BioShares Biotechnology Clinical Trials (BBC): $19.83, -$0.01, +7.7% YTD

BioShares Biotechnology Products (BBP): $35.50, +$0.24, +8.4% YTD

 

 

MARKET COMMENTARY

 

Wall Street looked set to open lower and the U.S. dollar dipped against a basket of major currencies after the Federal Reserve gave no clear sign on when it will next raise interest rates. With all the political uncertainties about, the big central banks appear to be lying low – or at least trying not to add to the volatility. Shares of Amazon.com, Visa, Amgen and Motorola Solutions will be in focus as they report quarterly results later in the day. Challenger layoffs, initial claims, labor costs and productivity data are on the economic calendar. European markets were mixed, while Asian shares mostly closed lower. Oil prices stabilized after a sharp rise in U.S. crude stockpiles. Gold edged higher.

 

 

MARKET HIGHLIGHTS

 

Bioblast Pharma announced new clinical and development updates related to its proprietary intravenous (IV) form of trehalose 90 mg/mL solution, which is being evaluated as a potential therapeutic, initially for oculopharyngeal muscular dystrophy.  Bioblast management will provide a comprehensive overview of the Company’s ongoing development work with trehalose, including OPMD activities in clinical and preclinical development, manufacturing and pre-commercial market planning, at a KOL meeting today, Thursday, February 2nd at 12pm ET.  A webcast of the event is available here:  http://lifesci.rampard.com/20170202/reg.jsp.

 

CymaBay Therapeutics announced the pricing of its previously announced underwritten public offering of 5,181,348 shares of its common stock at a public offering price of $1.93 per share, before underwriting discounts and commissions. All of the shares of common stock are being offered by CymaBay. In addition, CymaBay has granted the underwriters a 30-day option to purchase up to an additional 777,202 shares of common stock at the public offering price less underwriting discounts and commissions solely to cover over-allotments, if any. CymaBay anticipates using the net proceeds from the offering to fund ongoing development of seladelpar and for working capital and general corporate purposes.  The offering is expected to close on February 7, 2017, subject to the satisfaction of customary closing conditions.  JonesTrading Institutional Services LLC is acting as the sole book-running manager for the offering, and LifeSci Capital LLC is acting as the lead manager.

 

Gemphire Therapeutics will participate in two investor conferences in New York City during the month of February.  The Canaccord Genuity Rare Disease & Biopharma 1×1 Day on Tuesday, February 7th, and the RBC Capital Markets Global Healthcare Conference on Thursday, February 23rd.

 

MediciNova will attend the Mizuho Healthcare Conference to be held at the Lotte New York Palace in New York City on March 2-3, 2017.

 

Prothena Corporation plc will report its fourth quarter and full year 2016 financial results on Tuesday, February 14th, after the close of the U.S. financial markets.

 

Kamada will release financial results for the fiscal year ended December 31, 2016 prior to the open of the U.S. financial markets on Monday, February 6, 2017.

 

Dow Jones reported that Merck posted a revenue decline in its latest quarter as generic competition hurt results, though the top line was still better than expectations. For 2017, Merck projects per-share adjusted earnings between $3.72 and $3.87 on revenue between $38.6 billion and $40.1 billion. Analysts polled by Thomson Reuters expect annual earnings per share of $3.85 on revenue of $40.04 billion. In all for the quarter, the company posted a profit of $1.18 billion, or 42 cents a share, up from $976 million, or 35 cents a share, a year prior. Excluding restructuring and acquisition-related costs and other items, per-share earnings fell to 89 cents from 93 cents. Profit rose as the company cut costs and from income tax differences in the quarters. Sales fell 1% to $10.12 billion. Analysts polled by Thomson Reuters had forecast per-share earnings of 89 cents a share on revenue of $10.22 billion.

 

Dow Jones reported that AstraZeneca said net profit increased in the fourth quarter as a rise in proceeds from licensing deals offset sharply lower sales of the company’s top-selling cholesterol pill, after several cheap generic versions launched earlier this year. The company said net profit more than doubled to $1.8 billion in the three months to Dec. 31, from $808 million in the same period a year earlier. Analysts expected net income of $616 million. Revenue slipped 13% to $5.6 billion, from $6.4 billion a year ago, meeting analysts’ expectations. The boost in net income was largely thanks to a rise in proceeds from a string of licensing deals struck by the company to offload drugs that fall outside its core areas of focus, in order to raise funds for developing new medicines. The company notched $1.1 billion from these deals in the fourth quarter, compared with $471 million the same period a year earlier.

 

Dow Jones reported that Novo Nordisk the company now sees 2017 sales in a range between a decline of 1% to growth of 4% while operating profit will be in a range between a decline of 2% and a rise of 3% in local currency terms. It previously saw low single-digit growth in sales and flat to low single-digit growth in operating profit. The company reported a 5% rise in fourth-quarter net profit to DKK8.7 billion ($1.26 billion), missing analysts’ expectations for DKK8.92 billion according to a FactSet poll. Sales were DKK29.57 billion.

 

After yesterday’s close, Qiagen reported 4Q16 non-GAAP EPS of $0.15, which compares to $0.32 for the same period a year ago. The company missed the non-GAAP EPS mean estimate of $0.34. Total revenue for 4Q16 was $366.5 million, which compares to $348.5 million for the same period a year ago. This compares to a mean estimate of $370.7 million.

 

TheStreet.com reported that Stemline‘s SL-401 has demonstrated robust overall tumor response rates in its clinical trial but the drug is also now tied to three patient deaths from capillary leak syndrome.

 

Aviragen Therapeutics announced top-line data from its double-blind, placebo-controlled Phase IIa study of BTA585 in adults challenged intranasally with respiratory syncytial virus (RSV). The data indicate there was not a significant reduction in the primary endpoint, which was viral load. The overall safety profile of BTA585 was favorable and consistent across treatment groups.

 

Swedish Orphan Biovitrum and Bioverativ announce that results from the Kids B-LONG Phase III clinical trial, which studied Alprolix  [eftrenonacog alfa, Coagulation Factor IX (Recombinant), Fc Fusion Protein] in previously-treated children with severe haemophilia B, were published in The Lancet Haematology. The primary outcome measure of the trial was development of inhibitors, and no patients treated with Alprolix in the study developed inhibitors. Treatment was generally well tolerated and resulted in low bleeding rates in participants, most of whom remained on once-weekly dosing during the study. The manuscript, entitled "Recombinant Factor IX Fc Fusion Protein in Children with Haemophilia B (Kids B-LONG): Results from a Multicentre, Non-Randomised Phase III Study," was published in the February 2017 issue of The Lancet Haematology.

 

BioMarin Pharmaceutical announced that the EMA has granted access to its Priority Medicines (PRIME) regulatory initiative for the company’s investigational gene therapy treatment for severe hemophilia A, BMN 270.  To be accepted for PRIME, an investigational therapy has to show its potential to benefit patients with unmet medical needs based on early clinical data.

 

AbbVie announced that the FDA has accepted its NDA and granted priority review for its investigational, pan-genotypic regimen of glecaprevir/pibrentasvir (G/P), being evaluated for the treatment of all major genotypes (GT1-6) of chronic hepatitis C virus (HCV). The FDA grants priority review designation to medicines that it determines have the potential to provide significant improvements in the safety and effectiveness of the treatment of a serious disease. The NDA is supported by data from eight registrational studies in AbbVie’s G/P clinical development program, which evaluated more than 2,300 patients in 27 countries across all major HCV genotypes and for special populations. Glecaprevir (GLE) was discovered during the ongoing collaboration between AbbVie and Enanta Pharmaceuticals for HCV protease inhibitors and regimens that include protease inhibitors.

 

Abbott announced that the FDA has authorized its molecular test, the Abbott RealTime ZIKA test, to detect Zika virus in whole blood (when collected alongside a patient-matched serum or plasma sample) for emergency use. This is the first molecular test made by a commercial manufacturer authorized to detect Zika in whole blood samples, which is significant since recent research suggests Zika virus can be detected in whole blood for a longer period of time (up to two months) and at higher levels versus testing with serum and urine sample types.

 

AMAG Pharmaceuticals announced results from its definitive pharmacokinetic (PK) study designed to demonstrate comparable bioavailability of the subcutaneous auto-injector product and the current intramuscular (IM) injection form of Makena in approximately 120 healthy post-menopausal women. Makena administered subcutaneously demonstrated bioequivalence to the IM injection on area under the curve (AUC(0-to-inf) 2,386 ng/mL compared to 2,086 ng/mL), with the 90 percent confidence interval for the ratio of AUC (105.17 to 124.39) falling within the 80 to 125 percent range, which the FDA uses to define bioequivalence. The mean maximum or peak plasma concentration (Cmax) for Makena subcutaneous was slightly higher than for the IM (7.3 ng/mL compared to 6.3 ng/mL), with the 90 percent confidence interval for the ratio of Cmax (96.6 to 138.7 percent) falling outside of the bioequivalence range of 80 to 125 percent.

 

Teva Pharmaceutical Industries announced that it has filed an ANDA with the FDA seeking approval to market the generic version of Novo Nordisk‘s Victoza (liraglutide) injection, in the U.S. Victoza is indicated as an adjunct to diet and exercise to improve glycemic control in adults with Type II diabetes mellitus. Based on available information, Teva believes it is a "first applicant" to file an ANDA for the generic version of Victoza; should its ANDA be approved, Teva may be entitled to 180 days of generic market exclusivity.

 

CytomX Therapeutics announced the treatment of the first patient in the PROCLAIM (Probody Clinical Assessment In Man) CX-072 study, a Phase I/II clinical trial evaluating CX-072, a PD-L1-targeting Probody therapeutic, as monotherapy and in combination with Yervoy (ipilimumab) or Zelboraf (vemurafenib) in patients with all types of cancers.

 

Cytokinetics and Royalty Pharma announced that Cytokinetics has agreed to sell to Royalty Pharma a portion of the potential royalty due to Cytokinetics from Amgen on worldwide sales of omecamtiv mecarbil. Cytokinetics has also agreed to exercise its option to co-invest with Amgen in the Phase III development program of omecamtiv mecarbil in exchange for increased royalties from Amgen on worldwide sales of omecamtiv mecarbil outside Japan and co-promotion rights. 

 

Janssen Research & Development announced several new collaborations through its Janssen Human Microbiome Institute (JHMI) focused on advancing the discovery, development and commercialization of next-generation microbial products. The JHMI, together with the Janssen Disease Interception Accelerator (DIA), has initiated a collaboration with DayTwo and the Weizmann Institute of Science in Rehovot, Israel, to pursue microbiome-based health solutions targeting metabolic disorders. In addition, with the scientific support of the JHMI and Bioqube Ventures, Caelus Health (Caelus) has become a portfolio company of Johnson & Johnson Innovation – JJDC, Inc. (JJDC) and one of the first companies to join the JLINX community in Beerse, Belgium, which nurtures early-stage companies by providing access to resources to grow and network across the European life science ecosystem. The agreements were facilitated by Johnson & Johnson Innovation.

 

MyoKardia announced dose administration for the first cohort of healthy subjects in its Phase I single ascending dose study of MYK-491.

 

Eli Lilly announced that the Japan IP High Court confirmed the decisions of the Japan Patent Office and ruled in Lilly’s favor in the invalidation trials initiated by Sawai regarding Lilly’s vitamin regimen patents for Alimta. In the fourth quarter of 2015, the Japan Patent Office issued written decisions upholding the validity of the Alimta vitamin regimen patents. If the patents are ultimately upheld through all challenges, they could provide intellectual property protection for Alimta in Japan until June 2021. Lilly will take necessary actions to defend and enforce the patents.

 

Galapagos announced dosing of the first patient with cystic fibrosis (CF) Class III (F508del and a gating mutation like G551D) with novel CF corrector GLPG2222 as an add-on to Kalydeco in a Phase IIa study. Galapagos further announced the opening of an IND file with the FDA for GLPG2222, triggering a $10 million milestone payment. The ALBATROSS Phase IIa study is a multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a diagnosis of CF harboring one F508del CFTR mutation and one gating mutation. Up to 35 evaluable subjects are planned to be included in the study. Eligible subjects must be on stable treatment with physician prescribed Kalydeco for at least 28 days at the baseline visit. They will receive one of two active doses of GLPG2222 or placebo q.d. administered for 29 days. The primary objective of ALBATROSS is to evaluate safety and tolerability of GLPG2222 in patients. Secondary objectives will include the assessment of ppFEV1, changes of sweat chloride, and CFQ-R. Topline results are expected in Q4 2017.

 

Swedish Orphan Biovitrum has received confirmation by the EC approving a reduced dosing frequency for Orfadin (nitisinone) from twice daily to once daily, in people with hereditary tyrosinemia type 1 (HT-1) with a body weight >20 kg.

 

Targovax announced encouraging overall survival data from an analysis of the first cohort of patients in its ongoing, phase I/II clinical trial evaluating TG01 (co-administered with GM-CSF) in resected pancreatic cancer given in combination with chemotherapy, gemcitabine, the current standard of care, study CTTG01-01. Data from this patient cohort showed that 68% of evaluated patients (13/19) were still alive after two years if survival is counted from time of resection which occurred on average two months prior to first treatment, or 12/19 if counted from time of first treatment. While the cohort is small and there is no control arm, this rate compares favorably with the available published historical two-year survival rates of resected cancer patients treated with gemcitabine alone of between 30% and 53% (J Neoptolemos 2010, J van Loethem 2010, H Oettle 2013, M Sinn 2015, K Uesaka 2016; In these reported studies Overall Survival measured either from surgery or treatment randomization).

 

ARCH Venture Partners and Versant Ventures announced the launch of Vividion Therapeutics, a biotechnology company focused on developing innovative therapeutics that treat major unmet clinical needs using the first platform for proteome-wide ligand and target discovery. ARCH and Versant co-led today’s $50 million Series A financing and were joined by founding investor Cardinal Partners.

 

Soligenix announced SGX301 (synthetic hypericin) has been granted Promising Innovative Medicine (PIM) designation in the United Kingdom (UK) by the Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of cutaneous T-cell lymphoma (CTCL).

 

NephroGenex and Medpace entered into a plan support agreement with respect to the terms of a restructuring to be implemented through a chapter 11 plan of reorganization to be proposed by the Company in place of the Company’s plan of liquidation currently on file with the Bankruptcy Court. Under the terms of the PSA, Medpace has agreed to waive its cash distribution with respect to its asserted $4.31 million unsecured claim against the Company in exchange for 100% of the newly issued common stock in the reorganized Company, subject to the Bankruptcy Court’s approval of the Company’s entry into the PSA and confirmation of the Plan.

 

Nabriva Therapeutics announced that following a blinded interim analysis, an independent committee recommended no sample size adjustment for the lefamulin evaluation against pneumonia (LEAP) 1 Phase III clinical trial. This was a prospectively defined, blinded interim analysis to confirm the sample size and power assumptions that was planned to occur after the trial achieved 60% of its enrollment target. The company expects to complete LEAP 1 enrollment of approximately 550 patients with moderate to severe community-acquired bacterial pneumonia (CABP) in the second quarter of 2017 and announce topline LEAP 1 data by the end of the third quarter of 2017.

 

Ionis Pharmaceuticals announced it has earned a $5 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. For this newest target, Ionis will continue to evaluate the target with the goal of advancing this program into development.

 

Fortress Biotech announced that Mustang Bio, a subsidiary of Fortress, has closed on a total of $94.5 million, prior to fees and expenses, in a private placement financing. OPN Capital Markets, the healthcare related investment banking and research division of National Securities Corporation, a wholly owned subsidiary of National Holdings Corporation, acted as sole placement agent in connection with the financing.

 

Hemispherx Biopharma announced that it has entered into definitive agreements with several institutional investors for an offering of shares of common stock with gross proceeds of approximately $1 million in a registered direct offering. The closing of the offering is expected to take place on or about February 6, 2017, subject to the satisfaction of customary closing conditions. In connection with the offering, the Company will issue registered shares of common stock at a purchase price of $0.55 per share. Concurrently in a private placement, for each share of common stock purchased by an investor, such investor will receive from the Company an unregistered warrant to purchase 0.75 of a share of common stock. The warrants have an exercise price of $0.75 per share, will be exercisable six months after issuance, and will expire five years from the initial exercise date. Rodman & Renshaw, a unit of H.C. Wainwright is acting as the exclusive placement agent in connection with this offering.

 

Corium International announced the pricing of its underwritten public offering of 6,666,667 shares of common stock at a public offering price of $3.00 per share. Gross proceeds to Corium from this offering are expected to be approximately $20 million. Corium intends to use the net proceeds from this offering for product development and general corporate purposes, which may include funding research and development, increasing its working capital, reducing indebtedness, acquisitions or investments in businesses, products or technologies that are complementary to its own and capital expenditures. Corium has granted the underwriters a 30-day option to purchase up to an additional 1,000,000 shares of its common stock offered in the public offering. All of the shares will be offered and sold by Corium. The offering is expected to close on February 7, 2017, subject to customary closing conditions. Cantor Fitzgerald is acting as the sole book-running manager for the offering.

 

Cantabio Pharmaceuticals announced that it has entered into a definitive agreement with an investment fund managed by Yorkville Advisors Global for the sale of up to $600,000 of convertible debentures. Cantabio has closed on the initial tranche of $300,000 and is scheduled to close on an additional $150,000 upon the filing of a registration statement registering for resale the shares of common stock underlying the convertible debentures and an additional $150,000 upon the effectiveness of such registration statement. The conversion of the Yorkville debentures shall be at the lower of $0.3107 or a 7% discount to market, with a floor price of $0.10.

 

Athersys announced the closing of its previously announced underwritten public offering of 22,772,300 shares of its common stock, par value $0.001 per share, at a price to the public of $1.01 per share, which includes the underwriters’ full exercise of their option to purchase an additional 2,970,300 shares of Common Stock that was exercised on January 31, 2017. Gross proceeds to Athersys from the offering are approximately $23.0 million, before deducting underwriting discounts, commissions and estimated offering expenses. William Blair & Company is acting as sole book-running manager and Needham & Company is acting as lead manager for the offering. Maxim Group served as financial advisor to Athersys. Athersys intends to use the net proceeds from the offering for working capital and general corporate purposes, including funding towards its Phase III MultiStem Administration for Stroke Treatment and Enhanced Recovery Study-2 (“MASTERS-2”) clinical study and its other ongoing clinical programs.

 

 

ANALYST RECOMMENDATIONS

 

Ladenburg Thalman analyst Christopher James initiated coverage of the following companies: Trevena with a “buy” rating and $15 price target; Achillion Pharmaceuticals with a “buy” rating and $10 price target; Alnylam Pharmaceuticals with a “buy” rating and $75 price target; Corcept Therapeutics with a “buy” rating and $14 price target. The analyst also assumed coverage of Acadia Pharmaceuticals with a “buy” rating and decreased the price target to $48 from $49.

 

The following analysts initiated coverage of Biovertiv: Morgan Stanley analyst Matthew Harrison with an “underweight” rating and $41 price target; Gabelli analyst Jing He with a “buy” rating and $48 price target.

 

Maxim Group analyst Jason Kolbert downgraded Galena Biopharma to “hold” from “buy” and removed his price target, citing the announcement of Galena’s CEO Dr. Mark Schwartz’s departure Tuesday evening.

 

Oppenheimer analyst Hartaj Singh decreased his price target of Cellectis to $40 from $65, citing early stage of development and lack of visibility.

 

Wedbush analyst David Nierengarten initiated coverage of the following companies: Juno Therapeutics with a “neutral” rating and $24 price target; Kite Pharma with a “neutral “rating and $46 price target.

 

H.C. Wainwright analyst Shaunak Deepak increased his price target of Calithera to $10 from $9, citing Calithera found a strong partner for CB-1158 in Incyte.

 

Following Biogen’s spinoff of Biovertiv; RBC analyst Michael Yee decreased his price target of Biogen to $350 from $375; Goldman analyst Terence Flynn decreased his price target to $282 from $310; Stifel analyst Thomas Shrader decreased his price target to $265 from $290.