On May 9, 2018 XOMA Corporation (Nasdaq: XOMA), a pioneer in the discovery, development and licensing of therapeutic antibodies, reported its first quarter 2018 financial results (Press release, Xoma, MAY 9, 2018, View Source [SID1234526401]).

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"Our efforts in the first quarter were dedicated to identifying, assessing and analyzing out-license and asset acquisition opportunities. These included potential partnering conversations regarding our novel IL-2 antibody program, as well as discussions with companies seeking to monetize future potential milestone and royalty revenue streams," stated Jim Neal, Chief Executive Officer at XOMA. "While the number of opportunities continues to expand, we are focused on identifying a narrower set of high quality potential transactions. With a cash runway that spans multiple years and additional access to capital from our credit facility, we remain intensely focused on allowing our fully-funded programs to mature in the hands of our partners while expanding and diversifying our portfolio of potential future revenue streams to drive both near- and long-term value."

Financial Results

XOMA recorded total revenues of $0.5 million for the first quarter of 2018, compared to $0.3 million for the first quarter of 2017.

Research and development (R&D) expenses were $0.4 million for the first quarter of 2018, compared to $4.0 million for the first quarter of 2017. The decrease in R&D expenses was due primarily to reductions of $1.0 million in clinical trial costs, $0.8 million in consulting costs, $0.6 million in the allocation of facilities costs, $0.3 million in salaries and related expenses, $0.3 million in stock-based compensation, and $0.3 million in external manufacturing costs. The significant reduction in R&D spending year-over-year is a result of the execution of the Company’s royalty-aggregator business model that is designed to leverage its extensive portfolio of partnered programs and licensed technologies.

General and administrative (G&A) expenses were $5.2 million for the three months ended March 31, 2018 and 2017, respectively. The minimal change in G&A expenses for the three months ended March 31, 2018 was due primarily to decreases of $0.8 million in consulting services, $0.3 million in legal and audit fees, and $0.2 million in information technology costs, partially offset by increases of $0.7 million in stock compensation cost and $0.6 million in the allocation of facilities costs due to a greater proportion of general and administrative personnel after the Company’s restructuring activities.

Net loss for the first quarter of 2018 was $3.8 million. Net loss for the first quarter of 2017 was $16.3 million and included non-recurring and restructuring charges totaling $7.6 million.

On March 31, 2018, XOMA had cash and cash equivalents of $42.0 million. The Company ended December 31, 2017, with cash and cash equivalents of $43.5 million. The Company’s current cash and cash equivalents are expected to be sufficient to fund its operations for multiple years.

In May 2018, the Company announced a flexible $20 million credit facility with Silicon Valley Bank. The credit facility is available to XOMA through March 2019 and may be extended to March 2020 upon certain conditions. The credit facility includes the opportunity to increase the borrowing capacity to an aggregate amount of $40 million.

On MaY 9, 2018 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today reported its financial results for the first quarter ended March 31, 2018 and provided an update on the company’s recent achievements and anticipated upcoming milestones.

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"During the first quarter of 2018 we continued to make excellent progress across our product portfolio," stated Matthew R. Patterson, Chief Executive Officer of Audentes. "In particular, we are excited by the positive preliminary results we have observed in our XLMTM program and we look forward to reporting additional interim data from the first dose cohort of ASPIRO patients at the upcoming ASGCT (Free ASGCT Whitepaper) annual meeting. We are also pleased by the preliminary data from the first patient treated in our Crigler-Najjar program, which demonstrated an encouraging early safety profile and initial proof-of-concept for efficacy. We look forward to treating our next patient at a higher dose in the coming weeks."

Mr. Patterson continued, "Finally, we are excited by the growing momentum behind our product candidate AT982, which we believe offers a best-in-class approach for the treatment of Pompe disease. Based on recent discussions with regulatory authorities, we have expanded our clinical vision and plan to conduct two separate Phase 1 / 2 clinical studies for the infantile and late onset Pompe disease patient populations in parallel. As such, we have expanded the scope of our IND-enabling preclinical program, and now plan to include both studies in the initial IND filing targeted for the fourth quarter of 2018, which will support the initiation of the studies in the first half of 2019."

Recent Achievements & Upcoming Key Events

AT132 for XLMTM:
Completed dosing of three additional patients (cohort 1 expansion) in the 1×1014 vg/kg cohort of ASPIRO, bringing the total number of patients enrolled to date to seven (six AT132-treated and one delayed-treatment control). No significant treatment-related safety signals have been identified to date in cohort 1 expansion patients.
Plan to report additional interim data during an oral presentation at ASGCT (Free ASGCT Whitepaper) on May 16, 2018. Presentation to include up to 24-week data for the first four subjects enrolled and up to 4-week data in the cohort 1 expansion patients.
Plan to report six-month biopsy data from the first three patients dosed and announce plans with regard to dose escalation in the third quarter of 2018
AT342 for Crigler-Najjar Syndrome:
Initial proof of concept established based on 12-week data from first patient enrolled in VALENS study, a 12-year-old male, at a dose of 1.5×1012 vg/kg
AT342 has been well-tolerated with no significant treatment-related safety signals to date
Treatment resulted in a rapid decline in total bilirubin levels from approximately 11 mg/dL at baseline to 4 mg/dL at week 2 post-dosing, with a gradual return to baseline by week 12. A similar efficacy result was observed with low doses of AT342 in a dose ranging study in the mouse model of Crigler Najjar, while higher doses demonstrated durable bilirubin reduction.
Plan to dose escalate to the 6 x1012 vg/kg and enroll the next patient in VALENS in the coming weeks
Plan to report interim 12-week data in the first patient dosed in VALENS at the 51st Annual Congress of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) on May 10, 2018 and at ASGCT (Free ASGCT Whitepaper) on May 17, 2018
Plan to report the next interim data update from VALENS in the second half of 2018
AT982 for Pompe Disease:
Announced selection of the clinical development candidate, AT982, a novel AAV8 vector designed to express GAA in tissues relevant to Pompe disease, including skeletal muscle, the heart and the nervous system, and in the liver to reduce immunogenicity, thereby addressing the key limitations of existing enzyme replacement therapy for Pompe disease
Plan to present additional data from AT982 in a Pompe mouse model at ASGCT (Free ASGCT Whitepaper) on May 16, 2018
Plan to expand the preclinical program to support the filing of an IND to study AT982 in both infantile and late onset Pompe disease patients
Plan to file the IND in the fourth quarter of 2018 and initiate both Phase 1 / 2 clinical studies in the first half of 2019
AT307 for CASQ2-CPVT:
IND has been submitted. FDA has completed its initial review and provided a short list of questions to be addressed prior to the IND becoming active. We expect to submit responses in the coming weeks.
Received Fast Track designation from the FDA
Continuing patient identification activities to better characterize CASQ2-CPVT prevalence. Results from these efforts will inform clinical plans as they relate to the timing of a potential Phase 1 / 2 study.
Key Corporate Milestones:
In January 2018, Audentes strengthened its balance sheet with the completion of a follow-on financing, issuing 6,612,500 shares of common stock at an offering price of $35.00 per share, resulting in net proceeds of approximately $217.2 million after the deduction of underwriting discounts, commissions and offering expenses
Announced the promotion of Natalie Holles to President and Chief Operating Officer. In this new role, Ms. Holles will oversee the day-to-day operations of the company, including research, development, manufacturing, program management and corporate development.
First Quarter 2018 Financial Results

Cash Position: At March 31, 2018, Audentes had cash, cash equivalents, and short-term investments of $326.1 million. Current cash, cash equivalents and short-term investments are planned to fund operations into the second half of 2020.
Research and Development Expenses: Research and development expenses were $19.9 million for the first quarter of 2018 compared to $14.6 million for the same period in 2017, an increase of $5.3 million. The increase in research and development expenses was primarily attributable to an increase in development costs related to our AT982 program, increased headcount and related facility costs, increased internal manufacturing costs and higher stock compensation expense, and is partially offset by a decrease in the estimated fair value of the contingent liability associated with the 2015 acquisition of Cardiogen Sciences, resulting in a $2.3 million reduction in research and development expense during the first quarter of 2018. Research and Development expenses included $2.1 million of non-cash stock-based compensation expense.
General and Administrative Expenses: General and administrative expenses were $6.5 million for the first quarter of 2018 compared to $3.6 million for the same period in 2017, an increase of $2.9 million. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs, increased professional service fees, higher stock compensation expense and higher costs driven by continued public company regulatory compliance initiatives. General and administrative expense includes $1.3 million of non-cash stock-based compensation expense.
Net Loss: Net loss was $25.6 million for the first quarter of 2018 compared to $18.1 million for the same period in 2017.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its first quarter 2018 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call 1-833-659-8620 (U.S.) or 1-409-767-9247 (international) and dial the conference ID 9789828 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.

On May 9, 2018 Ophthotech Corporation (Nasdaq:OPHT) reported financial and operating results for the first quarter ended March 31, 2018 and provided a business update (Press release, Ophthotech, MAY 9, 2018, View Source [SID1234526316]).

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"We continue to build on our Zimura program with on-going clinical trials in wet age related macular degeneration (AMD) with patient recruitment completed and topline data expected by the end of 2018, in geographic atrophy secondary to dry AMD where recruitment is on-track for topline data in the second half of 2019 and in autosomal recessive Stargardt disease which began enrolling patients earlier in the year," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "In parallel, we are excited to have initiated our first innovative gene therapy program in collaboration with Horae Gene Therapy Center at the University of Massachusetts Medical School to treat orphan degenerative retinal diseases. A key aspect of our strategy is to continue to build on this momentum to uncover novel and differentiating technologies and product candidates through collaborations with leading companies and academic institutions from around the world."

Recent Key Highlights

Zimura Complement Factor C5 Inhibitor Program

In April 2018, the Company completed patient recruitment in its dose-ranging, open-label, multi-center Phase 2a clinical trial of Zimura (avacincaptad pegol) in combination with the anti-vascular endothelial growth factor (anti-VEGF) agent Lucentis (ranibizumab) in patients with wet age-related macular degeneration (AMD) who have not been previously treated with any anti-VEGF agents. This uncontrolled trial is designed to assess safety at different dosages and to detect a potential efficacy signal. The Company will evaluate data at month six.
The Company’s ongoing Zimura clinical trial for the treatment of geographic atrophy secondary to dry AMD is on track for initial top-line data to be available during the second half of 2019.
In January 2018, the first patient was enrolled in the Company’s Phase 2b randomized, double-masked, sham-controlled clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1). Initial top-line data is expected to be available in 2020.
Scientific details for two of the Company’s ongoing Zimura clinical trials were presented at medical conferences:
The scientific details of the geographic atrophy secondary to dry AMD clinical trial were presented at the 41st Annual Macula Society Meeting in Beverly Hills, California, February 21-24, 2018.
The scientific details of the STGD1 clinical trial were presented at the 2018 Annual Meeting of the Association for Research in Vision and Ophthalmology in Honolulu, Hawaii, April 29-May 3, 2018 and at the International Symposium on Ocular Pharmacology and Therapeutics in Tel-Aviv, Israel, March 1-3, 2018.
Gene Therapy Program

In February 2018, the Company initiated an innovative gene therapy program focused on applying novel gene therapy technology to discover and develop new therapies for ocular diseases.
In February 2018, the Company announced its first gene therapy collaboration, as it entered into a series of sponsored research agreements with the University of Massachusetts Medical School (UMMS) and its Horae Gene Therapy Center to utilize their "minigene" therapy approach and other novel gene delivery methods to target retinal diseases. UMMS has granted Ophthotech an option to obtain an exclusive license to any patent or patent applications that result from this research.
Corporate Highlight

In January 2018, the Company announced the election of Jane PritchettHenderson, Chief Financial Officer and Senior Vice President of Corporate Development at Voyager Therapeutics, to its Board of Directors. Ms. Henderson has also been elected the Chair of the Ophthotech Audit Committee.

2018 Operational Update

As of March 31, 2018, the Company had $155 million in cash and cash equivalents.

The Company’s estimates its year end 2018 cash and cash equivalents will range between $112 million and $117 million based on its current 2018 business plan and planned capital expenditures. This estimate includes continuation of the Company’s development programs for Zimura and the initiation of its collaborative gene therapy research programs as currently planned. This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.

First Quarter 2018 Financial Highlights

Revenues: Collaboration revenue was $0 for the quarter ended March 31, 2018, compared to $1.7 million for the same period in 2017. Collaboration revenue decreased due to the completion of the Company’s licensing and commercialization agreement with Novartis Pharma AG and the recognition of all associated deferred revenue during the third quarter of 2017.
R&D Expenses: Research and development expenses were $7.7 million for the quarter ended March 31, 2018, compared to $32 million for the same period in 2017. As the Company pursues its ongoing and planned Zimura development programs, research and development expenses decreased primarily due to decreases in expenses related to the discontinuation of the Company’s FovistaPhase 3 clinical program and decreases in costs associated with the Company’s 2017 reduction in personnel program.
G&A Expenses: General and administrative expenses were $5.6 million for the quarter ended March 31, 2018, compared to $13.2 million for the same period in 2017. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure and decreases in costs associated with its 2017 reduction in personnel program, which included facilities lease termination expenses incurred during the first quarter of 2017.
Net Loss: The Company reported a net loss for the quarter ended March 31, 2018 of $13.1 million, or ($.36) per diluted share, compared to a net loss of $43.1 million, or ($1.20) per diluted share, for the same period in 2017.
Conference Call/Web Cast Information

Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for May 9, 2018 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-239-9838 (USA) or 323-794-2551 (International), passcode 2075643. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 2075643.

On May 9, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on myelodysplastic syndromes, reported that the Company will release its first quarter 2018 financial results on May 15, 2018 before the market opens (Press release, Onconova, MAY 9, 2018, View Source [SID1234526382]). The Company will host a conference call on May 15, 2018 at 9:00 a.m. Eastern Time to discuss these results.

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Interested parties may access the call by dialing toll-free (855) 428-5741 from the US or (210) 229-8823 internationally, and using conference ID 2573768.

The call will also be webcast live. Please click here to access the webcast.

A replay will be available at that link until August 31, 2018.

On May 9, 2018 BioClin Therapeutics, Inc., a clinical stage drug development company developing a first-in-class anti-FGFR3 (fibroblast growth factor receptor 3) monoclonal antibody for metastatic bladder cancer, reported
expansion of its Series B financing to $50 million to broaden its clinical development activities (Press release, BioClin Therapeutics, MAY 9, 2018, View Source [SID1234526402]). New investors, Sectoral Asset Management and INKEF Capital, joined existing investors Sofinnova Ventures, Ysios Capital, HealthCap, Tekla Capital Management funds, and Life Sciences Partners (LSP) in the round. Since its founding, the company has raised a total of $79 million.

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The proceeds from this financing will be used to advance the company’s lead development candidate, B-701, for the treatment of patients with metastatic bladder cancer, or metastatic urothelial carcinoma (mUC). "This financing supports our Phase 2 trials evaluating B-701 both as a single agent, as well as in combination with pembrolizumab and separately with docetaxel. We anticipate completing enrollment in these Phase 2 studies by the second half of 2018, with initial data expected by yearend," said Stephen Lau, CEO of BioClin Therapeutics.

"We are delighted that Sectoral Asset Management and INKEF Capital have joined the BioClin investor syndicate. The additional funding will allow the Company to move forward with the only targeted biologic specific for FGFR3 in clinical development for bladder cancer," said David Kabakoff of Sofinnova and board member of BioClin Therapeutics.