On May 8, 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 quarter ended March 31, 2018 (Press release, Aeglea BioTherapeutics, MAY 8, 2018, View Source [SID1234526225]).

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"The first quarter was a terrific start to our year, with a number of positive and encouraging developments in our lead clinical investigational program, pegzilarginase," said Anthony G. Quinn, M.B Ch.B, Ph.D., interim chief executive officer of Aeglea. "I’m excited that we are seeing the first evidence that marked and sustained reductions in plasma arginine with pegzilarginase translated into clinically relevant treatment effects for two patients with Arginase 1 Deficiency. We plan to continue to build on this momentum by reporting additional repeat dose data in patients with Arginase 1 Deficiency in the third quarter of 2018 and finalizing our pivotal study design by the end of the year. In addition, we expect to report topline safety and clinical data from our cancer trials in the fourth quarter of 2018.

"Our April follow-on offering provides us with capital to continue to advance our planned operations and further develop our capabilities as we transition into a pivotal study and start planning for a commercial launch. Our strong cash position and our worldwide commercial rights for pegzilarginase position us favorably to build on recent clinical achievements with investments focusing on accelerating our clinical and pipeline programs."

Corporate Update

Arginase 1 Deficiency:

Aeglea presented initial data that it believes demonstrated clinically relevant treatment effects with pegzilarginase in two Arginase 1 Deficiency patients after only eight weeks of dosing and confirmed the utility of standardized assessment tools in quantifying disease manifestations at the 2018 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics (ACMG) in April. This built upon data presented at the 2018 Annual Meeting of The Society for Inherited Metabolic Disorders (SIMD) that demonstrated pegzilarginase produces marked and sustained reductions in plasma arginine in patients with Arginase 1 Deficiency.
Aeglea expects to report additional pediatric and adult repeat dose data in patients with Arginase 1 Deficiency in the third quarter of 2018.
Cancer:

Aeglea dosed the first patients with pegzilarginase in two small cell lung cancer (SCLC) trials: the single-agent Phase 1 cohort expansion and the Phase 1/2 combination trial with KEYTRUDA, an anti-PD-1 therapy marketed by Merck (known as MSD outside the United States and Canada).
Aeglea presented Phase 1 dose escalation data regarding pegzilarginase in patients with advanced solid tumors at the 2018 Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April.
The Company expects to report topline data, including safety and clinical activity, for the advanced solid tumor cohort expansions and the SCLC combination trial in the fourth quarter of 2018.
Aeglea expects to initiate Phase 2 of its combination trial for patients with SCLC in the third quarter of 2018.
Upcoming Events

Aeglea will participate and provide a corporate update at the UBS Global Healthcare Conference in New York, May 21-23, 2018.

First Quarter 2018 Financial Results

As of March 31, 2018, Aeglea had available cash, cash equivalents and marketable securities of $43.5 million, which excludes approximately $37.7 million in net proceeds from a follow-on public offering which closed on April 23, 2018. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the middle of 2020.

Aeglea recognized grant revenues of $1.5 million in the first quarter of 2018, compared with $1.0 million in the first 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 first quarter of 2018 compared with the first quarter of 2017.

Research and development expenses totaled $6.8 million for the first quarter of 2018, compared with $4.9 million for the first 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 three single-agent cohort expansions in advanced solid tumor patients and a Phase 1/2 combination trial with KEYTRUDA in patients with small cell lung cancer.

General and administrative expenses totaled $2.8 million for the first quarter of 2018, compared with $2.3 million in the first quarter of 2017. This increase was primarily due to additional employee headcount and compensation costs to further strengthen Aeglea’s management team and support expanding research and development activities.

Net loss totaled $8.1 million and $6.2 million for the first quarter of 2018 and 2017, respectively.

Inducement Grants

Aeglea also announced today that the Compensation Committee of its Board of Directors has granted non-qualified stock options to purchase an aggregate of 21,000 shares of Aeglea’s common stock to three 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 $9.76 per share, which is equal to the closing price of Aeglea’s common stock on May 3, 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

On May 8, 2018 Advanced Proteome Therapeutics Corporation ("APC" or the "Company") (TSXV: APC) (FSE: 0E8) reported an update of progress during the third quarter, FY 2018 (Press release, Advanced Proteome Therapeutics, MAY 8, 2018, View Source [SID1234526224]). The Company, in its pursuit of commercial success is vigourously applying innovative core principles ((Marketwired – January 31, 2018) to advance its proprietary technology and create superior versions of the antibody-drug conjugate (ADC), with the ultimate goal of perfecting this type of therapeutic agent and standardizing the development process.

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In pursuing a critical milestone in animal studies of demonstrable efficacy of its lead ADC candidate, the Company has recently outsourced the scaling up for production of material to be tested, ensuring that the quality control of the final product conforms to industry standards.

In this regard, APC is pleased to report that the impressive potency against cancer cell lines previously announced with our Heidelberg Pharma partners (Marketwired – January 08, 2018), has been replicated and substantiated by an independent laboratory (National Research Council of Canada (NRC)) and the technology effectively transferred to NRC and scaled to an intermediate level, attesting to its robustness. By overcoming these traditional barriers, the company is positioned for final scale-up and animal testing during the 4th quarter, activities which have now been scheduled.

During the 3rd quarter, APC has also been actively engaged in converting the series of related antibodies provided under the CCAB/University of Toronto Agreement (Marketwired-October 19, 2016) into bona fide ADCs for evaluation as to their suitability for animal testing. The target criteria involving site-selectively linking up two copies of toxin moieties per antibody, defined by an Industrial Research Assistance Program (NRC IRAP) supported initiative (Marketwired – November 20, 2017, has been achieved.

From this accelerated effort, a clearer understanding of how to match toxins to APC’s linker technology has emerged and has resulted in the identification of site-selectively conjugated ADCs with excellent biophysical properties that qualify for scale-up, and potentially animal testing as well, subject to results in preliminary cell cytotoxicity studies. This project is ongoing and should be completed in the 4th quarter, wherein a decision regarding a lead candidate will be made.

Work has also been initiated that directly relates to the Collaboration and Option agreement with the ImmunoBiochem Corporation (Feb. 07, 2018 (GLOBE NEWSWIRE)) with the goal of producing ADCs targeting triple-negative breast cancer. Design elements for construction of target ADCs have been formulated and synthetic work has begun, to enable feasibility studies.

During the 3rd quarter the company has been fortunate in recruiting Scientists/Physicians possessing high level expertise in antibody technology and profound clinical experience with ADCs to the Scientific Advisory Board to align with the company’s objectives and projected business activities in the path ahead. The Company is also gratified that

the relevant parties have amended the License for space to extend the current term of residency at JLABS until May 1st, 2019.

On May 8, 2018 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN:ATNM) ("Actinium" or "the Company"), reported that it will be attending and presenting at the 19th Annual Bio€quity Europe Conference being held on May 14 – 16, 2018 at the Het Pand Convention Center in Ghent, Belgium (Press release, Actinium Pharmaceuticals, MAY 8, 2018, View Source [SID1234526223]). Details of Actinium’s presentation are as follows:

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Date: Tuesday, May 15, 2018
Time: 4:20 p.m. CEST
Room: Level 1+, Priorzaal Room
Venue: The Het Pand Convention Center

Management will conduct one-on-one meetings with investors during the conference. To arrange a meeting with Actinium, please contact, Steve O’Loughlin, Actinium’s Principal Financial Officer at [email protected].

About the 19th Annual Bio€quity Europe Conference

Bio€quity Europe pioneered the turf-neutral concept, creating an open door for all members of the financial community and business development and licensing professionals to do business with independently selected presenting companies.

Now celebrating its 19th meeting, Bio€quity Europe is the seminal industry event for financial dealmakers looking for investor-validated life science companies positioning themselves to attract capital, and for pharmaceutical licensing professionals to assess top prospects. Bio€quity Europe has showcased more than 700 leading European companies to thousands of investment and pharma business development professionals. Delegates from 24 nations attended Bio€quity Europe last year in Paris. To learn more, please click this link.

On May 8, 2018 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in aesthetic and medical dermatology, and immunology, reported financial results for the first quarter 2018 and provided an update on its clinical development and commercial programs (Press release, Aclaris Therapeutics, MAY 8, 2018, View Source [SID1234526222]).

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"The first quarter of 2018 was a busy one as we prepared for the launch of ESKATA (hydrogen peroxide) Topical Solution, 40% (w/w), the first and only FDA-approved topical treatment for raised seborrheic keratosis (SK). We held the ESKATA Launch Meeting last week, and ESKATA is now officially available for physicians and their patients," said Brett Fair, Chief Commercial Officer of Aclaris.

Commercial Update:

Successful rollout and implementation of the ESKATA Early Experience Initiative (EEI).

Program expanded to over 700 accounts to accommodate market demand for ESKATA.

Ongoing in-service programs to support successful training and product integration.

Positive initial ESKATA feedback from EEI program captured in physician and patient post-application surveys.

Aclaris sales force successful in generating a significant number of ESKATA pre-orders from targeted accounts ahead of official launch meeting.

Commenced health care provider (HCP) order processing and shipping the week of April 23, 2018.

ESKATA Launch Campaign Highlights:

ESKATA Launch Meeting held April 30 – May 4, 2018; Unveiled New ESKATA Campaign; sales force trained on new tools and resources to support a successful ESKATA launch.

ESKATA Consumer website (www.eskata.com) launched May 1, 2018; includes "Find A Doctor" resource for patients seeking ESKATA treatment.

ESKATA HCP website (www.eskatahcp.com) updated with new campaign and downloadable tools/resources for offices.

ESKATA Peer-to-peer Speaker Programs beginning in May 2018.
"In March, we announced positive results from the 3-month follow-up portion of the WART-203 Phase 2 clinical trial of A-101 45% Topical Solution (A-101 45%) for the treatment of common warts (verruca vulgaris). We are also advancing our topical Janus kinase (JAK) inhibitor programs, with results from multiple Phase 2 trials expected this year. As our early-stage pipeline compounds advance towards the clinic, we continue to progress towards our goal of becoming a vertically integrated, commercial-stage biopharmaceutical company with a robust clinical-stage pipeline and drug discovery engine," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris.

Clinical Pipeline Update:

A-101 45% Topical Solution

In March 2018, announced positive results from the 3-month, post-treatment, follow-up evaluation period of the twice-weekly placebo-controlled Phase 2 trial (WART-203) of A-101 45% Topical Solution (A-101 45%), an investigational new drug consisting of a proprietary high-concentration stabilized hydrogen peroxide topical solution being developed as a prescription treatment for common warts (verruca vulgaris).

Over the 3-month post-treatment follow-up period, clinically and statistically significant greater improvements in common wart reduction and clearance vs. placebo were observed among subjects treated with A-101 45%.

Scheduled an End of Phase 2 meeting with the FDA for mid-2018, and plan to initiate two pivotal Phase 3 trials in the second half of 2018.
JAK Inhibitor Candidates

AA-202 Topical – an ongoing Phase 2 clinical trial of ATI-502 for the topical treatment of alopecia areata (AA). This trial will evaluate the pharmacokinetics, pharmacodynamics and safety of ATI-502 compared with placebo in 12 patients with AA. This randomized, double-blind clinical trial is being conducted at two investigational centers within the United States, and topline data are expected in the first half of 2018. After completing the 28-day portion of the trial, patients will then enter a 6-month open label extension during which all patients will receive drug.

AUATB-201 Topical – an ongoing Phase 2 open-label clinical trial of ATI-502 for the topical treatment of AA. This trial will evaluate the effect of ATI-502 on the regrowth of eyebrows in up to 24 patients with AA. This trial is being conducted at two investigational centers in Sydney and Melbourne, Australia, and topline qualitative data are expected mid-2018.

AA-201 Topical – an ongoing Phase 2 dose ranging trial of ATI-502 for the topical treatment of AA. This trial will evaluate the effect of two concentrations of ATI-502 on the regrowth of hair in a randomized, double-blinded, parallel-group, vehicle-controlled trial in up to 120 patients with AA. This trial is being conducted in the United States and data are expected by year end 2018.

VITI-201 Topical – an ongoing Phase 2 open-label clinical trial of ATI-502 for the topical treatment of vitiligo. This trial will evaluate the effect of ATI-502 on the repigmentation of facial skin in up to 24 patients with vitiligo and data are expected in the first half of 2019.

AGA-201 Topical – an ongoing Phase 2 open-label clinical trial of ATI-502 for the topical treatment of androgenetic alopecia (AGA), also known as male/female pattern hair loss. This trial will evaluate the effect of ATI-502 on the regrowth of hair in up to 24 patients with AGA and data are expected in first half of 2019.

AUAT-201 Oral – a planned Phase 2 dose ranging trial of ATI-501, an oral JAK inhibitor for the treatment of AA, which is anticipated to begin in the first half of 2018. This trial will evaluate the effect of two concentrations of ATI-501 on the regrowth of hair in a randomized, double-blinded, parallel-group, vehicle-controlled trial in 120 to 160 patients with AA. This trial will be conducted in the United States and data are expected in mid-2019.
ATI-450 (MK-2 Inhibitor)

Investigational New Drug application on track for submission to the FDA in mid-2019.
Recent Corporate Highlights

Exclusively licensed the Canadian rights to commercialize A-101 40% Topical Solution for the treatment of raised seborrheic keratoses to Cipher Pharmaceuticals.

Appointed Bryan Reasons as a director and Chairman of the Audit Committee.
Financial Highlights

Liquidity and Capital Resources

As of March 31, 2018, Aclaris had aggregate cash, cash equivalents and marketable securities of $187.0 million compared to $208.9 million as of December 31, 2017. The $21.9 million decrease during the quarter ended March 31, 2018 included:

Net loss of $30.2 million, offset by $5.4 million of non-cash stock-based compensation expense, depreciation and amortization, $2.1 million of net cash provided by changes in operating assets and liabilities, and $0.9 million for a non-cash expense associated with an increase in the fair value of a contingent consideration liability.

$0.3 million of cash used for purchases of property and equipment.

$0.4 million in cash proceeds from the exercise of employee stock options.
Aclaris anticipates that its cash, cash equivalents and marketable securities as of March 31, 2018 will be sufficient to fund its operations into the second half of 2019, without giving effect to any potential new business development transactions or financing activities.

First Quarter 2018 Financial Results

Net loss was $30.2 million for the first quarter of 2018, compared to $12.6 million for the first quarter of 2017.

Revenue of $1.1 million and cost of revenue of $1.0 million for the first quarter of 2018 related to Aclaris’s contract research business acquired in August 2017.

Total operating expenses for the first quarter of 2018 were $31.1 million, compared to $12.9 million for the first quarter of 2017.
Research and development expenses were $13.6 million for the first quarter of 2018, compared to $7.8 million for the first quarter of 2017. The increase of $5.8 million was primarily attributable to a $2.7 million increase in expenses related to the preclinical and clinical development of Aclaris’s JAK inhibitor portfolio, a $1.3 million increase in medical affairs activities and early-stage drug discovery, a $0.9 million increase in fair value of the contingent consideration liability, a $0.8 million increase in Aclaris’s A-101 45% topical solution program as Phase 2 clinical trials were initiated in June 2017, and a $0.6 million increase in personnel-related expenses, including stock-based compensation, due to increased headcount. These increases were partially offset by a $0.5 million decrease in ESKATA development expenses in the first quarter of 2018.

Sales and marketing expenses were $11.2 million for the first quarter of 2018, compared to $1.4 million for the first quarter of 2017. The $9.8 million increase is mainly due to increases in direct marketing and professional fees, as well as other sales and marketing expenses of $5.8 million, in preparation for the commercial launch of ESKATA in the second quarter of 2018. Personnel expenses, including stock-based compensation, increased by $4.0 million as Aclaris completed the hiring of its field sales force in the first quarter of 2018.

General and administrative expenses were $6.3 million for the first quarter of 2018, compared to $3.7 million for the first quarter of 2017. The increase of $2.6 million was primarily attributable to $1.6 million in higher personnel-related expenses, including stock-based compensation, due to increased headcount, a $0.4 million increase in professional and legal fees, and a $0.5 million increase in facility, support and other general and administrative expenses.
2018 Financial Outlook

Aclaris reiterated its expected 2018 GAAP research and development (R&D) expenses to be in the range of $67 to $75 million, including estimated stock-based compensation of $9 million. The anticipated increase in R&D expenses in 2018 is mainly due to the planned execution of Phase 2 clinical trials in AA, AGA, and vitiligo, two planned pivotal Phase 3 trials in common warts, and the development of our early stage pipeline compounds.

Aclaris reiterated its expected 2018 GAAP selling, general and administrative (SG&A) expenses to be in the range of $80 to $86 million, including estimated stock-based compensation of $14 million. The anticipated increase in SG&A expenses in 2018 is primarily the result of the deployment of Aclaris’s new sales force in January 2018 and the additional selling, marketing and consumer initiatives to support the commercial launch of ESKATA.
Company to Host Conference Call
Management will conduct a conference call at 5:00 P.M. ET today to discuss Aclaris’ financial results and provide a general business update. The conference will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 7386579 prior to the start of the call

On May 8, 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 upcoming presentations during two conferences, both to take place May 16 – 19, 2018; the 21st annual American Society of Gene and Cell Therapy 2018 conference being held in Chicago, IL, and the International Investigative Dermatology conference being held in Orlando, FL (Press release, Abeona Therapeutics, MAY 8, 2018, View Source;p=RssLanding&cat=news&id=2347659 [SID1234526221]).

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American Society of Gene and Cell Therapy
View Source

Oral Presentations:

Phase 1/2 Clinical Trial for Recessive Dystrophic Epidermolysis Bullosa Using EB-101 (COL7A1 Gene-Corrected Autologous Keratinocytes)
Presenter: Jean Yuh Tang, M.D., Ph.D., Stanford University
Thursday, May 17, 2018
Time: 8:20 AM Central Time

Update on Phase 1/2 Gene Transfer Clinical Trial of Systemic Gene Transfer of scAAV9.U1a.hSGSH for MPSIIIA (Sanfilippo Syndrome)
Presenter: Kevin Flanigan, M.D., Nationwide Children’s Hospital
Friday, May 18, 2018
Time: 8:30 AM Central Time

A Phase 1/2 Clinical Trial of Systemic Gene Transfer of rAAV9.CMV.hNAGLU for MPS IIIB: Safety, Tolerability, and Preliminary Evidence of Biopotency
Presenter: Kevin Flanigan, M.D., Nationwide Children’s Hospital
Friday, May 18, 2018
Time: 4:30 PM Central Time

Symposium and Forum:

Assessing Neurodevelopment in Neurodegenerative Disease: The Use and Interpretation of Cognitive Scales
Presenter: Maria Escolar, M.D., Children’s Hospital of Pittsburgh of UPMC
Moderator: Barry Byrne, M.D., Ph.D., University of Florida
Thursday, May 17, 2018
Time: 7:00 AM Central Time

Tools and Technologies Forum
Co-Chair: S. Kaye Spratt, Ph.D., Abeona Therapeutics Inc.
Friday, May 18, 2018
Time: 5:45 PM – 7:45 PM Central Time

Poster Presentations:

Poster 547: Development of a Multi-Domain Responder Index for Clinical Trials with Multi-Domain Diseases
Presenter: Magdalena Tyrpien, Abeona Therapeutics Inc.
Thursday, May 17, 2018
Time: 5:15 PM Central Time

Poster 399: Identification of Novel AAV Capsids for Enhanced CNS Gene Transfer
Presenter: Daphne Chen, University of North Carolina, Chapel Hill
Thursday, May 17, 2018
Time: 5:15 PM Central Time

International Investigative Dermatology

View Source

Poster Presentation:

Poster 328: 50% Wound Healing Correlates with RDEB Patient Reported Outcomes in Pain, Itch, and Skin Durability
Friday, May 18, 2018
Time: 12:00 PM Eastern Time