Lexicon Pharmaceuticals Inc. (NASDAQ:LXRX) surged $5.16 (61%) to $13.60 on Monday, bringing the company's market cap to $1.4 billion, after telotristat etiprate met the primary endpoint in the Phase III TELESTAR trial to treat carcinoid syndrome. The company plans to submit an NDA to FDA in 1Q16.
Patients received 250 or 500 mg of oral telotristat etiprate thrice daily for 12 weeks. Both doses led to a statistically significant reduction from baseline vs. placebo in the average number of daily bowel movements (p<0.001).
After 12 weeks, 44% of patients in the 250 mg dose arm and 42% of patients in the 500 mg dose arm achieved a durable response vs. 20% in the placebo arm (p<0.04). A durable response was defined as at least a 30% reduction in daily bowel movements over at least half of the days of the study period.
Patients will continue to receive 500 mg of telotristat etiprate thrice daily for 36 weeks in an open-label extension study.
The tryptophan hydroxylase 1 (TPH1; TPH) inhibitor has Fast Track and Orphan Drug designation in the U.S. for this indication and Orphan Drug designation in the EU for carcinoid tumors.
Ipsen Group (Euronext:IPN; Pink:IPSEY) holds exclusive rights to commercialize telotristat epitrate outside of the U.S. and Japan.
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported earnings and said it is planning an early 2016 launch for drisapersen, its therapy for Duchenne muscular dystrophy amenable to exon 51 skipping. The antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene is under Priority Review by FDA with a Dec. 27 PDUFA date; EMA accepted for review an MAA for drisapersen in June.
CEO Jean-Jacques Bienaime reiterated on the earnings call that the company expects FDA to hold an advisory committee committee this year for drisapersen.
BioMarin reported revenues of $250.5 million in 2Q15, up 31% from 2Q14's $191.8 million and beating the consensus revenue estimate of $218.2 million. CFO Daniel Spiegelman said BioMarin would not change its full-year guidance.
Sales of Vimizim elosulfase alfa were $53.9 million; BioMarin launched the Orphan drug last year to treat mucopolysaccharidosis IVA (MPS-IVA; Morquio's syndrome). Naglazyme galsulfase sales were $111.1 million, up 13% from $98.3 million in 2Q14, but BioMarin said revenue from the MPS VI treatment "benefited from a large order in Latin America" that is "consistent with historically uneven sales in that region" and is unlikely to represent a positive trend.
The company's 2Q15 non-GAAP net loss was $5.4 million vs. income of $10.1 million in 2Q14, which BioMarin attributed to increased operating expenses partially offset by sales growth following Vimizim's launch.
BioMarin gained $0.90 to $147.17 on Monday. It reported earnings after market close.
AstraZeneca plc (LSE:AZN; NYSE:AZN) and Isis Pharmaceuticals Inc. (NASDAQ:ISIS) partnered to discover and develop antisense therapeutics against undisclosed targets for cardiovascular, metabolic and renal diseases.
Isis spokesperson Amy Blackley said Isis will be responsible for discovery, and AstraZeneca will lead development once candidates are selected.
Isis will receive $65 million up front and is eligible for undisclosed milestones for each program AZ chooses to advance into clinical development, plus tiered double-digit royalties.
The deal builds upon an existing 2012 collaboration to discover antisense therapeutics against four cancer targets. The partners expanded the deal in 2013 to include CV and metabolic diseases. AZ and Isis also partnered in 2014 to develop targeted delivery methods for antisense therapies (see BioCentury Extra, Nov. 13, 2014).
AZ is also collaborating with mRNA therapeutics developer Moderna Therapeutics Inc. (Cambridge, Mass.) to discover, develop and commercialize treatments for cancer and CV, metabolic and renal diseases (see BioCentury Extra, March 21, 2013).
Isis shed $0.97 to $53.96 on Monday.
Synthon B.V. (Nijmegen, the Netherlands) granted Pfizer Inc. (NYSE:PFE) exclusive U.S. commercialization rights to a generic version of glatiramer acetate for relapsing/remitting multiple sclerosis. Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) markets Copaxone glatiramer acetate.
The deal, for which terms were not disclosed, includes Synthon's once-daily and thrice-weekly formulations of glatiramer. Both are under FDA review. Synthon said it believes the thrice-weekly version "may be eligible for 180 days of shared marketing exclusivity" under Hatch-Waxman, but Pfizer spokesperson MacKay Jimeson declined to clarify who would share the rights, and would not tell BioCentury whether Pfizer or Synthon is planning to challenge Teva's U.S. patent on the thrice-weekly formulation.
In June, the Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) launched once-daily Glatopa glatiramer acetate after a federal appeals court invalidated the last U.S. patent for once-daily Copaxone.
Teva is converting patients to its thrice-weekly formulation of the selective major histocompatibility complex class II (MHC II) modulator, which has patent protection until 2030.
Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) will acquire a 51% stake in computational genomic analysis company Immuneering Corp. (Cambridge, Mass.). Teva spokesperson Denise Bradley said Teva will pay Immunoneering $45 million in three tranches, and will receive exclusive right of first refusal for Immuneering projects addressing CNS disorders.
Immuneering analyzes genomic and proteomic data to generate hypotheses and design follow-up studies for compounds in development. Last year, the companies co-authored a study comparing transcriptional responses to Teva's multiple sclerosis drug Copaxone glatiramer acetate versus a generic formulation.
Teva markets a portfolio of CNS drugs. The top seller by far is Copaxone, with $1.1 billion in sales during 2Q15. Sales of Parkinson's disease drug Azilect rasagiline were $105 million during the quarter, while sleep disorder drug Nuvigil armodafinil brought in $91 million. It also has MS therapy Nerventra laquinimod (SAIK-MS), an oral quinoline-3-carboxamide immunomodulator, in Phase III to treat relapsing-remitting MS.
Immuneering CEO Ben Zeskind told BioCentury the company's technology is applicable to diseases outside the CNS, and Teva will also have right of first look at internal candidates for other indications.
Bradley said Teva has the right to nominate two directors to Immuneering's board, and has an option to take control of the board.
Shire plc (LSE:SHP; NASDAQ:SHPG) acquired ophthalmic play Foresight Biotherapeutics Inc. (New York, N.Y.) for $300 million in cash. The deal gives Shire global rights to FST-100, an ophthalmic formulation of dexamethasone and povidone-iodine to treat infectious conjunctivitis. Shire expects to begin a Phase III study of the candidate, and spokesperson Gwen Fisher said the company has not yet determined a timeline.
Shire's ophthalmic pipeline includes lifitegrast (SAR 1118), which is under FDA review to treat dry eye with an Oct. 25 PDUFA date (see BioCentury Extra, April 9).
Shire gained $1.27 to $268.08 on Monday.
BeiGene Co. Ltd. (Beijing, China) said China FDA approved a clinical trial application (CTA) for BGB-283. The small molecule, second-generation BRAF inhibitor is BeiGene's first candidate to receive a CTA approval.
BeiGene said it plans to start its Chinese trial in 4Q15; the study will test BGB-283 in cancer patients with BRAF, K-Ras (KRAS), or neuroblastoma Ras viral (v-Rase) oncogene (NRAS) mutations. Last month, BeiGene started a Phase Ib study in Australia and New Zealand of BGB-283 in solid tumor patients. BeiGene did not respond to inquiries.
The company raised RMB600 million ($96.6 million) in May (see BioCentury, May 18).
Clovis Oncology Inc. (NASDAQ:CLVS) submitted an NDA to FDA and an MAA to EMA for rociletinib (AVL-301) to treat non-small cell lung cancer (NSCLC) in patients who have the EGFR T790M mutation and have been previously treated with an EGFR-targeted therapy.
Partner Qiagen N.V. (Xetra:QIA; NASDAQ:QGEN) plans to submit a supplemental PMA for a companion diagnostic that builds upon its FDA-approved therascreen EGFR Mutation Detection Kit RGQ, which has been established for 21 EGFR mutations including T790M. Qiagen and Clovis did not respond to inquiries.
Rociletinib, a small molecule that inhibits the T790M EGFR mutation and the initial activating EGFR mutations, has breakthrough therapy designation in the U.S. to treat EGFR T790-mutant NSCLC. Clovis has exclusive, worldwide rights to develop and commercialize rociletinib from Celgene Corp. (NASDAQ:CELG).
The Critical Path Institute formed the Duchenne Regulatory Sciences Consortium (D-RSC) with founding members Santhera Pharmaceuticals Holding AG (SIX:SANN); PTC Therapeutics Inc. (NASDAQ:PTCT); and Brenda Wong of Cincinnati Children's Hospital Medical Center. C-Path said additional members will be added to the consortium soon, including Sarepta Therapeutics Inc. (NASDAQ:SRPT), with which it is negotiating a final agreement.
C-Path said that the D-RSC will initially focus on building a disease progression model for DMD, and will seek U.S. and EU endorsement for new drug development tools.
Parent Project Muscular Dystrophy (PPMD) committed nearly $1 million towards the group. The Institute did not respond to inquiries.
inVentiv Health is a top tier professional services company that accelerates the clinical and commercial success of biopharmaceutical companies worldwide. inVentiv Health makes it possible for companies to be more efficient with resources and more effective throughout the product development life cycle. Click here for more information.