Celgene Corp. (NASDAQ:CELG) added $5.64 (5.9%) to $100.40 on Thursday after reporting adjusted net income of $806 million in 3Q14, up 20% from $669 million in 3Q13, and upped its revenue and EPS guidance for the year.
In the cancer portfolio, global sales of Revlimid lenalidomide were $1.3 billion in 3Q14, up 19% from $1 billion in 3Q13. On the conference call, the company noted near-term events that would drive the thalidomide analog's trajectory, including a Feb. 22, 2015, PDUFA date for use in newly diagnosed multiple myeloma and an opinion from EMA's CHMP for the indication in 1H15.
At the American Society of Hematology meeting in December, Celgene will present Phase III data for Revlimid to treat low-risk/intermediate-1 transfusion dependent myelodysplastic syndrome (MDS) without a 5q chromosomal deletion. On the call, President of Global Hematology and Oncology Jacqualyn Fouse said Celgene will "pursue global regulatory submissions for this indication, which would significantly increase the size of the MDS patient population for which Revlimid would be indicated."
The company also is working toward adding to its MDS porfolio with partner Acceleron Pharma Inc. (NASDAQ:XLRN), saying it would report Phase II data for luspatercept (ACE-536) in MDS and beta-thalessemia and sotatercept in MDS at ASH. Acceleron added $2.99 (10%) to $32.03 on Thursday.
Celgene also highlighted the prospects for its budding inflammation and immunology franchise. The company said it expects a CHMP opinion for Otezla apremilast for psoriatic arthritis and psoriasis by year end. The oral phosphodiesterase-4 (PDE-4) inhibitor was approved in the U.S. in March to treat active psoriatic arthritis and in September for moderate to severe plaque psoriasis. The drug posted $18 million in its first full quarter of sales in 3Q.
Celgene also said it expects to begin Phase III testing by year end of mongersen (GED-0301) to treat Crohn's disease. The company announced positive data from a Phase II trial earlier this week (see BioCentury, Oct. 20).
Celgene paid $710 million up front in April to obtain exclusive, worldwide rights to the oral antisense oligonucleotide targeting SMAD family member 7 (MADH7; SMAD7) from Nogra Pharma Ltd. (Dublin, Ireland).
The company increased its 2014 revenue guidance to above $7.6 billion, from "approximately" $7.6 billion in prior guidance. Celgene also increased its non-GAAP diluted EPS guidance for 2014 to $3.65-$3.70 from $3.60-$3.65.
Celgene Corp. (NASDAQ:CELG) paid Sutro Biopharma Inc. (South San Francisco, Calif.) $95 million up front in a deal to develop cancer immunotherapies using Sutro's cell-free protein synthesis technology. Celgene also enlarged its equity stake in Sutro to 15% and obtained an option to acquire the company at an unspecified time.
Celgene may pay Sutro up to an additional $90 million during the collaboration's initial research term, based on milestones. Sutro is eligible for more than $1 billion in total milestones, plus royalties. Celgene has the option to extend the collaboration beyond initial terms in exchange for additional payment.
The companies said they will investigate both novel targets and established ones, including programmed cell death 1 (PDCD1) (PD-1) (CD279) and programmed cell death 1 ligand 1 (PD-L1) (B7-H1) (CD274), and apply Sutro's Xpress CF and Xpress CF+ technologies to develop multi-specific antibodies and antibody-drug conjugates (ADC). Sutro will be responsible for discovery, preclinical development and manufacturing.
In December 2012, Celgene and Sutro agreed to jointly develop bi-specific antibodies and ADCs against two undisclosed targets (see BioCentury Extra, Dec. 18, 2012).
In September, the EMD Serono division of Merck KGaA (Xetra:MRK) partnered with Sutro to use Xpress CF and Xpress CF+ to discover and develop ADCs against undisclosed cancer targets (see BioCentury Extra, Sept. 17).
NPS Pharmaceuticals Inc. (NASDAQ:NPSP) said FDA has delayed the PDUFA date for Natpara from Oct. 24 to Jan. 24, 2015, to review a major amendment to the BLA. NPS said the agency has also asked for a REMS and will likely require a post-approval study.
The announcement came after market close; NPS shares were up $1.42 to $29.08 on Thursday.
In September, FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 8-5 to recommend approval of the recombinant human parathyroid hormone 1-84 (PTH) to treat hypoparathyroidism. But in their briefing documents, the agency's reviewers voiced concerns about the compound's safety, including risks of hypocalcemia and hypercalcemia.
They also noted that osteosarcoma rates in Natpara-treated rats were similar to those seen with Forteo teriparatide, a PTH fragment from Eli Lilly and Co. (NYSE:LLY) that is approved for osteoporosis. Forteo has a black-box warning about osteosarcoma risk and is not indicated for use beyond two years.
Johnson & Johnson (NYSE:JNJ) announced plans to open the fourth incubator in its J-Labs network, formerly known as Janssen Labs, within the new Innovation Institute at Texas Medical Center in Houston. J-Labs @ TMC, a 30,000-square foot facility capable of housing up to 50 life science start-ups, is slated to open in 4Q15.
Melinda Richter, head of J-Labs, said the incubator will provide education, operations and business support and will connect local entrepreneurs with investors. She declinded to disclose how much funding J&J will contribute to the incubator.
J-Labs already has incubators in San Diego, San Francisco and Boston. Another Bay Area location is due to open in South San Francisco in early 2015.
Richter said TMC lacked a commercialization platform and that J-Labs' presence could offer local researchers who lack business experience a pathway from research project to product.
J&J does not take equity stakes in the companies incubated at J-Labs. Richter noted, however, that the company has signed deals with 12 of the 70 start-ups it has incubated so far.
Three days after an FDA committee backed the approval of Cosentyx secukinumab from Novartis AG (NYSE:NVS; SIX:NOVN) for moderate to severe plaque psoriasis, the compound met its primary and key secondary endpoints in two Phase III trials in ankylosing spondylitis, another indication dominated by anti-TNF therapies.
Novartis spokesperson Julie Masow said up to 40% of ankylosing spondylitis patients are unresponsive to anti-TNF drugs or are intolerant of treatment. She said the human IgG1 mAb targeting IL-17A is the first compound in its class to show efficacy in Phase III trials to treat ankylosing spondylitis.
The company did not release specific data from the Phase III trials, but Masow said full trial results will be presented at a medical congress this year. The studies' primary endpoint was the Assessment of Spondyloarthritis International Society criterion (ASAS 20).
Secukinumab is under review by FDA and EMA to treat moderate to severe plaque psoriasis with a decision expected by early 2015. Novartis plans to file joint regulatory applications for secukinumab in ankylosing spondylitis and psoriatic arthritis in 2015.
Novartis gained $1.04 to $89.24 on Thursday.
Rare disease specialists Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) and BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) both raised 2014 guidance in earnings reports on Thursday.
Alexion shares added $12.65 to $185.92 after the company raised its revenue guidance from $2.18-$2.20 billion to $2.22-$2.25 billion and its non-GAAP EPS guidance from $4.95-$5.05 to $5.15-$5.20.
The company, which recorded $555.1 million in net sales in 3Q14 from its only product, Soliris eculizumab for rare blood disorders, also said it will complete its rolling BLA submission to FDA for asfotase alfa (ENB-0040) by year end. The candidate has Breakthrough Therapy and Orphan Drug designations to treat hypophosphatasia.
BioMarin raised its revenue guidance to $700-$710 million from $680-$700 million. The company's revenue, almost entirely from orphan product sales, was up 29% to $176.8 million in 3Q14 compared to $136.9 million in 3Q13.
BioMarin noted GAAP net income of $7.4 million vs. a non-GAAP net loss of $22.9 million was largely due to the one-time sale in July of its rare pediatric disease Priority Review Voucher for $67.5 million to Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) and Sanofi (Euronext:SAN; NYSE:SNY).
BioMarin announced its results after market close; it was up $0.69 to $75.01 on Thursday.
Analgesic developer Quartet Medicine (Cambridge, Mass.) raised $17 million in a series A round led by Atlas Venture. Novartis Venture Funds, Pfizer Venture Investment and Partners Innovation Fund also participated.
Quartet is developing inhibitors of GTP cyclohydrolase 1 (GCH1) or sepiapterin reductase (SPR), which modulate tetrahydrobiopterin (THB; BH4) synthesis, for chronic neuropathic pain indications. Quartet expects to have a lead candidate in preclinical development next year.
Novartis Venture Funds' Henry Skinner joined Quartet's board. Atlas made a seed investment in the biotech last year; partner Bruce Booth is its chairman.
Probiodrug AG (Euronext:PBD) raised EUR 22.5 million ($28.7 million) in an IPO through the sale of 1.5 million shares at EUR 15.25 on Euronext Amsterdam, giving the company a market cap of EUR 102.4 million ($130.6 million). Probiodrug priced the offering at the low end of its proposed range of EUR 15.25-EUR 19. Kempen and Petercam were underwriters, and Close Brothers Seydler acted as selling agent. Trading of the shares will start on Oct. 27.
The company plans to begin a Phase II trial of lead candidate PQ912, a glutaminyl cyclase (QC) inhibitor, to treat Alzheimer's disease in 1Q15, with data expected in mid-2016. Probiodrug said pyroglutamated beta amyloid, which is produced from QC, has been correlated with cognitive decline in AD.
S1 Biopharma Inc. (New York, N.Y.) amended its IPO on NASDAQ and plans to sell 2.8 million shares at $12-$14. At $13, the company would raise $35.8 million and be valued at $118.6 million. S1 Biopharma filed to raise up to $40.3 million on Oct. 1 underwritten by MLV.
S1 plans to begin Phase IIb testing next year of lead candidate Lorexys, an oral fixed-dose enteric formulation of bupropion and trazodone to treat hypoactive sexual desire disorder (HSDD) in women.
Erytech Pharma S.A. (Euronext:ERYP) raised EUR 30 million ($38.2 million) through the sale of 1.2 million shares at EUR 24.50 in a follow-on. U.S. investors provided 68% of the funds.
Erytech will use the money to further develop lead compound Graspa, including an ongoing study to treat acute lymphoblastic leukemia (ALL) and an additional planned study in the U.S. Erytech also plans to run two new Phase II or Phase III studies in oncology or hemato-oncology.
Earlier this month, the L-asparaginase encapsulated in red blood cells met the co-primary endpoints in a European Phase III trial to treat children and adults with relapsed or refractory ALL. The company plans to submit an MAA to EMA in 1H15.
Bryan Garnier served as a global coordinator and LifeSci Capital served as a placement agent in the U.S.
Erytech raised EUR 16.7 million in an IPO last year through the sale of 14 million shares at EUR 11.60.
Erytech was down EUR 0.66 to EUR 26.37 on Thursday.
The World Health Organization is calling for a strict timeline for public disclosure of clinical trial results, citing analysis showing that "a significant proportion of healthcare research remains unpublished," and its concerns that "trial results which may be viewed as 'negative' are less likely to be submitted or accepted" for publication or otherwise made public.
WHO requested public comment on its new "Statement on the Public Disclosure of Clinical Trial Results," which specifies that study results be reported in both of two settings within 30 months after a study is completed.
First, it calls for the main findings to be submitted for publication in a peer reviewed journal no later than 18 months after a study is completed and published in an open access setting.
Second, it advocates "key outcomes" of a study also be posted in a primary clinical trial registry, with the caveat that key outcomes do not include primary data disclosure.
Comments are due by Nov. 15.
Tobira Therapeutics Inc. has begun a Phase IIb trial of cenicriviroc to treat non-alcoholic steatohepatitis. The Oct. 21 BioCentury Extra misstated the status of the trial.
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