Mylan and Theravance Biopharma’s COPD treatment gets FDA approval

Theravance Biopharma Inc and partner Mylan NV on Friday won U.S. regulatory approval for their treatment for a chronic lung condition that causes breathing-related problems.

The treatment, Yupelri,  https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/210598s000lbl.pdf,   is once-daily inhalable solution to be used by patients of chronic obstructive pulmonary disease (COPD), a lung disease characterized by wheezing or chronic cough.

https://kfgo.com/news/articles/2018/nov/09/mylan-and-theravance-biopharmas-copd-treatment-gets-fda-approval/

Arrowhead presents clinical data from Phase 1 study of ARO-AAT

Arrowhead Pharmaceuticals announced clinical data from a Phase 1 study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency, will be presented in a late-breaking poster at The Liver Meeting, the Annual Meeting of the American Association for the Study of Liver Disease, being held in San Francisco. In the AROAAT1001 study, 45 normal healthy volunteers received a single dose of ARO-AAT, three monthly doses of ARO-AAT or placebo. Key data presented include the following: ARO-AAT at single- and multiple-doses produced robust and consistent reductions in serum AAT levels. Single-doses of 200 and 300 mg resulted in greater than 91% serum AAT reduction, with 3 of 4 subjects having concentrations below the level of quantitation. In 200 and 300 mg single-dose cohorts, an average serum AAT reduction of greater than 90% was sustained for 6 weeks. In the multiple-dose cohorts of 200 and 300 mg, for subjects receiving all 3 doses, an average of greater than 90% reduction in serum AAT was sustained for longer than 14 weeks. The maximum NADIR reduction is 94%. Monthly serum AAT follow up is ongoing with 9 of 10 subjects at BLQ in the multiple-dose cohorts, including 100% of subjects from the 300 mg cohort. Duration of response indicates that quarterly or less frequent dosing appears feasible. ARO-AAT has been well tolerated at all doses tested given three times every 28 days. The most common adverse events were upper respiratory tract infection and headache.
https://thefly.com/landingPageNews.php?id=2821689

Celyad presents update on CYAD-01 solid tumor clinical program

Celyad announced updated clinical results for the CYAD-01 program in solid tumors as well as translational research data presented at the Society for Immunotherapy of Cancer, or SITC, 33rd Annual Meeting. In the THINK Phase 1 dose-escalation trial, 14 patients with relapsed/refractory disease were enrolled in the trial, evaluating CYAD-01 without preconditioning chemotherapy at three different dose levels of one cycle of three administrations with two-week intervals. Overall four patients experienced confirmed disease stabilization, three mCRC patients and one patient with ovarian cancer, according to RECIST 1.1 criteria. As a monotherapy treatment, CYAD-01 was well tolerated. The peak level of peripheral CYAD-01 cells detected seem to correlate with the dose level and clinical response. In the SHRINK Phase 1 open-label, dose-escalation trial, patients will receive six cycles of FOLFOX chemotherapy every two weeks and three administrations of CYAD-01 every two weeks 48 hours after the end of chemotherapy at cycles two, three and four. To date, enrollment of dose level one has been completed with three metastatic treatment-naive patients. All patients have undergone resection without delays in surgery. Initial activity results assessed by pathological response criteria showed all three patients achieved an objective clinical response, including one patient with a pCR and two patients with pPR. Concurrent treatment of CYAD-01 with FOLFOX chemotherapy appears to be well tolerated, with no occurrence of serious AEs nor increase of treatment-related AEs rate. In addition, the expansion of peripheral CYAD-01 cells with a concurrent administration of FOLFOX chemotherapy is similar to the one observed with the standalone CYAD-01. Full data from the SHRINK Phase 1 trial are expected in mid-2019. Frederic Lehmann, VP of Clinical Development & Medical Affairs at Celyad, commented, “Solid tumors remain the greatest current challenge for any T cell therapy. One of the major hurdles is the lack of suitable targets, and in our perspective, NKG2D ligands that are targeted by CYAD-01 represent an attractive family of targets on solid tumors that may be exploited by our clinical candidates. I am encouraged that to date CYAD-01 is well tolerated as a monotherapy for the treatment of mCRC, while preliminary observations of clinical activity in the form of disease stabilization imply that there is potential for the approach. Furthermore, the initial findings of clinical activity reported from the initial dose level of CYAD-01 when administered concurrently with standard-of-care chemotherapy in the SHRINK trial are encouraging and provide support for this view.”
https://thefly.com/landingPageNews.php?id=2821709

BioLife Q3 beat propels shares, up 18%

Thinly traded micro cap BioLife Solutions (BLFS +17.5%) is up on average volume following its Q3 report. Highlights:

Revenue was up 79% to $5.3M, product sales were up 80% to $2.9M.

15 new customers closed in the regenerative medicine segment.

Net income jumped almost four-fold to $1.2M.

2018 guidance: Biopreservation media sales: $19M – 20M (unch); positive GAAP net income.

Previously: BioLife Solutions beats by $0.01, beats on revenue (Nov. 8)

https://seekingalpha.com/news/3408180-biolife-q3-beat-propels-shares-18-percent

FDA extends review of Samsung Bioepis’ trastuzumab biosimilar

A Samsung Bioepis Co. Ltd. spokesperson told BioCentury that FDA extended by three months its review of a BLA for SB3, a biosimilar of cancer drug Herceptin trastuzumab from Roche (SIX:ROG; OTCQX:RHHBY) and its Genentech Inc. unit. The company did not disclose details regarding the delay.

FDA accepted the BLA in December 2017, and the company now expects a decision in January. The biosimilar is approved in the EU as Ontruzant (see “EMA Approves First Herceptin Biosimilar”).

Trastuzumab is a humanized mAb against HER2.

The setback is the second this month for a biosimilar in the U.S. The Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) said Nov. 2 that it would no longer seek U.S.approval of GP2013, its biosimilar of cancer and autoimmune drug Rituxan/MabThera rituximab from Roche (see “Sandoz Gives Up on Rituxan Biosimilar in U.S.”).

Samsung Bioepis is a JV between Biogen Inc. (NASDAQ:BIIB) and Samsung BioLogics Co. Ltd. (KOSDAQ:20740).

https://www.marketscreener.com/BIOGEN-4853/news/FDA-extends-review-of-Samsung-Bioepis-trastuzumab-biosimilar-27580503/

EU regulators to clear $62 billion Takeda, Shire deal

Regulators are set to approve Japanese drugmaker Takeda Pharmaceutical’s $62-billion (47.70 billion pounds) offer for London peer Shire on condition that Shire sells a drug it has in development, a person familiar with the matter said on Friday.

Last month, Takeda offered to divest Shire’s pipeline compound SHP647 along with some associated rights after the European Commission voiced concerns about the overlap with its own drug for inflammatory bowel disease.

https://www.marketscreener.com/TAKEDA-PHARMACEUTICAL-CO-6491073/news/Exclusive-EU-regulators-to-clear-62-billion-Takeda-Shire-deal-source-27580494/

CVS Health price target raised to $80 from $70 at Loop Capital

Loop Capital analyst Andrew Wolf raised his price target on CVS Health shares to $80 from $70, citing improved sales results from CVS’s core business units in Q3 and the company’s maintenance of 2018 guidance. He also notes that the sector rotation into more defensive stocks has boosted the valuations of most of the other large-cap names in the group. However, Wolf keeps a Hold rating on CVS shares, noting that while its Q3 adjusted EPS of $1.73 topped consensus, it missed his own $1.78 forecast.
https://thefly.com/landingPageNews.php?id=2821657