BioShares Biotechnology Clinical Trials (BBC): $20.82, +$0.04
BioShares Biotechnology Products (BBP): $31.58, -$0.01
U.S. stock index futures tracked gains in global stocks as stronger-than-expected Chinese inflation data eased some concerns about the health of the world’s second-biggest economy. Markets will look for a flurry of economic data scheduled to release later in the day including producer prices, retail sales, business inventories and U-Mich sentiment data. Gold was down as the dollar rose, and the precious metal was on track for weekly gains. Oil was supported by a drop in U.S. fuel inventories and moved further above $52 a barrel.
TxCell SA announced the appointment of Li Zhou, PhD, as Vice President, Cell Engineering. Dr. Zhou brings extensive pharmaceutical experience in antibody engineering and T-cell engineering to strengthen and accelerate the development of TxCell’s ENTrIA CAR-Treg platform.
OncoSec Medical announced financial results for the fourth quarter and fiscal year ended July 31, 2016. For the fourth quarter of fiscal 2016 and the fiscal year ended July 31, 2016, OncoSec reported a net loss of $6.6 million and $26.9 million, or $0.39 per share and $1.63 per share, respectively, compared to a net loss of $6.5 million and $21.2 million, or $0.48 per share and $1.67 per share, respectively, for the same period last year. At July 31, 2016, OncoSec had $28.7 million in cash and cash equivalents, as compared to $32.0 million of cash and cash equivalents at July 31, 2015.
Adverum Biotechnologies provided an update on its development program for ADVM-043 (formerly "ANN-001"), a novel gene therapy for Alpha-1 Antitrypsin (A1AT) Deficiency, a rare genetic disorder that may result in serious respiratory and liver disease. Adverum has decided to upgrade the ADVM-043 manufacturing process by implementing its proprietary baculovirus-based production system and plans to transfer the third-party contract manufacturing for ADVM-043 to a large-scale contract manufacturer. This upgrade is designed to increase the production scale, and comply with industry standards so that the same production process is used from clinical trials through commercial stage. The company now expects to begin enrolment of patients in the Phase I/II clinical trial for ADVM-043 in the fourth quarter of 2017. In other company news, Adverum Biotechnologies announced that former CEO Paul B. Cleveland has been appointed Executive Chairman of the Board and that Amber Salzman, Ph.D., formerly President and CEO, has been appointed CEO.
Egalet announced that the FDA will not meet the previously announced October 14 PDUFA date for ARYMO ER (morphine sulfate).
Bristol-Myers Squibb announced the CHMP of the EMA has recommended the approval of Opdivo (nivolumab) for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL) after autologous stem cell transplant (ASCT) and treatment with brentuximab vedotin. The CHMP recommendation will now be reviewed by the European Commission, which has the authority to approve medicines for the European Union.
AbbVie announced that the CHMP has granted a positive opinion for VENCLYXTO (venetoclax) tablets for the treatment of chronic lymphocytic leukaemia (CLL) in the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor; and for the treatment of CLL in the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemo-immunotherapy and a B-cell receptor pathway inhibitor. The European Commission will review the opinion and make a final decision in late 2016. VENCLYXTO is being developed by AbbVie and Genentech, a member of the Roche Group.
Tetraphase Pharmaceuticals announced dosing of the first patient in IGNITE4, the Company’s Phase III clinical trial evaluating the efficacy and safety of intravenous (IV) eravacycline compared to meropenem in complicated intra-abdominal infections (cIAI). Eravacycline is a novel antibiotic candidate with potent activity against multidrug-resistant (MDR) pathogens, including carbapenem-resistant enterobacteriaceae (CRE), Acinetobacter baumannii, and colistin-resistant bacteria carrying the mcr-1 gene, that is being developed for the treatment of serious and life-threatening bacterial infections.
Intercept Pharmaceuticals announced that the EMA’s CHMP adopted a positive opinion recommending marketing authorization of the company’s MAA for obeticholic acid (OCA), an FXR agonist, for the treatment of primary biliary cholangitis (PBC) conditional to the company providing further data post-approval to confirm benefit.
Cardiome Pharma announced that it has initiated commercial sales operations in Canada. The Canadian sales force will immediately begin detailing AGGRASTAT (tirofiban hydrochloride) to Canadian hospitals but is expected to expand its efforts to include the detailing of BRINAVESS (vernakalant hydrochloride), XYDALBA (dalbavancin hydrochloride) and TREVYENT (treprostinil sodium), if and when marketing approval for each respective drug is granted by Health Canada’s Therapeutic Products Directorate.
BioMarin Pharmaceutical announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK approved continued enrollment into the open-label Phase I/II study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. BioMarin had previously announced that after enrolling the first 9 patients in the study, that dosing of patients had been suspended due to observed increases in alanine aminotransferase (ALT) levels that exceeded a pre specified threshold set by the company. Following study suspension, the company reviewed safety and efficacy data on the 9 patients with the MHRA, and based on its review, the MHRA approved resumption of the study. The agency also approved the company’s proposed amendments to the study, which included eliminating the requirement for prophylactic corticosteroids and increasing potential additional enrollment from up to three additional patients to up to six additional patients. BioMarin intends to resume enrollment in the Phase I/II study before the end of 2016. Based on protocol amendments, three patients will be enrolled at a dose of 4 x 10(13) vg/kg, and an additional three may be enrolled at this dose or the previously tested high dose of 6 x 10(13) vg/kg. In the up to six additional patients, the requirement for prophylactic corticosteroids has been removed and the threshold for starting therapeutic corticosteroids has been increased. Safety and efficacy data from these patients will inform the Phase IIb study planned to begin in the second half of 2017.
Sunesis Pharmaceuticals announced that it has submitted its responses to EMA Day 120 List of Questions issued by the CHMP as part of the centralized review process of the MAA for vosaroxin as a treatment for relapsed/refractory acute myeloid leukemia (AML) in patients aged 60 years and older. Sunesis expects to receive the EMA Day 180 List of Outstanding Issues before year-end.
Eisai announced the presentation of preliminary results of a Phase Ib clinical study (Study 111) of its in-house developed multiple receptor tyrosine kinase inhibitor lenvatinib (marketed in the U.S. under the brand name Lenvima) in combination with the anti-PD-1 immunotherapy pembrolizumab (marketed under the brand name Keytruda, developed by Merck & Co.) in patients with selected solid tumors at the ESMO Congress. Development of this potential combination regimen, which is not approved by the FDA, is being overseen by a Joint Development Committee formed by both companies. According to the results of the Phase Ib part of the study, there were two dose-limiting toxicities (DLT) at the lenvatinib dose level 1 group (24 mg; 1 grade 3 arthralgia and 1 grade 3 fatigue) and no DLTs were reported in the lenvatinib 20 mg plus pembrolizumab 200 mg group. The maximum tolerated dose was confirmed as 20 mg of lenvatinib per day plus 200 mg of pembrolizumab once every three weeks. All patients reported at least one treatment-emergent adverse event (TEAE). Grade 3 or higher TEAEs were observed in 69.2% of patients, and no patients had discontinued treatment due to TEAEs. The most frequently observed adverse events in either the lenvatinib 24 mg plus pembrolizumab 200 mg group or the lenvatinib 20 mg plus pembrolizumab 200 mg group were decreased appetite, diarrhea and fatigue.
On October 13, 2016, Vertex amended and expanded its Research, Development and Commercialization Agreement, dated May 24, 2004, by and between Cystic Fibrosis Foundation Therapeutics Incorporated (“CFFT”) and Vertex Pharmaceuticals Incorporated, in order to update and clarify the terms of the relationship. The Amendment provides for an upfront program award from CFFT to Vertex of $75.0 million and development funding from CFFT to Vertex of up to $6.0 million annually. Pursuant to the Amendment, Vertex agreed to pay royalties ranging from low single digits to mid-single digits on certain compounds first synthesized and/or tested between March 1, 2014 and August 31, 2016. Vertex will continue to pay royalties ranging from single digits to sub-teens on any approved drugs first synthesized and/or tested on or before February 28, 2014. The parties also clarified that net sales on combination products will be allocated equally to each of the active pharmaceutical ingredients in the combination product consistent with the allocation of net sales for ORKAMBI and provided further clarification with respect to the calculation of royalties on products covered by the Collaboration Agreement. Independently, Vertex entered into a data license agreement with the Cystic Fibrosis Foundation pursuant to which Vertex will pay for continuing access to data from the CFF’s patient registry, which Vertex believes will be important for research, development and approval of future CF medicines.
Biocept announced the pricing of an underwritten public offering of 9,100,000 shares of its common stock and warrants to purchase up to an aggregate of 9,100,000 shares of its common stock at a combined offering price of $1.10. The warrants will have a per share exercise price of $1.10, are exercisable immediately and will expire five years from the date of issuance. The gross proceeds to Biocept from this offering are expected to be approximately $10,010,000, before deducting the underwriting discount and estimated offering expenses payable by Biocept. Biocept has granted the underwriters a 30-day option to purchase up to 1,365,000 additional shares of common stock at a purchase price of $1.0331 per share and/or additional warrants to purchase up to 1,365,000 shares of its common stock at a purchase price of $0.0009 per warrant to cover over-allotments, if any. The offering is expected to close on October 19, 2016, subject to customary closing conditions.
UroGen Pharma announced that it has completed an agreement to license worldwide rights to UroGen’s RTGel delivery system technology for use with neurotoxins to Allergan.
Chardan analyst Gbola Amusa upgraded Arbutus Biopharma to “neutral” from “sell,” citing 4Q16/2017 clinical catalysts in the Phase II study of ARB-1467.
Cantor analyst Mara Goldstein downgraded Flex Pharma to “hold” from “buy” and decreased her price target to $10 from $22, citing inconclusive results of the proof of concept study of FLKS-787.
Following bluebird bio’s analyst day, Cantor analyst Elemer Piros downgraded the stock to “sell” from “hold” and decreased his price target to $37 from $42; Leerink analyst Michael Schmidt increased his price target to $80 from $67; BTIG analyst Dane Leone increase his price target to $83 from $72.
Credit Suisse analyst Vamil Divan increased his price target of Esperion Therapeutics to $14 from $10, citing encouraging top-line results from the Phase 2 -035 study and Phase 1 -037 study that help address the questions about the safety of combining bempedoic acid with high-dose statins.