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.

On May 9, 2018 Chia Tai Tianqing Pharmaceutical Group reported the 1.1-type new drug Anlotinib Hydrochloride Capsules (Focavi) obtained the registration approval document approved by the State Drug Administration (Press release, Jiangsu Chia-tai Tianqing, MAY 9, 2018, View Source [SID1234576212]). This marks the official launch of Anlotinib, an original and innovative drug in the Chinese oncology field that has received much attention. Wang Shanchun, president of CP Tianqing Pharmaceutical Group, said: "We expect that this new national drug will benefit more Chinese patients and even patients around the world after it is launched."

Anlotinib refers to advanced non-small cell lung cancer

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After more than 10 years of hard work, the R&D team of CP Tianqing finally made a breakthrough in the development of oncology drugs. The 1.1-type new drug Anlotinib Hydrochloride Capsules was approved for marketing. This product is a new type of small molecule multi-target tyrosine kinase inhibitor, which can effectively inhibit VEGFR, PDGFR, FGFR, c-Kit and other kinases, and has the dual effects of anti-tumor angiogenesis and inhibiting tumor growth. Clinical trials have confirmed that Focavi is the only effective single-drug oral preparation among the anti-angiogenesis targeted drugs for advanced non-small cell lung cancer, and its adverse reactions are relatively mild, and it is well tolerated by patients. According to analysis by industry experts, Anlotinib is expected to become the standard drug for the third-line treatment of patients with advanced non-small cell lung cancer.

"The listing of Anlotinib is also due to the ongoing reform measures for drug review and approval by the State Food and Drug Administration. As clinical studies have shown that Anlotinib has obvious clinical advantages over existing treatments, after the application That is, it was included in the priority review sequence by the drug review center. It is precisely because of the attention of the drug review department and the review experts working overtime that Anlotinib completed the marketing review and was approved in a short period of time. This will enable the majority of patients to use safe and effective anti-tumor innovative drugs as soon as possible." Wang Shanchun believes that the drug review period coincides with the national drug review and approval policy reform period, and the country’s orientation to encourage R&D and innovation is becoming more and more obvious. The approval of innovative drugs with outstanding clinical value such as Anlotinib has been guaranteed and encouraged. Through the priority review procedure, the Center for Drug Evaluation has provided an effective guarantee for meeting clinical drug needs, reducing drug costs, and promoting public health.

The clinical research results show that Focavi not only has a good therapeutic effect on non-small cell lung cancer, but also on soft tissue sarcoma, ovarian cancer and other cancers. CT Tianqing is actively carrying out multi-center clinical research including the United States. . The results of the Phase 2b clinical study of Anlotinib in the treatment of soft tissue sarcoma were reported orally by ASCO (Free ASCO Whitepaper) this year, and the results of the pathological subgroup of the Phase 3 clinical study for the treatment of non-small cell lung cancer were displayed in the ASCO (Free ASCO Whitepaper) this year, and were written into the "2018 CSCO Lung Cancer Guidelines" . Wang Shanchun said: "It is not easy for China’s original innovative drugs to have won such an honor before they went on the market. This shows the international recognition of Chinese innovative drugs."

Malignant tumors are the unbearable weight of life

There are more than 4 million new cancer patients in China each year, and an average of more than 10,000 people are diagnosed as new patients every day, and the incidence rate is increasing year by year. In particular, lung cancer ranks first in morbidity and mortality among men, and second in morbidity and mortality among women. The five-year survival period of cancer patients is much lower than the average in developed countries.

As an ordinary citizen, once diagnosed with a malignant tumor disease, not only the patient himself will suffer both physical and mental torture, loss of hope of survival, loss of dignity of life, but also a family may be crushed by this, and bear a heavy burden from then on The unbearable spiritual and economic burden. As an enterprise with a social conscience, CP Tianqing is also working hard, hoping to one day conquer the persistent disease of malignant tumors through technological innovation.

In recent years, with the development of traditional chemotherapy, targeted therapy and immunotherapy have successively entered first-line and second-line treatment, and the treatment of advanced non-small cell lung cancer has been greatly improved. However, for Chinese patients who have failed first-line and second-line treatments, the existing third-line treatments are relatively lacking and the choices are confusing, and patients are often in the predicament of no medicines available. In this case, the new small molecule multi-target tyrosine kinase inhibitor anlotinib independently developed by CT Tianqing has finally succeeded, providing an effective new method for the third-line treatment of patients with advanced non-small cell lung cancer in China. treatment method.

CP Tianqing has a rich product line in the anti-tumor field

The listing of Anlotinib can be said to be a milestone event in the development history of CP Tianqing. The drug is Chia Tai Tianqing’s first innovative small molecule drug in accordance with international R&D procedures and standards, and it is also the company’s most invested anti-cancer drug so far. The successful listing of Anlotinib marks a solid step forward by CP Tianqing from "combination of imitation and innovation" to "combination of innovation and imitation". It is also a major breakthrough in the oncology field of the company based on the "focus on liver disease" strategy. .

Chia Tai Tianqing Pharmaceutical Group is an innovative pharmaceutical group enterprise integrating scientific research, production and sales. It is a well-known liver health drug research and development and production base in China. It is a national key high-tech enterprise and a national Torch Plan Lianyungang new pharmaceutical industry base. The backbone enterprise ranks 16th among the top 100 enterprises in China’s pharmaceutical industry. Chia Tai Tianqing currently invests more than 10% of its sales revenue every year. It has more than 1,000 R&D personnel and more than 180 products under research, including more than 40 innovative drugs and more than 20 biological drugs.

It is reported that in addition to the field of strong liver diseases, CP Tianqing has also formed a unique product line in the field of anti-tumor. Hematological tumor products Decitabine, Imatinib, and Dasatinib are the first imitation products in China. In the next three years, CP Tianqing will launch more products in the oncology field, such as bortezomib, bendamustine, lenalidomide, azacitidine, etc., to improve drug accessibility and quality of life for cancer patients.