The Trans-Pacific Partnership trade treaty will set a floor of five years of data protection for biologics, U.S. Trade Representative Michael Froman announced Monday. Details of the agreement have not been released. Statements from Froman and Australian Minister for Trade and Investment Andrew Robb stressed the role of regulatory and administrative mechanisms in addition to data exclusivity to delay the market entry of biosimilars, but did not provide details. The TPP was negotiated by 12 countries, including the U.S., Japan and Australia.
TPP will include commitments for administrative and regulatory procedures that will have the effect of extending market exclusivity in countries that adopt the five-year period, Froman said at a press conference. "What we are doing in TPP is recognizing that we are all trying to achieve effective market protection and deliver a comparable outcome through various mechanisms, including at least five years of data protection, plus other government measures that can achieve a comparable outcome," Froman said.
He noted that the U.S. provides 12 years of data protection for biologics, and said other countries can achieve "a comparable outcome in terms of incentivizing innovation" through requirements for approval of biosimilars. Froman said regulatory and administrative requirements can add "seven or eight years beyond five years of data protection for various biosimilars to be approved."
Australia provides five years of data protection for biologics. Robb said he agreed with Froman's assessment of the treaty's biologics provisions. "The agreement recognizes that we can travel on different roads but end up at the same outcome" on protecting market exclusivity for biologics, Robb said at the press conference.
The Biotechnology Industry Organization (BIO) and the Pharmaceutical Research and Manufacturers of America (PhRMA) each released statements expressing disappointment that the agreement does not mirror the data protection provided under U.S. law.
In addition to biologics exclusivity, TPP includes provisions intended to make government procurement, including purchases of pharmaceuticals, more transparent.
Congress will have 90 days to review the TPP, including 60 days for public review of the treaty text, after President Obama officially provides notification of the agreement. Congress cannot modify the deal. The administration will need a majority of votes in both the House and Senate to ratify the treaty.
Representatives from HHS, Express Scripts Holding Co. (NASDAQ:ESRX) and Pfizer Inc. (NYSE:PFE) suggested that innovative, value-based pricing models could help to address concerns about the high cost of specialty drugs.
The comments came Monday at a Pew Charitable Trusts forum to discuss policy options for managing the cost of PCSK9 inhibitors and other specialty drugs.
"We've turned around our R&D model to be more innovative, now we need to innovate on pricing," said Kirsten Axelsen, VP of worldwide policy at Pfizer.
The panelists did not suggest any specific new pricing models for the recently launched PCSK9 inhibitors Repatha evolocumab from Amgen Inc. (NASDAQ:AMGN) and Praluent alirocumab from Sanofi (Euronext:SAN; NYSE:SNY) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN).
Express Scripts CMO Steven Miller said the PBM is using more traditional utilization management tools such as prior authorization to control the costs of Repatha and Praluent.
Miller said Express Scripts has blocked about 60% of the prescriptions it has gotten for the new cholesterol-lowering medications either because the patients don't meet the labeled indications for the drug or because doctors haven't documented the patient's LDL levels and/or treatment history, which are among the prior authorization requirements for the mAbs.
He said that as physicians become more familiar with these requirements, he expects the rate of denials will decrease.
Both PCSK9 drugs are approved as an adjunct to diet and maximally tolerated statin therapy in adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD). Repatha is also approved to treat homozygous familial hypercholesterolemia (HoFH).
Although stakeholders did not discuss new models for PCSK9 drugs, HHS Assistant Secretary for Planning and Evaluation Richard Frank said the agency is evaluating potential new drug pricing models. He gave as an example an outcomes-based payment model for diabetes therapies in which the price of the drug might be higher for patients who attain a prespecified HbA1c target, and lower for those who do not.
Miller said Express Scripts plans to pilot an indication-based pricing model for cancer drugs for plan year 2016.
Acorda Therapeutics Inc. (NASDAQ:ACOR) gained $3.37 (12%) to $31.42 on Monday after it said it settled a patent dispute with Allergan plc (NYSE:AGN). According to a regulatory filing, Allergan will be permitted to market a generic version of Acorda's Ampyra dalfampridine "at a specified date in 2027," or potentially sooner "under certain circumstances." Details of the settlement are undisclosed.
Acorda filed suit in the U.S. District Court of Delaware in July 2014 after Allergan submitted an ANDA for generic Ampyra, claiming the generic would violate four patents covering the multiple sclerosis treatment. Allergan was then known as Actavis plc. The latest expiration date for an Ampyra patent listed in the Orange Book is May 2027.
Ampyra is a sustained-release formulation of 4-aminopyridine (4-AP).
Sequencing company Illumina Inc. (NASDAQ:ILMN) sank $23.66 (15%) to $139.51 in early after-hours trading Monday after reporting 3Q15 revenues and giving 4Q15 guidance that missed the Street's estimates.
Illumina posted $550 million in 3Q15 revenues, a 14% increase from $481 million in 3Q14 but below analysts' estimates of $568.6 million. The company also reported 4Q15 revenue guidance of $570 million, below the Street's estimate of $602.8 million.
The company said it was "disappointed with instrument sales in Europe and continued weakness in the Asia-Pacific region."
The company will report full 3Q15 results on Oct. 20.
Biosimliar candidates ONS-1045 from Oncobiologics Inc. (Cranbury, N.J.) and M923 from Baxalta Inc. (NYSE:BAX) and Momenta Pharmaceuticals Inc. (NASDAQ:MNTA) each reached clinical milestones on Monday.
Oncobiologics said ONS-1045, a biosimilar of cancer antibody Avastin bevacizumab from Roche (SIX:ROG; OTCQX:RHHBY), met the primary and secondary bioequivalence endpoints in a Phase I pharmacokinetic (PK) study. The company plans to begin a Phase III trial of ONS-1045 in 1H16.
Oncobiologics has eight biosimilar candidates in its pipeline. It raised $31 million in a venture round in July (see BioCentury Extra, July 27).
Amgen Inc. (NASDAQ:AMGN) and partner Allergan plc (NYSE:AGN); Pfizer Inc. (NYSE:PFE); and Boehringer Ingelheim GmbH (Ingelheim, Germany) all have Avastin biosimilar candidates in Phase III. AstraZeneca plc (LSE:AZN; NYSE:AZN) and Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151) have a biosimilar of Avastin in Phase I testing. Avastin is a humanized mAb against VEGF (see BioCentury Extra, Sept. 25).
On Monday, Baxalta and Momenta started a pivotal trial of M923, a biosimilar of Humira adalimumab from AbbVie Inc. (NYSE:ABBV), in patients with chronic plaque psoriasis. The partners hope to submit M923 to regulators in 2017 and launch the biosimilar in 2018.
Momenta partnered with Baxter International Inc. (NYSE:BAX) in 2011 to develop up to six biosimilars including M923. Baxalta spun out of Baxter in July.
Samsung Bioepis Co. Ltd. and Merck & Co. Inc. (NYSE:MRK) have said they plan to submit Humira biosimilar SB5 for approval by YE16, while Amgen previously disclosed plans to launch its Humira biosimilar ABP 501 in 2017 (see BioCentury Extra, July 8).
Momenta gained $0.59 to $17.42 on Monday.
Alkermes plc (NASDAQ:ALKS) gained $3.73 to $63.50 in after-hours trading after it said FDA approved Aristada aripiprazole lauroxil to treat schizophrenia. The company said it is preparing to launch Aristada "immediately." Alkermes spokesperson Kathryn Morris declined to disclose a price for the injectable, extended-release formulation of aripiprazole using LinkeRx technology.
Otsuka Pharmaceutical Co. Ltd. (Tokyo, Japan) and H. Lundbeck A/S (CSE:LUN) market Abilify Maintena, a once-monthly intramuscular depot formulation of aripiprazole (see BioCentury Extra, July 13).
Aripiprazole is a small molecule partial agonist of the dopamine D2 and serotonin (5-HT1A) receptors and an antagonist of the serotonin (5-HT2A) receptor.
Alkermes announced Aristada's approval after market close. It shed $0.71 to $59.77 on Monday.
Metabolic company Arena Pharmaceuticals Inc. (NASDAQ:ARNA) said President and CEO Jack Lief has retired at the request of the board of directors. Arena board member Harry Hixson will be interim CEO and interim principal financial officer while the company searches for a new CEO.
Oncology company Tokalas Inc. (Rancho Santa Fe, Calif.) hired James Breitmeyer as president and CEO. Breitmeyer was EVP at Bavarian Nordic A/S (CSE:BAVA).
Pain play Egalet Corp. (NASDAQ:EGLT) promoted Mark Strobeck to EVP and COO from CBO.
Spark Therapeutics Inc. (NASDAQ:ONCE) jumped $9.09 (21%) to $53.02 on Monday after its SPK-RPE65 met the primary endpoint and two of three secondary endpoints in a Phase III trial to treat retinal pigment epithelium-specific protein 65kDa (RPE65)-mediated inherited retinal dystrophies (IRDs). Spark plans to submit a BLA next year for SPK-RPE65.
The adeno-associated virus (AAV) carrying the RPE65 gene met the primary endpoint of improving functional vision vs. the control group (p=0.001). The endpoint was measured by change in bilateral mobility testing from baseline to one year; Spark co-founder and CEO Jeffrey Marrazzo said Spark developed the novel endpoint internally. On a conference call Monday, Marrazzo said two-thirds of patients in the treatment group were able to navigate the mobility course in the lowest lighting conditions vs. zero patients in the control group.
SPK-RPE65 also met the secondary endpoints of full-field light sensitivity threshold testing (p<0.001) and the change in mobility test score for the first eye injected from baseline to one year, both vs. control (p=0.001). The therapy did not show a statistically significant benefit in visual acuity (p=0.17).
Additional data from the 31-patient study will be presented at the Retina Society Annual Scientific Meeting in Paris on Oct. 10.
Mutations in the RPE65 gene are linked to subtypes of Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP). SPK-RPE65 has Orphan Drug designation in the U.S. and EU for LCA and RP and breakthrough therapy designation in the U.S. for LCA.
Fidelity Biosciences joined with the technology venture arm of Devonshire Investors to form F-Prime Capital. Devonshire is a private equity firm affiliated with Fidelity's parent company, FMR LCC. The firm said the re-branding would "clarify our mission" as a VC investor that is separate from Fidelity's mutual fund business.
Spokesperson Michael Aalto said Fidelity's investment strategies would remain unchanged following the name change, with investments in life sciences, healthcare and technology.
Fidelity Biosciences' investments include Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), Unum Therapeutics Inc. (Cambridge, Mass.) and Adaptimmune Therapeutics plc (NASDAQ:ADAP).
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