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BioCentury Extra
As published Wednesday, May 27, 2015 6:47 PM PST


  • Express Scripts mulls indication-based pricing in cancer

    Express Scripts Holding Co. (NASDAQ:ESRX) is in discussions with manufacturers about indication-based pricing for cancer drugs. Under the model, a drug approved for different indications could have different prices depending on the strength of evidence and the nature of outcomes it produces (see BioCentury, Sept. 1).

    Spokesperson David Whitrap said the company introduced the idea of indication-based payments to clients two weeks ago at its Outcomes Symposium, and said clients have been "overwhelmingly supportive" of the approach.

    Express Scripts CMO Steve Miller told BioCentury in March that he believes new drug pricing models are necessary to provide a more sustainable solution to delivering new drugs to patients without bankrupting the system. Miller said payers and manufacturers are more motivated to try new models and are in a better position to do so now with "improved IT systems, great new products in the marketplace and a real need for this because of the tremendously high price of drugs" (see BioCentury, March 23).

  • Astellas adds muscle disease, ophthalmology TAs

    Astellas Pharma Inc. (Tokyo:4503) released a new three-year strategic plan that adds muscle diseases and ophthalmology as therapeutic areas. The company's innovative medicines strategy for FY15-17 also will include development of next-generation vaccines and cell therapies to treat cardiovascular disease and cancer.

    The announcement comes about two years after the Japanese pharma said it would restructure R&D and focus on accessing earlier stage assets via partnerships (see BioCentury, June 17, 2013).

    That same year, Astellas partnered with Cytokinetics Inc. (NASDAQ:CYTK) to develop treatments for musculoskeletal diseases. Under the updated agreement, the partners are developing CK-2127107, a fast skeletal muscle troponin activator, to treat spinal muscular atrophy (SMA) (see BioCentury Extra, Jan. 5, 2015).

    Astellas also said it would explore muscle diseases in partnership with Mitobridge Inc. (formerly Mitokyne, Cambridge, Mass.).

    In its announcement, Astellas said it would focus its efforts in ophthalmology on posterior eye disorders, including retinitis pigmentosa, through a collaboration with Harvard Medical School. It said ASP8232, an oral compound whose mechanism has not been not disclosed, is in Phase II to treat diabetic macular edema (DME).

    The company said its regenerative medicine unit, which was established last month, is not expected to enter the clinic for "a few years."

    Astellas also gave financial guidance, saying it would aim to maintain at least a 15% return on equity beyond the plan's three-year term, with consolidated sales CAGR in the mid-single digits, operating profit exceeding sales growth rates and core EPS exceeding core operating profit.

    Sales growth would come outside of Japan, which now amounts to 40% of turnover, with high single-digit growth anticipated in Europe and the Americas, and mid-teens growth in Asia outside of Japan, which currently account for about 6% of global sales.

    Astellas expects about JY300 billion ($2.5 billion) in peak sales from compounds in eight Phase III indications and two others in registration. The latter includes evolocumab (AMG145), the PCSK9 inhibitor for hypercholesterolemia partnered with Amgen Inc. (NASDAQ:AMGN).

    Astellas expects mid- to long-term growth to be driven by its franchises in overactive bladder (OAB) and cancer. The company anticipates a high-single-digit compound growth for OAB drug Myrbetriq mirabegron, marketed in Europe as Betmiga and in Japan as Betanis, and a mid-20s CAGR for prostate cancer drug Xtandi enzalutamide.

    The company will seek to keep R&D expenses higher than 17% of sales, and provide at least a 6% dividend.

    The company's existing core therapeutic areas include urology, oncology, immunology, nephrology and neuroscience.

  • Acorda responds to first Bass challenge

    Acorda Therapeutics Inc. (NASDAQ:ACOR) responded to an inter partes review (IPR) filed by hedge fund manager Kyle Bass in February against U.S. Patent No. 8,633,685, a methods patent covering multiple sclerosis drug Ampyra dalfampridine.

    The patent, which expires in 2025, is one of five Orange Book-listed patents for the sustained-release formulation of 4-aminopyridine (4-AP). Bass filed another IPR in February challenging Acorda's U.S. Patent No. 8,007,826, which covers Ampyra and expires in 2027. Acorda intends to file its response to the second IPR by June 19.

    In the challenge against the '685 patent, Coalition for Affordable Drugs, an entity associated with Bass, argued that a method for improving walking in MS patients by administering 10 mg of Ampyra twice daily for at least two weeks was "known in the prior art" (see BioCentury Extra, Feb. 11).

    In its response, Acorda said the Patent Trials and Appeals Board (PTAB) of the U.S. Patent and Trademark Office should deny the IPR against the '685 patent, in part because use of the IPR process "as a tool to manipulate markets is not what Congress intended."

    Acorda also claimed the petition should be denied because the Coalition did not name its investors and identify them as "real parties-in-interest."

    The rebuttal said the Coalition's arguments that Acorda's claims were available in the prior art were flawed because they referenced poster presentations. Such arguments must refer to patents or printed publications; Acorda said the posters do not qualify as printed publications because the Coalition did not present evidence that they would have been available to anyone who did not attend the corresponding conferences.

    Acorda argued that the prior art claims challenged by the petitioner had already been considered by the PTO during the prosecution of both the '685 and '826 patents. Furthermore, Acorda said the petition failed to link Acorda's claims to specific prior art patents or publications.

    Lastly, Acorda said the time frame of administering a particular dose of Ampyra for "at least two weeks" was not explicitly disclosed in the prior art.

    In 1Q15, Acorda posted $92.4 million in Ampyra sales, constituting 93% of the company's total revenue.

    Bass has filed challenges against at least eight pharma patents so far. The PTO has not decided whether it will accept any of his petitions (see BioCentury, May 18).

    Acorda shed $0.12 to $31.49 on Wednesday. The company's share price has fallen 21% since closing at $39.91 on Feb. 9, before Bass' petition was filed.

  • Juno, Editas to collaborate on CAR T therapies

    Juno Therapeutics Inc. (NASDAQ:JUNO) and Editas Medicine (Cambridge, Mass.) partnered to develop chimeric antigen receptor (CAR) T cell and T cell receptor (TCR) therapies for cancer. The partners will develop three undisclosed programs using Juno's CAR and TCR technologies and Editas' gene editing platform that uses CRISPR/Cas9 (CRISPR-associated protein 9).

    Editas will receive $25 million up front and is eligible for $230 million in milestones for each research program, plus tiered royalties. Juno also will provide Editas with up to $22 million in total research funding over five years.

    Editas spokesperson Dan Budwick declined to discuss which targets or cancer types the companies will pursue, nor would he say which party will pay for development, commercial or regulatory costs.

  • SciClone buys Chinese rights to Vibativ

    Theravance Biopharma Inc. (NASDAQ:TBPH) granted SciClone Pharmaceuticals Inc. (NASDAQ:SCLN) exclusive rights to the antibiotic Vibativ telavancin in China, Hong Kong, Macau, Taiwan and Vietnam. Theravance is eligible to receive $6 million, including upfront and milestone payments. SciClone will be responsible for development and commercialization costs.

    Theravance expects SciClone to begin a Phase III trial in China once a clinical trial authorization (CTA) is approved. SciClone may pursue accelerated approval in China, where overprescription of antibiotics has led to issues with resistance.

    The injectable lipoglycopeptide is approved in the U.S. to treat complicated skin and skin structure infections (cSSSIs) caused by susceptible Gram-positive bacteria, as well as nosocomial and ventilator-associated pneumonia caused by Staphylococcus aureus. It is approved in the EU to treat hospital-acquired pneumonia known or suspected to be cause by methicillin-resistant S. aureus (MRSA).

    SciClone gained $0.12 to $9.58 on Wednesday. Theravance gained $0.38 to $14.55.

  • Retrophin rises on $245M voucher sale to Sanofi

    Retrophin Inc. (NASDAQ:RTRX) jumped $3.18 (12%) to $30.05 on Wednesday after selling a Priority Review voucher to Sanofi (Euronext:SAN; NYSE:SNY) for $245 million. Sanofi spokesperson Mary Kathryn Steel declined to disclose what the company will do with the voucher.

    Retrophin will receive $150 million upfront from Sanofi, plus payments of $47.5 million in 2016 and 2017.

  • FDA approves pair of IBS therapies

    FDA approved Viberzi eluxadoline (JNJ-270189066) from Actavis plc (NYSE:ACT) and Xifaxan rifaximin from Valeant Pharmaceuticals International Inc. (TSX:VRX; NYSE:VRX) to treat irritable bowel syndrome with diarrhea (IBS-D) in adults.

    Actavis obtained Viberzi, a locally acting mu opioid receptor agonist and delta opioid receptor antagonist, via its 2014 acquisition of Forest Laboratories Inc. The compound was under Priority Review and had Fast Track designation. Actavis said it expects to launch in 1Q16 after the drug receives final scheduling designation from the U.S. Drug Enforcement Administration.

    Valeant acquired rights to Xifaxan when it bought Salix Pharmaceuticals Ltd. earlier this year. Salix had submitted a response last year to an FDA complete response letter for its sNDA for Xifaxan, including data from the Phase III TARGET 3 trial. The product, an antibiotic derived from rifampin, is approved to treat travelers' diarrhea and to reduce recurrence of overt hepatic encephalopathy in adults.

  • Calimmune raises $15M in series B

    Gene therapy company Calimmune Inc. (Tucson, Ariz.) raised $15 million in a series B round led by AbbVie Biotech Ventures Inc., the venture arm of AbbVie Inc. (NYSE:ABBV). New investor Alexandria Venture Investments and existing investors RA Capital and Translational Accelerator also participated.

    Calimmune is conducting a Phase I/II study of lead candidate Cal-1, an autologous T cell and blood stem cell therapy that blocks CC chemokine receptor 5 (CCR5). The therapy is designed as a one-time infusion to prevent HIV patients from progressing to AIDS. The current cohort of 12 patients were previously treated with highly active antiretroviral therapy (HAART). CEO Louis Breton told BioCentury the company is planning to enroll additional cohorts over the next six to nine months, including HIV patients with a malignancy requiring bone marrow transplantation. The company expects to report some data within 12 months, and full data within 20-24 months.

    "Most gene medicines have been in monogenic diseases or sometimes very rare diseases," noted Breton, "but our focus is to look at very broad diseases."

  • SCOTUS declines to review drug disposal suit

    The U.S. Supreme Court declined to review a challenge from the biopharma industry against a drug disposal program in Alameda County, Calif., that requires drug companies to cover the cost of collecting and disposing of unused drugs from county residents.

    The Safe Drug Disposal Ordinance was passed in July 2012. In December 2012, The Pharmaceutical Research and Manufacturers Association (PhRMA), the Generic Pharmaceutical Association (GPhA) and the Biotechnology Industry Organization (BIO) filed a lawsuit against the county in the U.S. District Court for the Northern District of California, arguing that the ordinance violated the U.S. Constitution's Commerce Clause.

    In August 2013, the district court ruled that the ordinance was not unconstitutional. The U.S. Court of Appeals for the Ninth Circuit affirmed the district court decision in September 2014. PhRMA, GPhA and BIO subsequently asked SCOTUS to review the appellate court decision, arguing that the county program forces drug companies "to perform an uncompensated local public service at the expense of the interstate market."


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