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 8, 2018 Takeda Pharmaceutical Company Limited (TSE: 4502) ("Takeda") and Shire plc (LON: SHP) ("Shire") reported that they have reached agreement on the terms of a recommended offer pursuant to which Takeda will acquire the entire issued and to be issued ordinary share capital of Shire (Press release, Takeda, MAY 8, 2018, View Source [SID1234526178]). Under the terms of the acquisition, each Shire shareholder will be entitled to receive $30.33 in cash for each Shire share and either 0.839 new Takeda shares or 1.678 Takeda ADSs. The transaction has been approved by both companies’ boards of directors, and is expected to close in the first half of calendar year 2019. Upon the closing of the transaction, Takeda shareholders will own approximately 50 percent of the combined group.

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With leading market positions in prioritized therapeutic areas, an attractive geographic footprint, greater scale and efficiencies, and an even more productive R&D engine, the combined group will be better positioned to deliver highly-innovative medicines and transformative care providing better health and a brighter future for patients around the world.

"Since its inception, Takeda has transformed into an agile, R&D-driven global pharmaceutical company that is well-positioned to deliver innovative and transformative care to patients around the world," said Christophe Weber, president and chief executive officer of Takeda. "Shire’s highly complementary product portfolio and pipeline, as well as experienced employees, will accelerate our transformation for a stronger Takeda. Together, we will be a leader in providing targeted treatments in gastroenterology, neuroscience, oncology, rare diseases and plasma-derived therapies. We are looking forward to the benefits this combination will bring to patients worldwide, the opportunities it will bring for our employees and the returns it will deliver for our shareholders."

Susan Kilsby, chairman of Shire, said, "Over the last 30 years, Shire has become the global leader in treating rare diseases, delivering innovative products that transform patients’ lives. With this combination, Shire helps create an even stronger biopharmaceutical company, with a robust R&D pipeline and expanded global footprint. We are proud of what Shire has become and are grateful to all Shire employees for their contributions. We firmly believe that this combination recognizes the strong growth potential of our leading products and innovative pipeline and is in the best interests of our shareholders, our patients and the communities we serve."

Flemming Ornskov, chief executive officer of Shire, said, "I would like to thank the entire Shire team for all that we have accomplished over the last five years to transform Shire into a leading rare disease biotech company and a tenacious champion for patients in need. I am confident that this relentless focus will enable us to continue delivering against our priorities throughout this process. With a truly innovative portfolio and pipeline, I believe that the combination of the two companies is in the best interests of shareholders and offers an opportunity to improve the lives of even more patients globally with rare and highly specialized conditions."

Highly Compelling Strategic and Financial Rationale

Brings together complementary positions in GI and neuroscience; provides leading positions in rare diseases and plasma-derived therapies to complement strength in oncology and focused efforts in vaccines

The acquisition of Shire will accelerate Takeda’s transformation by bringing together Takeda and Shire’s complementary positions in GI and neuroscience. It will also provide the combined group with leading positions in rare diseases and plasma-derived therapies to complement strength in oncology and focused efforts in vaccines. Takeda will continue to focus on the acceleration of its oncology business, following its recent acquisition of ARIAD Pharmaceuticals. In addition, Takeda’s vaccine business will continue to address the world’s most pressing public health needs.

Creates a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, with an attractive geographic footprint and provides the scale to drive future development

The acquisition will build on Takeda’s long Japanese heritage and values-based culture to create a global biopharmaceutical leader, driven by innovative and world-class R&D. The combined group will have an attractive geographic footprint, with significantly increased exposure in the United States (U.S.), an important and growing market. In addition, Shire’s portfolio will benefit from Takeda’s strong international presence in emerging markets and Japan. The integrated company will continue to be headquartered in Japan, expand its R&D presence in the Boston area and have major regional locations in Japan, Singapore, Switzerland and the U.S. Together, the combined group will have leading positions in two of the largest drug markets globally: the U.S. and Japan. The acquisition is expected to result in Takeda being the only pharmaceutical company listed on both the Tokyo Stock Exchange in Japan, where it will continue to have its primary listing, and the NYSE in the U.S., enabling it to access two of the world’s largest capital markets.

Creates a highly complementary, robust, modality-diverse pipeline and a strengthened R&D engine focused on breakthrough innovation

Takeda and Shire have highly complementary pipelines. Shire has strong expertise in rare diseases, an attractive modality-diverse mid- and late-stage pipeline, enriched with large-molecule programs, as well as cutting-edge technologies in gene therapy and recombinant proteins. Combining this with Takeda’s early development and research-oriented R&D program will result in a highly complementary, robust, modality-diverse pipeline and a strengthened R&D engine focused on breakthrough innovation. The combined group will build on existing partnerships, including Takeda’s more than 180 active partnerships with academia, biotechnology companies and startups, to further enrich the pipeline.

Enhances Takeda’s cash flow profile, with management committed to delivering substantial annual cost synergies and generating attractive returns for shareholders

The acquisition of Shire will provide compelling financial benefits for the combined group. It will be significantly accretive to underlying earnings per share from the first full fiscal year following completion, and will produce strong combined cash flows. The transaction is also expected to result in attractive returns for shareholders, with the return on invested capital (ROIC) expected to exceed Takeda’s cost of capital within the first full fiscal year following completion. The substantial cash flow generation expected to result from the acquisition will enable the combined group to de-lever quickly following completion. Takeda intends to maintain its investment grade credit rating, with a target net debt to EBITDA ratio of 2.0x or less in the medium term.

Takeda is confident that the acquisition will create an opportunity to recognize significant recurring cost synergies, with potential for additional revenue synergies from the combination of Shire and Takeda’s combined infrastructure, market presence and development capabilities. Takeda expects recurring pre-tax cost synergies for the combined group to reach a run-rate of at least $1.4 billion per annum by the end of the third fiscal year following completion of the acquisition.

The acquisition will accelerate Takeda’s strategic transformation toward Vision 2025, and strong combined cash flows will enable continued investment in R&D. Takeda’s well-established dividend policy will remain a key component of future shareholder returns.

Execution

Takeda’s experienced management team has a proven track record of executing complex business integrations and large-scale transformations, and is well-positioned to successfully integrate Shire and maximize the value of the combination. The integration will be supported by the companies’ highly complementary organizational structures in geographic areas, including hubs in the Boston area, Switzerland and Singapore, as well as similar therapeutic area focus and complementary approaches to R&D. Takeda is dedicated to carrying out integration efforts in a manner consistent with the company’s core values of integrity, fairness, honesty and perseverance, building on the expertise of employees of both companies.

Transaction Terms

Under the terms of the acquisition, Shire shareholders will be entitled to receive, for each Shire share, $30.33 in cash and either 0.839 new Takeda shares or 1.678 Takeda ADSs.

The acquisition terms imply an equivalent value of:

£48.17 per Shire share based on the closing price of ¥4,535 per Takeda share on May 2, 2018, and the exchange rates of £:¥ of 1:147.61 and £:$ of 1:1.3546 on May 4, 2018 (being the latest practicable date prior to this announcement); and
£49.01 per Shire share based on the closing price of ¥4,923 per Takeda Share and the exchange rates of £:¥ of 1:151.51 and £:$ of 1:1.3945 on April 23, 2018 (being the day prior to the announcement that the Shire board would, in principle, be willing to recommend the consideration).

The equivalent value of £49.01 per Shire share values the entire issued and to be issued ordinary share capital of Shire at approximately £46 billion.

Immediately following completion of the transaction, Takeda shareholders will hold approximately 50 percent of the combined group.

The transaction has been approved by the boards of both companies, and is subject to the approval of Shire and Takeda shareholders and certain customary closing conditions, including regulatory approvals.

The acquisition is expected to close in the first half of calendar year 2019. Upon completion, the new Takeda shares will be listed on the Tokyo Stock Exchange, and local Japanese stock exchanges. In addition, Takeda will apply for its ADSs (each representing 0.5 Takeda shares) to be listed on the NYSE effective on or shortly after the effective date.

Financing

Takeda has entered into a bridge facility agreement of $30.85 billion with, among others, J.P. Morgan Chase Bank N.A., Sumitomo Mitsui Banking Corporation and MUFG Bank, Ltd., part of the proceeds of which will be used to fund the cash consideration payable to Shire shareholders in connection with the acquisition. It is currently contemplated that, prior to completion, the commitments under the bridge facility agreement will be reduced or refinanced with a combination of long-term debt, hybrid capital and available cash resources.

Conference Call Webcast Information

Takeda will host a transaction conference call at 4.15pm – 5pm JST / 8.15am – 9am BST / 3.15am – 4am EST on May 8, 2018 to discuss the transaction.

Investors and analysts can dial in to the conference call using the numbers below:

Standard International Access: +44 (0) 20 3003 2666; Japan Toll Free: 006633132499; UK Toll Free: 0808 109 0700; USA Toll Free: 1 866 966 5335; Tokyo Toll Free: +81 (0) 3 5050 5366; and Passcode: 161017

A presentation for the call will be available at:

View Source

Takeda will host an additional audio webcast at 10.00 p.m. JST / 2.00 p.m. BST / 9.00 a.m. ET on May 8, 2018 with Japanese translation, to discuss the transaction. The webcast can be accessed at the following link:

View Source

Replays of the conference calls will be available within 24 hours.

For more information, the full Rule 2.7 announcement setting out full details of the offer to Shire shareholders is available at: View Source

On May 8, 2018 Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, reported that it will participate in a fireside chat at the Bank of America Merrill Lynch 2018 Health Care Conference on Tuesday, May 15, 2018, at 8 a.m. PT (11:00 a.m. ET) in Las Vegas, Nevada (Press release, Editas Medicine, MAY 8, 2018, View Source;p=RssLanding&cat=news&id=2347637 [SID1234526203]).

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A live webcast of the presentation will be available on the Investors & Media section of the Editas Medicine website at www.editasmedicine.com. An archived replay will be available for approximately 30 days following the presentation.