Sangamo Therapeutics and Pfizer Announce Collaboration for Hemophilia A Gene Therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. announced this week, an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter.

“Sangamo brings deep scientific and technical expertise across multiple genomic platforms, and we look forward to working together to advance this potentially transformative treatment for patients living with Hemophilia A,” said Mikael Dolsten, MD, PhD, President of Worldwide Research and Development at Pfizer. “Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing. We believe SB-525 has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of Factor VIII protein with a single administration treatment.”

“With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program,” said Dr. Sandy Macrae, Sangamo’s Chief Executive Officer. “We believe Pfizer’s end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases.”

Under the terms of the collaboration agreement, Sangamo will receive a $70 million upfront payment from Pfizer. Sangamo will be responsible for conducting the SB-525 Phase 1/2 clinical study and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for SB-525 and additional products, if any. Sangamo is eligible to receive potential milestone payments of up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for additional Hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will also receive tiered double-digit royalties on net sales. Additionally, Sangamo will be collaborating with Pfizer on manufacturing and technical operations utilizing viral delivery vectors.

Gene therapy is a potentially transformational technology for patients, focused on highly specialized, one-time, treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patient’s cells to compensate for a defective one. The genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector such as rAAV. There have been no gene therapy products approved in the U.S. to date.

Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A. SB-525 is comprised of a rAAV vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has cleared initiation of human clinical trials for SB-525, which also has been granted orphan drug designation. Sangamo is on track this quarter to start a Phase 1/2 clinical trial to evaluate safety and to measure blood levels of Factor VIII protein and other efficacy endpoints.

 

Pfizer boosts cancer drug pipeline with $14 billion Medivation deal

BLOOMBERG — Pfizer Inc said on Monday it would buy U.S. cancer drug company Medivation Inc., in a deal valued at about $14 billion, adding blockbuster prostate cancer drug Xtandi to its portfolio.

Medivation shares were up 20 percent at $80.56 in premarket trade, just shy of the offer price of $81.50 per share in cash.

The offer is at a substantial premium to Sanofi SA’s (SASY.PA) initial offer to buy Medivation for $52.50 in April that pushed the San Francisco-based company to put itself up for sale.

The deal comes four months after Pfizer and Ireland-based Allergan Plc scrapped their $160 billion merger. Pfizer has since bought Anacor Pharmaceuticals Inc ANAC.O in a $5.2 billion deal to add an eczema gel to its portfolio.

The deals hints at a shift in Pfizer’s M&A strategy from lowering taxes – the rationale behind the failed Allergan deal – to strengthen its drugs portfolio ahead of a decision on selling or spinning off its generic drugs business by late 2016.

Pfizer, whose oncology offerings include breast cancer drug Ibrance and several other promising immuno-oncology products, will now get access to Xtandi as well as Talazoparib, another breast cancer treatment under development.

Xtandi, which generated U.S. net sales of $330.3 million in the second quarter, has posted double-digit growth, putting Medivation on track to achieve its target of more than 50 percent in revenue growth for the year.

Japanese drugmaker Astellas Pharma Inc (4503.T) owns the rights to sell Xtandi outside the United States.

“We believe the combination of Xtandi and talazoparib has the potential to be a uniquely value-added combination in the treatment of prostate cancer and should be closely watched,” Leerink analysts wrote in a client note.

Reuters had reported that Pfizer, Merck & Co Inc (MRK.N), Celgene Corp (CELG.O) and Gilead Sciences Inc (GILD.O) had submitted expressions of interest to buy Medivation.

“Given the already very high price being discussed, the difficult public relationship between Medivation and Sanofi … we see a higher bid as very unlikely, but not impossible,” RBC Capital Partners wrote in a client note ahead of the announcement.

Sanofi said while it recognized the potential strategic benefits of a combination with Medivation, it was a “disciplined acquirer and remained committed to acting in the best interests of Sanofi shareholders.”

Pfizer said it expects to complete the acquisition, which was approved by boards of both companies, in the third or the fourth quarter.

Pfizer’s financial advisers were Guggenheim Securities and Centerview Partners, with Ropes & Gray LLP providing legal counsel.

J.P. Morgan Securities and Evercore were Medivation’s financial advisers, while Cooley LLP and Wachtell, Lipton, Rosen & Katz served as its legal advisers.

Pfizer shares were marginally down at $34.80 in premarket.

Up to Friday’s close, Medivation shares had risen about 58 percent since Sanofi’s first offer.

(Reporting by Ankur Banerjee in Bengaluru; Editing by Don Sebastian and Anil D’Silva)

Pfizer acquires Bamboo Therapeutics to beef up gene therapy arsenal

(Reuters) – Pfizer Inc said it had acquired privately held gene therapy developer Bamboo Therapeutics Inc in a deal worth up to $645 million to boost its presence in the treatment of rare diseases.

Research into gene therapy, which aims to insert corrective genes into malfunctioning cells, goes back a quarter of a century but the field has experienced multiple setbacks and been plagued by safety concerns.

However, the discovery of better ways to carry replacement genes into cells is building optimism.

The U.S. Food and Drug Administration has yet to approve any gene therapies but Europe has approved two – a treatment from GlaxoSmithKline for a rare immune disorder in babies and one from uniQure NV for a serious blood condition.

Genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector.

Bamboo was formed in 2014 to advance the work of Dr. Richard Jude Samulski, who is considered a pioneer in the field after he became the first to realize the potential of using adeno-associated virus’s (AAV) as a vehicle to replace a defective gene with a healthy gene.

Through the acquisition, Pfizer will gain access to Bamboo’s experimental gene therapies for rare diseases such as Duchenne Muscular Dystrophy (DMD), giant axonal neuropathy (GAN), Friedreich ataxia (FA) and Canavan disease.

Focused on neurological and neuromuscular diseases, Bamboo’s drugs are still in the preclinical or early stages of development. Pfizer is paying the Chapel Hill, North Carolina-based company $150 million upfront, and Bamboo stands to make $495 million in milestone payments.

Pfizer has been investing in gene therapies – touted as a one-time cure for intractable and expensive-to-treat diseases – in recent years.

In 2014, the drugmaker entered into a collaboration with Philadelphia-based Spark Therapeutics Inc to develop SPK-9001, a gene therapy for hemophilia B.

Earlier this year, Pfizer signed a collaboration and license agreement with Emeryville, California-based 4D Molecular Therapeutics to develop targeted vectors for cardiac disease.

Other big drugmakers have made similar investments. Bristol-Myers Squibb Co has a tie-up with uniQure to develop gene therapies for heart diseases, while Celgene Corp has teamed up with bluebird bio Inc for cancer.

 

Pfizer to Acquire Anacor Citing Strong fit with Pfizer’s Inflammation and Immunology portfolio

Pfizer Inc. and Anacor Pharmaceuticals, Inc. today announced that they have entered into a definitive merger agreement under which Pfizer will acquire Anacor for $99.25 per Anacor share, in cash, for a total transaction value, net of cash, of approximately $5.2 billion, which assumes the conversion of Anacor’s outstanding convertible notes. The Boards of Directors of both companies have unanimously approved the transaction. Anacor’s flagship asset, crisaborole, a differentiated non-steroidal topical PDE4 inhibitor with anti-inflammatory properties, is currently under review by the U.S. FDA for the treatment of mild-to-moderate atopic dermatitis, commonly referred to as eczema.

“We believe the acquisition of Anacor represents an attractive opportunity to address a significant unmet medical need for a large patient population with mild-to-moderate atopic dermatitis, which currently has few safe topical treatments available”

“We believe the acquisition of Anacor represents an attractive opportunity to address a significant unmet medical need for a large patient population with mild-to-moderate atopic dermatitis, which currently has few safe topical treatments available,” said Albert Bourla, Group President of Pfizer’s Global Innovative Pharma and Global Vaccines, Oncology and Consumer Healthcare Businesses. “Crisaborole is a differentiated asset with compelling clinical data that, if approved, has the potential to be an important first-line treatment option for these patients and the physicians who treat them.”

“Anacor will be a strong fit with Pfizer’s innovative business, further supporting our strategic focus on Inflammation and Immunology, and is expected to enhance near-term revenue growth for the innovative business. Our dedicated Inflammation and Immunology group has strong existing in-market franchises with Enbrel and Xeljanz, as well as a robust mid-stage pipeline, and this acquisition has the potential to add a near-term U.S. product launch. We believe we are well positioned to maximize crisaborole’s commercial potential through our strong relationships with pediatricians and primary care physicians,” continued Bourla.

In both of its Phase 3 pivotal studies, crisaborole achieved statistically significant results on all primary and secondary endpoints and in March 2016, the FDA accepted for review Anacor’s New Drug Application seeking approval of crisaborole for the potential treatment of mild-to-moderate atopic dermatitis in children and adults. The Prescription Drug User Fee Act (PDUFA) goal date for the completion of the FDA’s review is January 7, 2017. If approved, Pfizer believes peak year sales for crisaborole have the potential to reach or exceed $2.0 billion.

“Today marks the beginning of an exciting new chapter for Anacor, which we believe will deliver significant value to our shareholders,” said Paul L. Berns, Anacor’s Chairman and Chief Executive Officer. “We have a deep respect for Pfizer, and it is clear that they share our commitment to addressing the significant unmet medical needs in inflammatory disease. We are proud of the innovative company that our team has built and are confident that Pfizer will help accelerate Anacor’s important mission given the strength of its global platform and resources.”

Atopic dermatitis is a common, relapsing, chronic, inflammatory skin disorder, with patients displaying a chronic rash characterized by inflammation and itching, often occurring in folds of the skin with symptoms lasting up to 14 days or more. Approximately 18 to 25 million people in the United States suffer from this condition, including between 8 and 18% of infants and children. Atopic dermatitis has been considerably underdiagnosed due to the lack of approved effective systemic agents, and limitations of current topical agents. There have been no new molecular entities for atopic dermatitis in the last 15 years.

Anacor also holds the rights to Kerydin, a topical treatment for onychomycosis (toenail fungus) that is distributed and commercialized by Sandoz Inc. in the U.S.

Pfizer anticipates financing the transaction through existing cash. Pfizer does not expect the transaction to impact its current 2016 financial guidance. Pfizer expects the transaction to be slightly dilutive to Adjusted Diluted Earnings Per Share (EPS)(1) in 2017 with accretion to Adjusted Diluted EPS(1) beginning in 2018 and increasing thereafter.

Under the terms of the merger agreement, a subsidiary of Pfizer will commence a cash tender offer to purchase all of the outstanding shares of Anacor common stock for $99.25 per share in cash. The closing of the tender offer is subject to customary closing conditions, including U.S. antitrust clearance and the tender of a majority of the outstanding shares of Anacor common stock. The merger agreement contemplates that Pfizer will acquire any shares of Anacor that are not tendered into the offer through a second-step merger, which will be completed promptly following the closing of the tender offer. Pfizer expects to complete the acquisition in the third-quarter 2016.

Pharma News: Pfizer approaches Medivation about potential takeover

Reuters – Pfizer Inc has approached U.S. cancer drug maker Medivation Inc to express interest in an acquisition, raising the possibility of a bid rivaling a $9.3 billion offer by Sanofi SA, people familiar with the matter said on Tuesday.

Pfizer’s approach comes less than a week after Sanofi went public with its $52.50 per share cash offer, complaining that Medivation refused to engage. Medivation subsequently rejected the offer as too low. Its shares closed on Tuesday at $57.52.

Medivation has not yet decided whether it should engage with Pfizer in negotiations and is in discussions with its financial and legal advisers, the people said. There is no certainty that Pfizer will press ahead with a bid, they added.

Sanofi currently has no plans to raise its offer and is waiting for Medivation to launch an auction to sell itself before it makes any new bid, some of the people said.

The sources asked not to be identified because the matter is not public. Medivation, Sanofi and Pfizer declined to comment.

Based in San Francisco, Medivation is best known for its oncology drug Xtandi, which treats prostate cancer.

For Pfizer, a deal with Medivation would mark another attempt at building scale in patented drugs after it scrapped its $160 billion acquisition of Dublin-based Allergan Plc (AGN.N) last month.

The breakdown came days after the U.S. Treasury issued new rules that weighed on Pfizer’s ability to slash its tax bill by using the deal to redomicile in Ireland.

Earlier on Tuesday, Pfizer Chief Executive Ian Read said in an interview with Reuters that he would consider another merger of any size, as long as the deal makes sense. He did not comment on Medivation.

Sanofi is vying for Medivation in an attempt to expand in the lucrative oncology sector, as it struggles to compensate for declining revenues from a key diabetes drug that recently lost patent protection.

Sanofi’s unsolicited approach for Medivation has echoes of its bid for rare disease drug maker Genzyme in 2011. It took Sanofi nine months to overcome Genzyme’s resistance. It also offered Genzyme shareholders so-called contingent value rights, which offered them additional payments if the acquired company was able to achieve certain performance milestones.

Using contingent value rights in the case of Medivation may be more challenging for Sanofi, given its lackluster track record in cancer drugs. However, Sanofi has no plans to use contingent value rights in any new offer, according to the sources.

Reuters  – Reporting by Lauren Hirsch and Carl O’Donnell in New York; Additional reporting by Ben Hirschler in London and Greg Roumeliotis in New York; Editing by Dan Grebler

 

Called Off: Pharmaceutical Giants Pfizer and Allergan to abandon $212 billion merger

US pharmaceutical giant Pfizer has announced it is abandoning plans for a US $160 billion ($212 billion) merger with Botox maker Allergan, citing new US rules cracking down on tie ups aimed at saving on taxes.

The deal with the Irish-based firm would have created the world’s largest pharmaceutical company.

Pfizer said in a statement that the two companies “terminated by mutual agreement” plans to merge.

“Pfizer approached this transaction from a position of strength and viewed the potential combination as an accelerator of existing strategies,” company chairman and CEO Ian Read said.

Pfizer also agreed to pay Allergan US$150 million to reimburse its expenses linked to the planned merger.

The decision to call off the merger came after the US Treasury Department announced new rules to discourage mergers between US and foreign businesses designed to sharply lower the US company’s tax bill.

The New York-based pharmaceutical giant said it remains on track to report its 2016 first quarter earnings on May 3.

For all of 2015, Pfizer reported earnings of US$7.7 billion, down 15.2 per cent from 2014. Revenues dipped 1.5 per cent to US$48.9 billion.

Allergan CEO Brent Saunders said in a separate statement that while he is “disappointed” that the merger will not proceed, his company is nevertheless “poised to deliver strong, sustainable growth built on a set of powerful attributes”.

Source AFP