FDA granted accelerated approval to Lartruvo olaratumab from Eli Lilly and Co. (NYSE:LLY) in combination with doxorubicin to treat advanced soft tissue sarcoma patients who cannot be cured with radiation or surgery and for whom chemotherapy is appropriate.
Lilly spokesperson Karen Glowacki told BioCentury that Lartruvo would be available "in the coming weeks," and that the company has not yet set the drug's price.
The human IgG1 mAb targeting platelet derived growth factor receptor A (PDGFRA; PDGFR2; CD140A) has breakthrough therapy, Fast Track and Orphan Drug status from FDA in the indication, and was under Priority Review.
Lartruvo is also under EMA review. Last month, the agency's CHMP recomended its conditional approval (see BioCentury Extra, Sept. 16).
Cerulean Pharma Inc. (NASDAQ:CERU) gained $0.27 (40%) to $0.95 after announcing a deal with Novartis AG (NYSE:NVS; SIX:NOVN) to develop nanoparticle-drug conjugates (NDCs). The partners will pair Cerulean's Dynamic Tumor Targeting platform with Novartis compounds against up to five targets. The platform is designed to create molecules that attack tumor cells selectively and reduce toxicity.
Cerulean received $5 million up front in the agreement and is eligible for $1.2 billion in milestones among the five programs, including $41.5 million in development and regulatory milestones and $185 million in sales milestones for each target, plus tiered royalties. Novartis will have exclusive development and commercialization rights to any candidates.
The partners will choose the targets jointly. Both companies declined to disclose potential indications or targets for the collaboration.
Also Wednesday, Cerulean said Aspire Capital agreed to invest up to $20 million in the company via a 24-month at-the-market (ATM) share purchase agreement. The firm has already invested $1 million.
Merck & Co. Inc. (NYSE:MRK) said letermovir (MK-8228) met the primary endpoint of a Phase III study to prevent cytomegalovirus infection in CMV-seropositive adults who have received a hematopoietic stem cell transplant. Spokesperson Pamela Eisele said the company is planning regulatory submissions of the candidate, but declined to disclose a timeline.
The study evaluated the percentage of patients with clinically significant CMV infection through 24 weeks after transplant in patients receiving once-daily letermovir or placebo. Merck said it will present the results at an upcoming medical meeting.
Letermovir is a quinazoline-based inhibitor of CMV terminase protein. It has Fast Track designation in the U.S. and Orphan Drug designation in the U.S. and EU to prevent CMV infection in at-risk populations. Merck has exclusive, worldwide rights to letermovir from AiCuris GmbH & Co. KG (Wuppertal, Germany) under a 2012 agreement.
Fujifilm Kyowa Kirin Biologics Co. Ltd. said biosimilar candidate FKB327 met the primary endpoint in a global Phase III trial to treat moderate to severe rheumatoid arthritis by showing equivalence to reference product Humira adalimumab, as measured by ACR20 response.
Fujifilm Kyowa Kirin is a JV between Fujifilm Holdings Corp. (Tokyo:4901) and Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151).
Worldwide sales of Humira were $7.7 billion in the first six months of 2016. AbbVie Inc. (NYSE:ABBV) markets the human mAb against tumor necrosis factor (TNF) alpha.
Last month, Amjevita from Amgen Inc. (NASDAQ:AMGN) became the first biosimilar adalimumab to gain FDA approval. The agency approved it to treat RA, psoriatic arthritis, ankylosing spondylitis, Crohn's disease, ulcerative colitis, plaque psoriasis and polyarticular juvenile idiopathic arthritis (see BioCentury Extra, Sept. 23).
Although Amgen has said it could launch Amjevita as soon as 2017, AbbVie has claimed its IP will keep biosimilars off the U.S. market until 2022 (see BioCentury, July 25).
In August, Coherus BioSciences Inc. (NASDAQ:CHRS) said its biosimilar candidate CHS-1420 met the primary endpoint of similarity to Humira in a Phase III trial to treat active, moderate to severe chronic plaque psoriasis. Coherus said it hopes to launch the product in mid-2018 (see BioCentury Extra, Aug. 8).
Daiichi Sankyo Co. Ltd. (Tokyo:4568) suspended enrollment in the Phase III ENLIVEN trial of pexidartinib (PLX3397) to treat symptomatic tenosynovial giant cell tumor (TGCT). A data monitoring committee recommended the suspension after reviewing two reports of non-fatal liver toxicity in ENLIVEN, spokesperson Jennifer Brennan told BioCentury.
At the time of suspension, 121 of a planned 126 patients had been enrolled. Brennan said FDA has agreed to additional measures to address safety concerns and will allow the company to continue the study to completion. Brennan was uncertain when Daiichi expects final ENLIVEN results.
Pexidartinib is a small molecule inhibitor of CSF1R, c-Kit and FMS-like tyrosine kinase 3 (FLT3; CD135). It has breakthrough therapy designation from FDA to treat TGCT where surgical removal of the tumor may worsen the patient's condition.
Gene editing company CRISPR Therapeutics AG (NASDAQ:CRSP) gained $0.09 to $14.09 in its first day of trading Wednesday after raising a combined $91 million in a downsized IPO and concurrent private placement.
CRISPR Therapeutics raised $56 million through the sale of 4 million shares at $14 in the IPO. Citigroup, Piper Jaffray, Barclays and Guggenheim were underwriters. Separately, the company raised $35 million through the sale of 2.5 million shares at $14 to existing shareholder Bayer Global Investments. The investor's parent company, Bayer AG (Xetra:BAYN), formed a joint venture with CRISPR Therapeutics in December 2015 (see BioCentury, Jan. 4).
The offering price valued CRISPR Therapeutics at $551 million. Earlier this month, the company said it hoped to sell 4.7 million shares in the IPO at $15-$17. At $16, a sale of that many shares in the IPO would have raised $75.2 million. Including the $35 million private placement, CRISPR Therapeutics would have had a post-money valuation of $636 million (see BioCentury Extra, Oct. 7).
CRISPR Therapeutics' most advanced programs are focused on ex vivo approaches for hemoglobinopathies. It is the third CRISPR company to go public, behind Editas Medicine Inc. (NASDAQ:EDIT) and Intellia Therapeutics Inc. (NASDAQ:NTLA).
Nektar Therapeutics (NASDAQ:NKTR) raised $175.5 million through the sale of 13 million shares at $13.50 in a follow-on underwritten by JPMorgan, Piper Jaffray, William Blair, Brean Capital, BTIG, Janney Montgomery Scott, Ladenburg Thalmann and Roth Capital Partners.
The company proposed the offering after market hours on Monday, when it closed at $15.67. Nektar lost $1.59 (10%) to $14.08 Tuesday, then slipped $0.73 to $13.35 on Wednesday.
Nektar plans to start a Phase III trial by YE16 of Onzeald etirinotecan pegol to treat patients with advanced breast cancer and brain metastases. It also expects top-line data in 1Q17 from a Phase III trial of NKTR-181 to treat chronic low back pain.
Ascendis Pharma A/S (NASDAQ:ASND) raised $120 million through the sale of 6.3 million ADSs at $19 in a follow-on underwritten by JPMorgan, BofA Merrill Lynch, Credit Suisse and Wedbush. The company proposed the offering after market hours Tuesday, when it closed at $20.09. Ascendis shed $1.09 to $19 on Wednesday.
Ascendis uses its TransCon technology to create sustained release formulations of products to prolong treatment effects. In August, the company began the Phase III heiGHt trial of TransCon Growth Hormone (ACP-001) to treat growth hormone deficiency.
The path that led to approval of Exondys 51 eteplirsen from Sarepta Therapeutics Inc. (NASDAQ:SRPT) is "not a good model for other development programs," John Jenkins, director of the Office of New Drugs at FDA's Center for Drug Evaluation and Research (CDER), said Tuesday.
In slides presented at a National Organization for Rare Diseases (NORD) meeting, Jenkins said that a "poorly planned and executed development program for a rare disease misuses valuable patient resources and serves to delay obtaining the knowledge required to understand the benefits and risks of a drug to support regulatory review and approval."
Describing lessons learned from Exondys 51 and other recent rare disease programs, Jenkins highlighted two flaws in Sarepta's development program: the use of unvalidated biomarkers and the lack of rigorous blinding of assay interpretation. Jenkins said that in many cases, randomized controlled clinical trials are the fastest way to evaluate a candidate's efficacy.
Jenkins said FDA welcomes engagement of patients and caretakers in helping design drug development programs. "Experience of patients enrolled in trials can be very helpful; discordant results between trial data and patient anecdotes are very hard to reconcile," he added.
Touching on the interactions between patient advocates and FDA staff, Jenkins said that "personal attacks on FDA reviewers" promote "distrust and isolation rather than collaboration."