Ultragenyx Secures $75 Million for Ultra-Rare Diseases

Ultragenyx Pharmaceutical Inc. recently announced the successful completion of a $75-million Series B round financing led by Adage Capital Partners, LP. Joining Adage as new investors in this financing are mutual funds and separate account clients advised by T. Rowe Price Associates, Inc., Jennison Associates LLC (on behalf of clients), funds and accounts under management by subsidiaries of BlackRock, Inc., Sanofi-Genzyme BioVentures1, Shire plc and additional blue chip public market funds. Existing investors, TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures also participated in the transaction.

Ultragenyx plans to use the proceeds from the financing primarily to advance development of the company’s lead clinical-stage programs, UX001 and UX003, and other undisclosed programs. UX001 is a potential substrate replacement therapy for hereditary inclusion body myopathy currently being investigated in a fully enrolled, randomized placebo-controlled Phase II clinical study with results anticipated in 2013. UX003 is a recombinant enzyme replacement therapy intended for the treatment of mucopolysaccharidosis type 7 (MPS 7), which will enter a Phase I/II clinical study in MPS 7 patients in 2013. The company intends to continue to expand its pipeline through the potential in-licensing of additional products.

“We welcome the participation of such highly regarded public market and strategic investors in this pivotal financing,” said Eran Nadav, PhD, Managing Director at TPG Biotech and Chairman of the Board of Ultragenyx. “Ultragenyx, led by a distinguished team experienced in rare disease drug development, is building a world-class orphan drug company with a rich pipeline of transformative therapeutics.”

“We deeply appreciate the support of all our investors, new and existing, and their confidence in our ability to find and efficiently develop compelling new treatments for devastating rare genetic disorders,” added Emil D. Kakkis, MD, PhD, Chief Executive Officer and Founder of Ultragenyx. “This financing transaction is critical to expanding our efforts to deliver profound novel therapies that benefit even more rare disease patients.”

Ultragenyx is a privately held, development-stage biotechnology company committed to bringing to market life-transforming therapeutics for patients with rare and ultra-rare metabolic genetic diseases. The company focuses on diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no effective treatments. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’ strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information, visit www.ultragenyx.com.

Independent AbbVie Officially Launches

AbbVie recently marked its launch as an independent biopharmaceutical company, with employees from more than 40 countries and patients joining AbbVie leaders as they rang the first opening bell of 2013 at the New York Stock Exchange.

With a rich, 125-year heritage of developing pharmaceuticals, AbbVie combines the focus and passion of a leading-edge biotech with the expertise and structure of a long-established pharmaceutical leader. A global enterprise that serves patients in more than 170 countries, AbbVie launches with an estimated $18 billion in annual revenues and a strong commitment to creating shareholder value through long-term growth potential and the continuation of the company’s history of shareholder dividends.

“Today, AbbVie launches with an outstanding portfolio, a solid pipeline, and enthusiastic people who will serve patients and deliver growth,” said Richard A. Gonzalez, Chairman of the Board and Chief Executive Officer, AbbVie. “With those assets and a relentless focus on innovation, we intend to create significant value for our shareholders.”

AbbVie launches with a team of experienced senior leaders who understand how to manage AbbVie for long-term, profitable growth and who have the proven ability to commercialize scientific discoveries for patients. As a biopharmaceutical company, AbbVie has adopted a streamlined and focused business model built on a broad portfolio of market leading medicines that will enable AbbVie to invest in tomorrow’s therapies. The portfolio includes both growth brands and durable performers, including HUMIRA, AndroGel, Lupron, Synagis, Creon, Synthroid, Kaletra, Norvir, and Zemplar.

AbbVie’s long-term growth will be fueled by a compelling pipeline of more than 20 mid- to late-stage clinical programs as well as new discoveries to address diseases, including hepatitis C, rheumatoid arthritis, psoriasis, multiple sclerosis, Alzheimer’s disease, Parkinson’s disease, spondyloarthropathies, multiple myeloma, and endometriosis. AbbVie has tripled the number of new molecular entities in its pipeline over the last several years.With these and other compounds in its pipeline, AbbVie is rapidly developing therapies that have strong clinical performance, patient benefit, and health economic value.

Making new discoveries and developing them into effective medicines is the foundation of the company’s mission and business. To accomplish those goals, AbbVie’s research is guided by a patient-driven research and development (R&D) approach that begins with a deep understanding of the serious diseases that are the company’s focus, as well as careful evaluation of the current needs of patients, payers, and regulators worldwide. The company’s scientists and physicians focus their work on targets that have the best potential to fundamentally transform the way diseases are treated in the future.

AbbVie is committed to an agile and collaborative approach to innovation, across its global R&D and manufacturing sites. The company’s scientists rely on proprietary technologies and methods to help them more quickly advance the most promising compounds from laboratory to clinical trials. AbbVie’s research operates with a collaborative model that seeks to build the pipeline of discoveries from inside and outside the company’s own walls.

AbbVie is focused on developing specialty pharmaceuticals that address complex, unsolved health problems, particularly chronic diseases, that now account for 75% of all healthcare costs. By developing new specialty treatments that improve patient outcomes, AbbVie and its people are helping to reduce the long-term health and economic impact of these conditions.

AbbVie’s 21,000 employees around the world are dedicated to meeting the needs of patients. They have the freedom, expertise, and ability to innovate and discover solutions to the world’s most pressing health needs.

“Our commitment at AbbVie is to build on Abbott’s 125-year heritage and address some of the world’s most serious health issues,” added Mr.Gonzalez. “We intend AbbVie’s enduring legacy to be one of finding treatments for patients with serious healthcare needs.”

AbbVie is a global, research-based biopharmaceutical company formed in 2013 following separation from Abbott. AbbVie combines the focus and passion of a leading-edge biotech with the expertise and structure of a long-established pharmaceutical leader. The company’s mission is to use its expertise, dedicated people, and unique approach to innovation to develop and market advanced therapies that address some of the world’s most complex and serious diseases. For more information, visit www.abbvie.com

Perrigo Acquires Remaining Stake in Drug Development Company for Nearly $45 Million

Perrigo Company recently announced it has signed a definitive merger agreement and has completed the acquisition of Cobrek Pharmaceuticals, Inc. (Cobrek), a privately held, Chicago, IL-based drug development company, for approximately $45 million on a cash free and debt free basis.

In May 2008, Perrigo acquired an 18.5% minority stake in Cobrek in conjunction with entering into a product development collaborative partnership agreement with the company focused on foam dosage form generic pharmaceutical products. To date, the partnership has successfully yielded two commercialized foam-based products and an additional two FDA-approved foam-based products awaiting date certain launches in Perrigo’s fiscal year 2013. Cobrek primarily derives its earnings stream from profit shares on products partnered with Perrigo, who is responsible for all commercial activities under the existing collaboration agreement. As part of the transaction, Cobrek will forgo any profit share payments earned during Perrigo’s fiscal second quarter 2013.

“We continue to market the only generic foam products in the US and expect to launch two additional products generic versions of Luxiq (betamethasone valerate) Foam and Olux-E (clobetasol propionate) Foam 0.05% during this fiscal year, which is a testament to the challenges of this dosage form and success of our partnership with Cobrek,” said Perrigo Chairman, President and CEO Joseph C. Papa. “The partnership also boasts an additional ANDA on file for a promising topical generic product. This acquisition allows us to capture the full economics going forward on our portfolio of prescription foams as well as maintain control over future development activities in this important category of extended topical products. Acquiring the remaining stake of Cobrek not already owned is the logical next step in this relationship and will allow us to continue to bring quality, affordable healthcare to our consumers and customers.”

From its beginnings as a packager of generic home remedies in 1887, Allegan, Michigan-based Perrigo Company has grown to become a leading global provider of quality, affordable healthcare products. Perrigo develops, manufactures and distributes OTC and generic prescription (Rx) pharmaceuticals, infant formulas, nutritional products, dietary supplements, and APIs. For more information, visit www.perrigo.com.

Infinity Pharmaceuticals Pays Millennium $15 Million

Infinity Pharmaceuticals, Inc. recently announced that the company amended its development and license agreement with Millennium: The Takeda Oncology Company and Takeda Pharmaceutical Company Limited for Infinity’s phosphoinositide-3-kinase (PI3K) program. Under the amended agreement, Infinity continues to retain full worldwide rights to IPI-145, a potent, oral inhibitor of PI3K-delta and PI3K-gamma, as well as worldwide rights to any future product candidates targeting PI3K-delta and/or PI3K-gamma, and Millennium remains entitled to receive success-based milestones and tiered royalties on future worldwide sales of products covered by this agreement.

In exchange for Infinity’s one-time payment of $15 million, Millennium waived its right to opt into a 50/50 US profit and loss-sharing arrangement in the US for any of Infinity’s PI3K inhibitors, including IPI-145. In addition, Infinity paid Millennium the $5-million development milestone for the August 2012 initiation of the Phase IIa clinical trial of IPI-145 in patients with asthma.

“We believe that the potency of IPI-145 against both PI3K-delta and PI3K-gamma contribute to its potential to become the best-in-class PI3K inhibitor,” added Lawrence Bloch, MD, JD, Executive Vice President, Chief Financial Officer and Chief Business Officer of Infinity. “Infinity is focused on rapidly advancing IPI-145 and additional follow-on PI3K product candidates with the goal that patients, our shareholders, and other financial stakeholders, including Millennium, will benefit from their development and commercialization.”

“We have had a successful relationship with Infinity since the December 2011 acquisition of Intellikine by Takeda Pharmaceutical Company Limited,” stated Kyle Kuvalanka, Vice President, Business Development and Corporate Strategy and Development of Millennium. “We look forward to Infinity’s continued progress and to participating in the downstream value of its PI3K program through future milestone and royalty payments.”

Under the terms of the amended agreement, Infinity continues to retain worldwide development and commercialization rights for its PI3K-delta,gamma program. In exchange for Infinity’s one-time payment of $15 million, Millennium waived its right to opt into a 50/50 US profit and loss-sharing arrangement and its right to participate in up to 30% of the detailing effort in the US for any of Infinity’s PI3K inhibitors. In addition, Infinity paid Millennium the $5-million milestone for the August 2012 initiation of the Phase IIa trial of IPI-145 in patients with asthma.

For Infinity’s first two distinct PI3K product candidates, Millennium is entitled to receive up to $15 million in remaining success-based development milestones and up to $450 million in success-based approval and commercial milestones, as defined in the original agreement. For all of Infinity’s PI3K-delta and/or PI3K-gamma inhibitors covered under the agreement, Infinity is obligated to pay Millennium tiered royalties on worldwide net sales ranging from 7% to 11%, which are the same royalty levels as those specified under the original agreement.

The PI3Ks are a family of enzymes involved in multiple cellular functions, including cell proliferation and survival, cell differentiation, cell migration, and immunity. The PI3K-delta and PI3K-gamma isoforms are preferentially expressed in leukocytes, where they have distinct and non-overlapping roles in immune cell development and function. Targeting PI3K-delta and PI3K-gamma may provide multiple opportunities to develop differentiated therapies for the treatment of inflammatory diseases as well as hematologic malignancies.

Infinity is developing a portfolio of novel PI3K-delta and/or PI3K-gamma inhibitors in both inflammatory diseases and oncology. IPI-145 is currently progressing in a Phase I trial in patients with advanced hematologic malignancies and in a Phase IIa trial in patients with mild, allergic asthma. Infinity is also planning to begin a Phase II trial of IPI-145 in patients with rheumatoid arthritis in the first half of 2013. Infinity expects to name its first follow-on PI3K development candidate by the end of this year and is planning to complete nonclinical studies, which are designed to enable Phase I development, by the end of 2013. For more information, visit www.infi.com.

goBalto Secures Additional Funding for Web-Based Clinical Study Platform

goBalto, Inc. recently announced it closed a $12 million round of venture financing, led by EDBI, the global investment arm of Singapore‘s Economic Development Board, with participation from new investor Qualcomm Life Fund, a $100-million investment allocation that is managed by Qualcomm Ventures, Qualcomm’s investment group. This round includes existing investors Aberdare Ventures, West Health Investment Fund, and Founding CEO Jae Chung. The new round of funding brings goBalto’s total financing to $21 million. goBalto plans to use these funds to enhance its flagship platform for clinical study start–up with new product capabilities that will help support the demands of the growing clinical research market.

“The clinical research industry still has to make do with decade-old legacy solutions. With this financing, we will be able to support the growing global demand of enterprise customers seeking user friendly, cloud-based collaboration technologies that accelerate the delivery of medicines to those in need,” said Jae Chung, CEO of goBalto.

“goBalto’s next-generation solution for clinical trials workflow will strengthen Singapore‘s position as the Asia Pacific clinical trials hub by enhancing the efficiency of top tier sponsors and CROs based here,” added Ms. Swee-Yeok Chu, CEO & President of EDBI. “We look forward to working closely with goBalto to extend its global footprint through Singapore, leveraging on EDBI’s extensive international networks and experience in Asia.”

“The cost of clinical development has been on the rise at an alarming rate in recent years,” said Jack Young, Director of Qualcomm Ventures. “Adopting digital and mobile technologies could potentially increase the efficiency and reduce errors. Qualcomm together with a growing number of eco-system partners that offer a variety of wireless health solutions are well-positioned to help the industry. We see synergy by teaming with goBalto.”

goBalto develops next-generation, cloud-based solutions that simplify how clinical trials are conducted in the pharmaceutical, biotechnology, and medical device industries. Our flagship product, Tracker, is a purpose-built software-as-a-service clinical research tool. It enables clinical trial sponsors and research organizations to track and collaborate on operational data in a transparent, regulatory-compliant, and user-friendly way. Founded 2008 in Silicon Valley by biotechnology industry veteran Jae Chung, goBalto is backed by leading health technology investors. For more information, visit www.gobalto.com.

Halozyme Therapeutics Enters Lucrative Agreement With Pfizer

Halozyme Therapeutics, Inc. recently announced it has entered into a worldwide collaboration and license agreement with Pfizer Inc. for the purpose of developing and commercializing products combining proprietary Pfizer biologics with Halozyme’s Enhanze technology. Enhanze is Halozyme’s proprietary drug delivery platform and is based on the company’s patented recombinant human hyaluronidase enzyme (rHuPH20).

Under the terms of the agreement, Halozyme has granted to Pfizer a worldwide license to develop and commercialize products combining rHuPH20 with Pfizer proprietary biologics directed to up to six targets. Targets may be selected on an exclusive or non-exclusive basis. Halozyme will receive an initial payment of $8 million, which includes the up-front fee for exclusive licenses to two specified therapeutic targets in primary care and specialty care indications and the right for Pfizer to elect up to four additional targets upon payment of additional fees.

“I am delighted about this opportunity as it has the potential to enhance Pfizer’s ability to optimize treatments for patients,” said Jose Carlos Gutierrez-Ramos, Senior Vice President, Pfizer BioTherapeutics R&D.

Halozyme is eligible to receive additional payments upon Pfizer’s achievement of specified development, regulatory, and sales-based milestones, totaling up to $507 million. Halozyme is also entitled to royalty payments based on net sales of any licensed products.

“We look forward to working with Pfizer to apply Enhanze to these exciting targets,” added Gregory I. Frost, PhD, President and Chief Executive Officer, Halozyme. “Enhanze enables biologics to be delivered as a simple subcutaneous injection.”

Enhanze is Halozyme’s proprietary drug delivery platform based on the company’s patented recombinant human hyaluronidase enzyme (rHuPH20). rHuPH20 acts by removing traditional limitations on the volume of biologics that can be delivered subcutaneously (just under the skin). By using Enhanze, some biologics that are administered intravenously may instead be delivered subcutaneously. Enhanze may also benefit subcutaneous biologics by reducing the need for multiple injections. This delivery may improve patient convenience and reduce overall costs to the healthcare system.

Halozyme Therapeutics is a biopharmaceutical company dedicated to developing and commercializing innovative products that advance patient care. With a diversified portfolio of enzymes that target the extracellular matrix, the company’s research focuses primarily on a family of human enzymes, known as hyaluronidases, that increase the absorption and dispersion of biologics. For more information, visit www.halozyme.com.