Sangamo Therapeutics presented interim data from the Phase 1/2 EMPOWERS Study evaluating the SB-318 zinc finger nuclease in vivo genome editing product candidate in patients with Mucopolysaccharidosis Type I. These data, along with interim results of the CHAMPIONS Study evaluating SB-913 for MPS II, were presented today at the WORLDSymposium 2019 being held in Orlando, Florida. Sangamo believes data from these two studies provide complementary evidence supportive of a favorable safety profile and of the activity of the ZFN in vivo genome editing technology used in both SB-318 and SB-913. “The results so far suggest a dose-dependent increase in leukocyte IDUA enzyme activity,” said Dr. Paul Harmatz, a professor at UCSF Benioff Children’s Hospital Oakland and a lead investigator on the study. “Leukocytes are an easily accessible target tissue for IDUA and therefore provide an estimate of tissue enzyme activity for patients with MPS I. Whether these observed increases will translate into clinical benefit from SB-318 is yet to be determined.” The primary objective of the EMPOWERS Study is to determine the safety and tolerability of SB-318, and secondary objectives include evaluation of change from baseline in IDUA activity and urine GAG levels. Biochemical measurements of urinary GAGs, as well as plasma and leukocyte IDUA activity, are assessed at screening and baseline visits, and every two to four weeks during the initial phase of the trial. Patients with mild MPS I receiving weekly ERT were enrolled in the study. The results suggest a dose-dependent increase in leukocyte IDUA activity, with activity levels rising above baseline and in the normal range. Plasma IDUA activity was unchanged from baseline in all three patients. Baseline urine GAG measurements for the three patients in the EMPOWERS Study were in a range considered to be at or slightly above normal. In the limited duration data set available at the time of the WORLDSymposium presentation, urine GAG measurements show no meaningful change.
https://thefly.com/landingPageNews.php?id=2861097
Thursday, February 7, 2019
Sanofi Cuts 38 R&D Programs to Refocus on Cancer, Immunology, Rare Diseases
What a difference a year makes. In January 2018, Paris-based Sanofi marked the start of the year by acquiring Waltham, Mass.-based Bioverativ for $11.6 billion and Ghent, Belgium-based Ablynx for $4.8 billion.
A year later, at its fourth-quarter and annual report today, Sanofi announced it has killed 38 R&D programs, prioritizing oncology, immunology, rare disease and rare blood disorders. Thirteen of those were development programs, the remaining 25 research-stage. In September, the company indicated it was on a cost-cutting program, planning to save 1.5 billion euros this year.
“In the fourth quarter, we continued the momentum of the previous quarter and we delivered 5 percent full-year business EPS growth, at the high end of our guidance,” stated Olivier Brandicourt, Sanofi’s chief executive officer. “In 2018, we executed on important launches including Dupixent, Libtayo and Cablivi, as the headwinds from our U.S. LoEs (loss of exclusivity) began to moderate. Additionally, the acquisitions of Bioverativ and Ablynx provided the foundation to build a leading Rare Blood Disorder franchise and to enhance our biologic discovery capabilities. As we enter 2019, our focus remains on delivering our business priorities and transforming Sanofi to address the evolving business dynamics facing our industry.”
Just yesterday, the U.S. Food and Drug Administration (FDA) approved Cablivi (caplacizumab-yhdp) in combination with plasma exchange and immunosuppression to treat acquired thrombotic thrombocytopenic purpura (aTTP) in adults. It is the first FDA approved drug for aTTP. Cablivi was a drug Sanofi picked up when it acquired Ablynx.
Of the programs it is dumping, the most prominent is a mid-phase drug for respiratory syncytial virus (RSV), ALX-0171. It is also a program it picked up with Ablynx.
The company has also ended a collaboration with MyoKardia, which is behind several of the abandoned programs, and last year the company ended a deal with Immune Design that was related to the TLR4 peanut allergy program.
Total net sales for the quarter were 8.997 billion euros, with full-year sales of 34.463 billion euros. The largest area of growth for the quarter and the year was Specialty Care, with sales of 2.328 billion euros, an increase of 35.2 percent, and 8.269 billion euros for the year, an improvement of 29 percent. The worst area, however, was Generics. For the quarter, Generics sales were 270 million euros, a decrease of 33.8 percent. For the year, Generics sales were 1.490 billion euros, a decrease of 9.8 percent.
The hottest drugs in its Pharmaceutical franchise were Cerdelga, for Gaucher’s disease, which brought in 44 million euros for the quarter, an increase of 33.3 percent, and 159 million euros for the year, an increase of 31 percent. This was followed by Aldurazyme, for Hurler and Hurler-Scheie diseases, which brought in 54 million euros for the quarter, up 16.7 percent, and 206 million euros for the year, an increase of 6.7 percent.
The company’s multiple sclerosis franchise, the drugs Aubagio and Lemtrada, brought in 542 million euros for the quarter and 2.049 billion euros for the year. The company’s Diabetes franchise, like much of the diabetes market, was down. Total Diabetes sales for the quarter was 1.375 billion euros, down 10.5 percent, and 5.472 billion euros for the year, down 10.4 percent.
The company also updated its R&D strategy, increasing its focus on Specialty Care areas, oncology, immunology, rare disease and rare blood disease, while also remaining committed to vaccines. Sanofi noted that, “Since 2017, the number of R&D programs in these areas has increased significantly, and they now represent over 90 percent of Sanofi’s clinical portfolio.”
Aside from the 38 programs it cut, the company says it potentially will be able to submit nine new drugs and 25 additional indications to regulators over 2019 to 2022.
Orasure hit on Q4 report
Orasure (NASDAQ:OSUR) Q4 results ($M): Revenues: 50.2 (-3.5%); Molecular Collection Systems: 25.4 (-14.8%); Infectious Disease Testing: 13.6 (-3.5%); OraQuick: 13.0 (-5.1%).
Net income: 10.3 (+40.8%); EPS: 0.16 (+33.3%).
Q1 guidance: Revenues: $29.0M – 30.5M.
Previously: OraSure Technologies beats by $0.05, beats on revenue (Feb. 6)
LivaNova risk/reward favorable into final Medicare decision, says Piper Jaffray
Piper Jaffray analyst Matt O’Brien is previewing LivaNova’s Medicare final decision on the coverage of Vagus Nerve Stimulation for treatment-resistant depression expected by end of next week. The key swing factor will be the response versus remission endpoint, O’Brien tells investors in a research note. The analyst believes that with little in shares for depression, if Centers for Medicare and Medicaid Services agrees to a response endpoint, shares of LivaNova “will lift significantly, as previous data has shown VNS delivers this result quickly.” If the final decision includes a remission endpoint, O’Brien anticipates disappointment and “potentially another modest move lower for shares.” However, in order to demonstrate statistical significance, the size of the trial will need to be quite large and could provide significant, high-margin revenue for LivaNova, he adds. For holders of the stock, he believes the risk/reward is favorable. For investors looking at the name, the analyst reiterates an Overweight rating on LivaNova “either way after the final decision is released.”
Sanofi sees FY19 business EPS up 3%-5% at current exchange rates
Sanofi expects 2019 Business EPS to grow between 3% and 5% at CER, barring unforeseen major adverse events. Applying average January 2019 exchange rates, the positive currency impact on 2019 Business EPS is estimated to be between 1% to 2%.
Mizuho likes Biogen’s odds in patent case against Mylan
Shares of Biogen (BIIB) are trading down about 3% after Mylan’s (MYL) inter partes review on key multiple sclerosis drug Tecfidera got “instituted,” Mizuho analyst Salim Syed tells investors in a research note. The timing was as expected, and most biotech IPRs in general get instituted, explains the analyst. He says that while historical biotech IPR data suggests Biogen has a 31% chance of losing, the company’s odds are “materially less” for two reasons. One, Tecfidera intellectual property is already “battle-tested,” and two, there are 20 claims being disputed in this IPR and Mylan needs to win on all of them while Biogen needs to win on only one claim to be successful, Syed writes. He has a Buy rating on Biogen with a $427 price target.
Mednax sees Q1 adjusted EPS 67c-75c, consensus 86c
Q1 adjusted EPS view excludes 14c per diluted share of estimated amortization expense, 9c per diluted share of estimated stock-based compensation expense and 3c per diluted share of transformation related expenses. Additionally, for the Q1, MEDNAX expects that Adjusted EBITDA will be between $108M-$118M, compared to the prior-year period Adjusted EBITDA of $133.6M. Q1 MEDNAX has one fewer weekday compared to the previous Q1, which is expected to impact Adjusted EBITDA unfavorably by approximately $4M. In addition, Q1 Adjusted EBITDA includes approximately $5M in contribution from the previously mentioned anesthesiology contract that was not renewed subsequent to that period. Factors include the incurrence of a disproportionate share of the annual expenses associated with Social Security payroll taxes and 401 match. In 2018, the Company’s total expenses related to these items were approximately $167 million, of which $67M, or 40% was incurred in the Q1. Consequently, MEDNAX’s Adjusted EBITDA and EPS for the Q1 of FY18 reflected expenses related to these items that were approximately $25M, or 21c, higher than if they were incurred ratably throughout the year. The Company’s outlook for the first quarter of 2019 reflects a similar impact due to the seasonality of these expense items.
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