy and remained on study for over a year.
Enrolled the first patients in a clinical study evaluating DKN-01 as a monotherapy and in combination with paclitaxel in patients with endometrioid gynecologic cancers, a population of cancers with frequent alterations of the Wnt signaling pathway resulting in increased expression of DKK1.
TRX518:

Enrolled the first patients in a clinical trial evaluating TRX518 in combination with gemcitabine chemotherapy or in combination with KEYTRUDA (pembrolizumab) or Opdivo (nivolumab), anti-PD-1 therapies marketed by Merck (known as MSD outside the United States and Canada) or Bristol-Myers Squibb, respectively.
Business Highlights

Completed a public offering for $16.1 million in gross proceeds, which supports further growth of the company and extends the cash runway into the fourth quarter 2019.
DKN-01 Program Update Call:

On Friday, May 18, 2018 at 12:00PM ET Leap will be hosting a conference call and webcast for the investment community with DKN-01 clinical investigators where the Company will provide a program update. To access the conference call, please dial (866) 589-0108 (US/Canada Toll-Free) or (409) 231-2048 (international) and refer to conference ID 7196723. The presentation will also be webcast live and will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source A replay of the webcast will be available on the Company’s website approximately two hours after the event and will be available for a limited time.

Selected First Quarter 2018 Financial Results

Net loss was $10.6 million for the first quarter 2018, compared to $9.4 million for the same period in 2017. This increase was primarily due to a non-cash change in the fair value of the warrant liability offset by a decrease in stock-based compensation expense.

Research and development expenses were $4.2 million for the first quarter 2018, compared to $6.4 million for the same period in 2017. This decrease was primarily due to a decrease in stock-based compensation expense and a decrease in manufacturing costs related to clinical trial material.

General and administrative expenses were $2.1 million for the first quarter 2018, compared to $3.8 million for the same period in 2017. This decrease was primarily due to a decrease in stock-based compensation expense and a decrease in legal, audit and consulting fees.

Cash, cash equivalents and marketable securities totaled $35.4 million at March 31, 2018. Research and development incentive receivables, current and long term, totaled approximately $1.6 million at March 31, 2018.

Chi-Med to Present at Bank of America Merrill Lynch Annual Health Care Conference

On May 11, 2018 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that Christian Hogg, Chief Executive Officer, will present at the Bank of America Merrill Lynch 2018 Health Care Conference on Wednesday, May 16, 2018 at 1:00 pm PDT in Las Vegas (Press release, Hutchison China MediTech, MAY 11, 2018, View Source [SID1234526527]).

The presentation and Q&A session will be webcast live and can be accessed at www.chi-med.com in the Shareholder Information section under "Events, Circulars & Forms." Investors interested in listening to the live webcast should log on before the start time to download any software required. A replay of the event will be available shortly thereafter, for 90 days.