On November 9, 2017 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported a corporate update and financial results for the third quarter ended September 30, 2017 (Press release, Onconova, NOV 9, 2017, View Source [SID1234521892]).

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"We are getting closer to two key milestones for the INSPIRE pivotal trial for IV rigosertib in patients with higher-risk MDS. A pre-planned interim analysis is anticipated in the coming months, and encouraging recent enrollment progress for this global trial suggests full enrollment may be achieved in the first half of 2018," said Dr. Ramesh Kumar, President and Chief Executive Officer.

"We have also made good progress on our oral rigosertib-azacitidine combination program and plan to initiate a Special Protocol Assessment process with the Food and Drug Administration for a pivotal Phase 3 trial early next year. In addition, we are advancing our collaborative pre-clinical RASopathies drug program and look forward to two poster presentations at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) conference."

INSPIRE Trial of IV Rigosertib in 2nd Line Higher-risk (HR) MDS

Interim Analysis (IA)

· In the quarter, the Company received guidance from the Food and Drug Administration (FDA) and European Medicines Agency (EMA) concerning the statistical analysis plan (SAP) for both the interim and final (top-line) analysis in the INSPIRE trial. Based on guidance received, Onconova finalized the SAP in preparation for the IA.
· The IA will be triggered with the 88th death event in this trial of 225 patients. Based on internal modeling, this could occur in the fourth quarter of 2017 or early 2018. The date of the IA is tied to reaching a pre-identified number of death events. Accordingly, the precise time of completing the IA, which will take place approximately a couple of weeks after reaching the number of events, cannot be accurately forecast.
· This adaptive trial design permits several options after the IA, including a futility analysis, trial expansion using pre-planned statistical criteria, or choosing one of two endpoints (survival analysis of the Intent-to-Treat population or the pre-defined Very High Risk subpopulation).

Trial Progress

· As of October 31, 2017, the INSPIRE study is active at approximately 170 sites in 22 countries across four continents. A final five sites will be opened, which is expected to occur in November.
· The INSPIRE trial has stringent selection criteria so as to identify a more homogenous MDS patient population. Accordingly, extensive eligibility verification and trial site education are integral to the Company’s plan.
· Due to efforts undertaken to increase participation, including the addition and replacement of CROs and opening sites in additional countries, the enrollment rate for the trial increased

recently. Consequently, Onconova expects full enrollment to be achieved in the first half of 2018, followed by top-line analysis after 176 death events in the second-half of 2018.

Oral Rigosertib in Combination with Azacitidine for 1st-line HR-MDS

Pivotal Phase 3 Trial Protocol

· Following input received from the FDA in an end-of-phase 2 meeting and from the EMA as part of the scientific advice process, Onconova has designed a Phase 3 protocol. The Company is awaiting the results of the ongoing Phase 1/2 Expansion Trial before engaging in further protocol development. Once the Expansion Trial is complete, which is expected to be in the fourth quarter of 2017, Onconova plans to submit the Pivotal Phase 3 protocol to the FDA in the first half of 2018 to initiate the SPA process with the FDA.
· Initiation of the Phase 3 trial, which is planned to be conducted globally, requires additional financing and/or business development transactions.
· This Expansion Trial is designed to enroll up to approximately 40 patients. More than half of the trial has been accrued in multiple sites in the USA. Based on this progress, the Company has decided to limit the trial to US sites.
· Onconova plans to present initial data from this study at a scientific conference in early 2018, highlighting the results of dose selection and optimization of the combination regimen.

Other Programs for Future Development or Partnership and Presentations

Rigosertib for Pediatric RASopathies

· On October 11, 2017, Onconova hosted a Key Opinion Leader meeting to discuss novel approaches to RASopathies. The meeting featured presentations by Bruce D. Gelb, M.D. (Mount Sinai, New York), and Elliot Stieglitz, M.D. (University of California San Francisco), who discussed new developments for pediatric patients with RASopathies, which are related genetic syndromes usually caused by mutations that alter the Ras subfamily and mitogen activated protein (MAP) kinases that control signal transduction. Onconova’s Chief Medical Officer, Steven Fruchtman, M.D., provided an update on rigosertib, which is initially planned to be studied in pediatric patients with RASopathies complicated by the development of associated cancers.
· The Company has completed and expects to sign a Cooperative Research and Development Agreement with the US National Institutes of Health (NIH) to advance rigosertib in pediatric clinical trials at the National Cancer Institute. This trial is expected to start next year and will be funded by the NIH.

Onconova Enters into Strategic Collaboration with Cellectar Biosciences

· On September 21, 2017, the Company announced it had entered into a strategic collaboration with Cellectar Biosciences to develop new phospholipid drug conjugates combining select proprietary compounds or payloads from Onconova’s early stage product pipeline with Cellectar’ s patented phospholipid ether delivery platform.
· Under the terms of the collaboration, Onconova will provide Cellectar with several compounds, including some from the family of molecules that contains Briciclib, which is an EIF4E targeting small molecule with early Phase 1 data. Cellectar will link the molecules to its phospholipid ether to create new, more precisely targeted antitumor agents.

Upcoming Presentations

Two abstracts relating to the Company’s lead product candidate, rigosertib, were accepted for poster presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, Georgia, which takes place December 9-12, 2017. Details of the presentations are listed below.

Long-term follow up of patients in a Phase 2 clinical trial of single agent oral rigosertib in lower-risk transfusion dependent MDS

Abstract Number: 1689

Title: Rigosertib Oral in Transfusion Dependent Lower Risk Myelodysplastic Syndromes (LR-MDS): Optimization of Dose and Rate of Transfusion Independence (TI) or Transfusion Reduction (TR) in a Single-Arm Phase 2 Study

Session Name: 637. Myelodysplastic Syndromes – Clinical Studies: Poster I

Date: Saturday, December 9, 2017; 5:30 – 7:30 PM EST

Studies on the mechanism of action of rigosertib azacitidine combination therapy for MDS

Abstract Number: 4235

Title: Effects of Rigosertib (RIGO) Alone or in Combination with Azacitidine or Vorinostat on Epigenetic Reprogramming of CD34+ Cells in the Myelodysplastic Syndrome

Session Name: 636. Myelodysplastic Syndromes – Basic and Translational Studies: Poster III

Date: Monday, December 11, 2017; 6:00 – 8:00 PM EST

Third-Quarter Financial Results:

· Cash, cash equivalents, and marketable securities as of September 30, 2017 totaled $7.6 million, compared to $21.4 million as of December 31, 2016. Based on our cash burn for the first three quarters of 2017 and our current projections, we expect that our cash and cash equivalents will be sufficient to fund our ongoing trials and operations through the end of 2017.

· Total net revenue was $0.1 million for the third quarter of 2017 and $0.6 million for the nine months ended September 30, 2017, compared to $1.7 million and $5.4 million, respectively, for the comparable periods in 2016.

· Research and development expenses were $5.1 million for the third quarter of 2017 and $14.6 million for the nine months ended September 30, 2017, compared to $4.0 million and $15.4 million, respectively, for the comparable periods in 2016.

· General and administrative expenses were $1.7 million for the third quarter of 2017 and $5.6 million for the nine months ended September 30, 2017, compared to $2.0 million and $7.2 million, respectively, for the comparable periods in 2016.

The Company will host a conference call on November 9th at 9:00 a.m. Eastern Time to provide a corporate update and discuss third quarter financial results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the US, or (210) 229-8823 internationally and using conference ID: 3588306.

The call will also be webcast live. Please click here to access the webcast.

A replay will be available at this link until February 23, 2018.

On November 9, 2017 Achieve Life Sciences, Inc. (NASDAQ: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisine for smoking cessation, reported financial results for the third quarter ended September 30, 2017 (Press release, OncoGenex Pharmaceuticals, NOV 9, 2017, View Source [SID1234521891]).

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Third Quarter 2017 Highlights

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Consummated merger with OncoGenex Pharmaceuticals

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Investigational New Drug (IND) application accepted by the U.S. Food and Drug Administration (FDA) enabling the commencement of cytisine development in the U.S.

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Initiated cytisine’s clinical development activities in preparation for pivotal U.S. Phase 3 program

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Commenced multi-dose study to evaluate the pharmacokinetic/pharmacodynamic characteristics of cytisine in smokers

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Completed enrollment in a study evaluating the effect of food on the bioavailability of cytisine

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Entered into a share purchase agreement with Lincoln Park Capital Fund, LLC

"I’m extremely pleased with our progress during the period across both clinical and corporate development fronts. Clearly, joining the NASDAQ Capital Markets platform via our merger with OncoGenex was a major milestone as we advance cytisine, our smoking cessation treatment, toward its pivotal U.S. Phase 3 program which remains on track to initiate in mid-2018," commented Rick Stewart, Chairman and Chief Executive Officer of Achieve.

On August 1, 2017, Achieve announced the closing of its merger with publicly listed OncoGenex Pharmaceuticals. The prevailing entity combined operations and employees from both companies with shareholders of Achieve becoming the majority stockholders of OncoGenex in a 1-for-11 reverse stock split with the trading symbol transitioning from OGXI to ACHV.

On August 10th, the U.S. Food and Drug Administration (FDA) accepted Achieve’s Investigational New Drug (IND) application for cytisine which provided the Company authorization to commence clinical development of the smoking cessation treatment in the U.S. Cytisine has been approved and marketed in Central and Eastern Europe for more than 25 years. It is a plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor. It is estimated that over 20 million people have used cytisine to help combat nicotine addiction, including approximately 2,100 patients in Phase 3 clinical trials conducted in Europe and New Zealand. Achieve has also collaborated with the National Center for Complementary and Integrative Health (NCCIH) at the National Institutes of Health (NIH), which has sponsored and completed a number of the preclinical IND-enabling studies .

In preparation for initiating cytisine’s pivotal Phase 3 program in the U.S., the Company announced on August 16th, its Clinical Development Plan which entails two phase 1/2 studies aimed at assisting the design and implementation of the subsequent Phase 3 program. The first study was the evaluation of the effect of food on the bioavailability of cytisine, which has completed and data analysis is expected in Q4 2017. The second study was the evaluation of repeat-dose pharmacokinetic and pharmacodynamic characteristics of cytisine in smokers, which commenced in Q4 2017. Data from this repeat-dose study is expected in the first quarter of 2018.

On September 14, the Company announced that it had entered into a share purchase agreement with Lincoln Park Capital Fund, LLC in which Achieve may sell up to $11.0 million of shares of common stock over a 30 month term subject to certain limitations and conditions set forth in the purchase agreement. Through September 30, 2017, the company offered and sold 408,947 shares of common stock to Lincoln Park resulting in proceeds of $1.2 million net of offering costs. As consideration for entering into the Purchase Agreement, we issued to LPC 123,516 shares of common stock; no cash proceeds were received from the issuance of these shares. Achieve plans to utilize the net proceeds from this offering to advance its product candidate cytisine as well as for general corporate purposes. From October 1, 2017 through November 9, 2017, we offered and sold 464,831 shares of our common stock pursuant to our Purchase Agreement with LPC. These sales resulted in gross proceeds to us of approximately $0.9 million.

Financial Results

As of September 30, 2017, the company’s cash and cash equivalents were $8.0 million compared with $15,000 as of December 31, 2016.

Total operating expenses for the three and nine months ended September 30, 2017 were $2.4 million and $2.9 million, respectively, compared to $0.4 million and $1.1 million for the three and nine months ended September 30, 2016, respectively.

Net loss for the three and nine months ended September 30, 2017 was $6.5 million and $6.8 million, respectively, compared to $0.3 million and $0.7 million for the three and nine months ended September 30, 2016, respectively.

As of November 9, 2017 Achieve had 11,947,676 shares outstanding.

On November 9, 2017 Omeros Corporation (NASDAQ: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases and disorders of the central nervous system, reported recent highlights and developments as well as financial results for the third quarter ended September 30, 2017, which include (Press release, Omeros, NOV 9, 2017, View Source;p=RssLanding&cat=news&id=2316004 [SID1234521890]):

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3Q 2017 total and OMIDRIA revenues were $21.7 million. Revenues from OMIDRIA sales rose 26 percent from 2Q 2017 and 92 percent from the prior year’s third quarter. Units sold to wholesalers, or "sell-in," increased 26 percent quarter-over-quarter and 125 percent year-over-year.
Net loss in 3Q 2017 was $7.5 million, or $0.16 per share, which included $4.2 million ($0.09 per share) of non-cash expenses. Net loss in the prior year’s third quarter was $14.0 million or $0.34 per share, which included $3.1 million ($0.08 per share) of non-cash expenses.
At September 30, 2017, the company had cash, cash equivalents and short-term investments available for operations of $86.8 million plus the ability to borrow an additional $45.0 million from existing lenders.
Settled patent infringement lawsuit against Par Pharmaceutical, Inc. and its affiliate (collectively, Par) on favorable terms in October 2017.
Met with FDA in follow-up to FDA’s granting breakthrough designation for OMS721 in immunoglobin A (IgA) nephropathy; the Agency’s meeting minutes state that approval can be obtained with a single successful Phase 3 trial with reduction in proteinuria as the primary efficacy endpoint.
FDA granted OMS721 orphan drug designation in IgA nephropathy.
"OMIDRIA revenues sustained their strong growth in the third quarter and this momentum continues into the current quarter," said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. "We have also made substantial progress across our OMS721 programs – in addition to our Phase 3 aHUS program, we have a clear roadmap for the Phase 3 IgA nephropathy trial, including FDA confirmation of proteinuria as the primary efficacy endpoint, and compelling data to support our advancing to a Phase 3 program in stem cell transplant-associated TMA. Further adding to our clinical pipeline, OMS527, our PDE7 inhibitor for the treatment of addictions and compulsive disorders, is on track to enter Phase 1 in the first half of next year."

Third Quarter and Recent Highlights and Developments

In October, the company entered into a settlement agreement and consent judgment with Par, which resolved Omeros’ patent litigation against Par. The litigation concerned Par’s filing of an Abbreviated New Drug Application (ANDA) seeking approval from the FDA to market a generic version of OMIDRIA. Pursuant to the settlement agreement and consent judgment, Par is prohibited from launching a generic version of OMIDRIA until April 1, 2032 or as detailed in the settlement agreement. Par also acknowledged and confirmed the validity of Omeros’ OMIDRIA patents at issue in the lawsuit in the settlement agreement.
Highlights and developments regarding OMS721, Omeros’ lead human monoclonal antibody in its mannan-binding lectin-associated serine protease-2 (MASP-2) programs for the treatment of thrombotic microangiopathies (TMAs), including atypical hemolytic uremic syndrome (aHUS) and hematopoietic stem cell-associated TMA (HSCT-TMA), and for the treatment of complement-related renal diseases, including IgA nephropathy, include:
Omeros met with the FDA in follow-up to the FDA’s granting breakthrough designation for OMS721 in IgA nephropathy to discuss Phase 3 trial design. The Agency’s meeting minutes make clear that approval can be obtained with a single successful Phase 3 trial with reduction in proteinuria as the primary efficacy endpoint. Depending on the size of the effect on proteinuria, either full approval or accelerated approval is possible. If full approval is granted based on reduction in proteinuria, estimated glomerular filtration rate (eGFR) will be followed as part of the safety assessment. Any effect of OMS721 on eGFR is likely to result in additional label claims for the product. If, based on the effect on proteinuria, accelerated rather than full approval is granted, marketing of OMS721 would be allowed during which time confirmatory data on long-term effects of OMS721 on eGFR would be collected. These eGFR data, if satisfactory, would then form the basis for full approval.
Omeros reported in August that the FDA granted orphan drug designation to OMS721 for the treatment of IgA nephropathy. The FDA has also granted breakthrough therapy designation to OMS721 for the treatment of IgA nephropathy. In Europe, Omeros is pursuing orphan designation and Priority Medicines (PRIME) status from the European Medicines Agency (EMA) for OMS721 in the treatment of IgA nephropathy.
In October, Omeros announced the presentation by a trial investigator of a case report of a patient having co-existing HSCT-TMA and graft-versus-host disease (GvHD), which both resolved following OMS721 treatment. This case was presented at the European Society for Blood and Marrow Transplantation Crash Course on Diagnosis and Treatment of Noninfectious Complications after HCT in Granada, Spain. The company plans to initiate a Phase 3 clinical program in HSCT-TMA before year-end. Omeros is also pursuing breakthrough therapy designation from FDA and PRIME status from the EMA in this indication.
In November, Omeros announced the presentation at the American Society of Nephrology Conference of follow-up data on the four IgA nephropathy patients in the open-label portion of the Phase 2 trial. As previously reported, all four patients demonstrated a substantial reduction in proteinuria during the clinical trial. In the extended (up to one year) follow-up after completion, proteinuria reduction was maintained in three of the four patients. Specifically, those three patients maintained partial remission relative to baseline (76 percent to 86 percent decrease in albumin/creatinine ratios (uACRs)) during extended follow-up. After a substantial drop in uACR during the trial, the fourth patient’s uACR returned to 88 percent of baseline at four months post-treatment. eGFR improved in three of the four patients during the extended follow-up, with increases ranging from 7 to 17 mL/min/1.73 m2 (up to 57 percent improvement) relative to baseline. The fourth patient demonstrated stable eGFR relative to baseline. OMS721 was well-tolerated.
In August, Omeros sold 3.0 million shares of common stock in a public offering with a price to the public of $22.75 per share, receiving net proceeds of $63.6 million.
In October, Omeros extended the borrowing capacity under its existing credit facility allowing the company to borrow, at its sole discretion, up to $45.0 million through March 21, 2018 subject only to customary closing conditions.
Financial Results

For the quarter ended September 30, 2017, total revenues were $21.7 million, all from sales of OMIDRIA. This compares to OMIDRIA revenues of $11.3 million for the same period in 2016. On a sequential quarter-over-quarter basis, OMIDRIA revenue grew $4.5 million, or 26 percent, and grew 92 percent year-over-year. The quarter-over-quarter increases in OMIDRIA revenue and units sold are due to both an increase in the number of customers purchasing OMIDRIA and increased penetration into existing customer accounts.

Total costs and expenses for the three months ended September 30, 2017 were $26.8 million compared to $23.3 million for the same period in 2016. The increase in the current year quarter was primarily due to increased third-party manufacturing scale-up costs associated with OMS721, increased preclinical and development costs as Omeros continues to advance drug candidates toward the clinic and increased legal costs associated with the Par lawsuit, which settled in October 2017 on favorable terms to Omeros.

Interest expense for the three months ended June 30, 2017 was $2.8 million as compared to $2.1 million in the prior year third quarter. The increase is due to incremental funds borrowed by the company in November 2016.

For the three months ended September 30, 2017, Omeros reported a net loss of $7.5 million, or $0.16 per share, which included non-cash expenses of $4.2 million ($0.09 per share). This compares to the prior year’s third quarter where Omeros reported a net loss of $14.0 million, or $0.34 per share, which included non-cash expenses of $3.1 million ($0.08 per share).

As of September 30, 2017, the company had $86.8 million of cash and cash equivalents available for operations and $5.8 million in restricted cash. The company has the ability, at its sole discretion, to borrow up to an additional $45.0 million from its existing lenders through March 21, 2018 subject only to customary closing conditions.

Conference Call Details

Omeros’ management will host a conference call to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 8389089. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 8389089.

To access the live or subsequently archived webcast of the conference call on the internet, go to the company’s website at www.omeros.com and select "Events" under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

On November 9, 2017 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three and nine months ended September 30, 2017, and provided an operating forecast and program updates (Press release, Ligand, NOV 9, 2017, View Source [SID1234521879]).

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"Ligand’s diversified business is performing well on all levels. In the third quarter, we posted strong financial results driven by royalties and significant contribution from Captisol. Of note, for the first nine months of 2017, royalties are more than 50% higher than those of the same period last year. This past quarter, we entered three new licensing deals and saw substantial news flow from our corporate partners as many pipeline programs advanced. We announced positive results for a major phase 2 trial for our diabetes drug candidate and are in discussions with partners for potential licensing," said John Higgins, Chief Executive Officer of Ligand. "Just after the quarter ended, we closed our acquisition of Crystal Bioscience providing a highly-complementary antibody technology to our OmniAb antibody drug discovery business. We now have three species for fully-humanized antibody discovery, four proprietary technology platforms driving licensing and more than 160 fully-funded Shots on Goal."

Ligand management will discuss third quarter financial and operational results during the Company’s upcoming Analyst Day presentation, which will take place next Tuesday, November 14 from 4:00 p.m. to 5:30 p.m. Eastern time (1:00 p.m. to 2:30 p.m. Pacific time) in New York City. The event will be webcast live and can be accessed at www.ligand.com. As such, Ligand will not be hosting an earnings conference call this quarter.

Third Quarter 2017 Financial Results

Total revenues for the third quarter of 2017 were $33.4 million, compared with $21.6 million for the same period in 2016. Royalties were $21.9 million, compared with $15.7 million for the same period in 2016, an increase of 40%, primarily due to higher royalties from Promacta, Kyprolis and EVOMELA. Material sales were $7.7 million, compared with $4.2 million for the same period in 2016 due to the timing of Captisol purchases for use in clinical trials and commercial products. License fees, milestones and other revenues were $3.8 million, compared with $1.7 million for the same period in 2016.

Cost of goods sold was $2.4 million for the third quarter of 2017, compared with $1.0 million for the same period in 2016. Amortization of intangibles was $2.7 million in both periods. Research and development expense was $4.8 million, compared with $5.9 million for the same period of 2016. General and administrative expense was $7.0 million, compared with $6.6 million for the same period in 2016.

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Net income for the third quarter of 2017 was $8.4 million, or $0.36 per diluted share, compared with $1.1 million, or $0.05 per diluted share, for the same period in 2016. Adjusted net income for the third quarter of 2017 was $15.3 million, or $0.69 per diluted share, compared with $9.6 million, or $0.44 per diluted share, for the same period in 2016.

As of September 30, 2017, Ligand had cash, cash equivalents and short-term investments of $202.3 million, or approximately $175 million after deducting the upfront cash paid in the recent acquisition of Crystal Bioscience. Cash generated from operations was $27.7 million for the 2017 third quarter.

Year-to-Date Financial Results

Total revenues for the nine months ended September 30, 2017 were $90.6 million, compared with $70.8 million for the same period in 2016. Royalties were $60.4 million, compared with $39.8 million for the same period in 2016, an increase of 52%, primarily due to higher royalties from Promacta, Kyprolis and EVOMELA. Material sales were $14.3 million, compared with $13.4 million for the same period in 2016 due to the timing of Captisol purchases for use in clinical trials and commercial products. License fees, milestones and other revenues were $15.9 million, compared with $17.5 million for the same period in 2016, due primarily to the timing of milestones and license fees earned.

Cost of goods sold was $3.6 million for the nine months ended September 30, 2017, compared with $2.7 million for the same period in 2016 due to the timing and mix of Captisol sales. Amortization of intangibles was $8.1 million, compared with $7.9 million for the same period in 2016. Research and development expense was $18.3 million, compared with $14.8 million for the same period of 2016 due to enrollment costs of our Phase 2 GRA trial and non-cash stock-based compensation expense. General and administrative expense was $20.9 million in both periods.

Net income for the nine months ended September 30, 2017 was $19.6 million, or $0.84 per diluted share, compared with $1.5 million, or $0.07 per diluted share, for the same period in 2016. Adjusted net income for the nine months ended September 30, 2017 was $42.9 million, or $1.94 per diluted share, compared with $30.6 million, or $1.41 per diluted share, for the same period in 2016.

2017 Financial Forecast

Ligand updates guidance for 2017 revenue to be between $134 and $136 million. Adjusted earnings per diluted share is now expected to be between $2.95 and $3.00. Previous guidance was for revenue to be at least $134 million plus up to an additional $9 million in contract payments. Ligand previously noted that with $134 million of revenue, adjusted earnings per share would be $2.93.

Recent Acquisition

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In October 2017, Ligand acquired Crystal Bioscience and its OmniChicken antibody discovery technology for $25 million cash at closing, up to $10.5 million of success-based milestones and revenue sharing from existing licensees for a defined period. The acquisition initially added four Shots on Goal to Ligand’s portfolio, and the OmniChicken technology may be utilized by multiple current OmniAb partners as they seek to develop antibodies for difficult-to-address epitopes.

Third Quarter 2017 and Recent Business Highlights

Promacta/Revolade

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Novartis reported third quarter 2017 net sales of Promacta/Revolade (eltrombopag) of $227 million, a $59 million or 35% increase over the same period in 2016.
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Novartis announced long-term study results supporting the positive safety and efficacy of Revolade (eltrombopag) in adults with chronic/persistent (6 or more months from diagnosis) immune (idiopathic) thrombocytopenia (ITP) were published online in Blood. The EXTEND study found that a majority of patients maintained a substantial clinical response and many no longer needed concomitant ITP medications.

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Novartis highlighted the product in abstracts for the upcoming 59th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting

Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

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On October 25, 2017, Amgen reported third quarter net sales of Kyprolis of $207 million, a $24 million or 13% increase over the same period in 2016. On November 6, 2017, Ono Pharmaceutical Company reported Kyprolis sales in Japan of approximately $13.1 million for the most recent quarter.
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On October 23, 2017, Amgen announced top-line results of the Phase 3 ARROW trial, which showed Kyprolis administered once-weekly at the 70 mg/m2 dose with dexamethasone allowed relapsed and refractory multiple myeloma patients to live 3.6 months longer without their disease worsening than Kyprolis administered twice-weekly at the 27 mg/m2 dose with dexamethasone.
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On August 30, 2017, Amgen announced that the FDA accepted a supplemental New Drug Application (sNDA) based on the overall survival (OS) data from the Phase 3 ENDEAVOR trial demonstrating that Kyprolis and dexamethasone (Kd) reduced the risk of death by 21% and increased OS by 7.6 months versus Velcade (bortezomib) and dexamethasone (Vd) in patients with relapsed or refractory multiple myeloma. The FDA has set an action date of April 30, 2018.
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On July 12, 2017, Amgen announced positive results from final analysis of the Phase 3 ASPIRE trial, showing the study met the key secondary endpoint of OS, demonstrating that Kyprolis, lenalidomide and dexamethasone (KRd) reduced the risk of death by 21% over lenalidomide and dexamethasone alone.

Additional Pipeline and Partner Developments

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Sage Therapeutics announced positive top-line results from two Phase 3 trials of brexanolone in severe postpartum depression (PPD) and in moderate PPD. Sage plans to file a New Drug Application (NDA) with the FDA in 2018.
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Spectrum Pharmaceuticals reported third quarter 2017 net sales of EVOMELA of $10.5 million.
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CASI Pharmaceuticals announced that China’s Food and Drug Administration granted priority review for CASI’s import drug registration clinical trial application for EVOMELA.
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Melinta Therapeutics announced a merger with NASDAQ-listed Cempra, Inc. to form a company focused on developing and commercializing important anti-infective therapies including recently-approved Baxdela.
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Melinta Therapeutics announced that its commercialization and distribution agreement with Eurofarma Laboratórios for delafloxacin (Baxdela in the U.S.) had been expanded to include 19 countries in South America, Central America and the Caribbean.
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Zydus Cadila announced that it received approval to market its bevacizumab biosimilar in India and subsequently launched the drug, which is marketed as Bryxta.
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Exelixis announced that Daiichi Sankyo reported positive top-line results from a Phase 3 pivotal trial of esaxerenone in patients with essential hypertension in Japan and that a Japanese regulatory application is expected to be submitted in the first quarter of 2018.
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Retrophin announced that it presented new data from the open-label extension portion of the Phase 2 DUET study of sparsentan for the treatment of focal segmental glomerulosclerosis (FSGS) at the American Society of Nephrology Kidney Week 2017.
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Aldeyra Therapeutics announced positive results from a Phase 2a clinical trial of topical ocular ADX-102 in patients with dry eye disease.
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Aldeyra Therapeutics announced it will present data from its Phase 2 clinical trial in noninfectious anterior uveitis at the American Uveitis Society Fall Meeting.
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Viking Therapeutics announced enrollment completion in the ongoing Phase 2 clinical trial of VK5211 in patients who recently suffered a hip fracture.
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Viking Therapeutics announced results of gene expression analysis from its in vivo study of VK2809 in Non-Alcoholic Steatohepatitis (NASH) and presented data at the Annual Meeting of the American Association for the Study of Liver Diseases.
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Viking Therapeutics announced presentation of data from an in vivo proof-of-concept study of VK2809 in Glycogen Storage Disease Ia at the 13th International Congress of Inborn Errors of Metabolism.

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Viking Therapeutics announced positive top-line results from a 25-week proof-of-concept study of VK0214 in an in vivo model of X-linked adrenoleukodystrophy (X-ALD) and presented data at the 87th Annual Meeting of the American Thyroid Association.
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Sermonix Pharmaceuticals announced completion of a financing round to fund a Phase 2 clinical trial of lasofoxifene in Estrogen Receptor Positive (ER+) Metastatic Breast Cancer.
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Opthea announced further positive results from its Phase 1/2a clinical trial of OPT-302 for wet age-related macular degeneration (wet AMD).
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CStone Pharmaceuticals announced that it received Clinical Trial Application approval from the China Food and Drug Administration to conduct clinical trials in China with CS1001, an OmniAb-derived full-length anti-PDL1 monoclonal antibody.
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Aptevo Therapeutics announced that it had presented new preclinical data on OmniAb-derived APVO436 at the World Bispecific Summit and also at the AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics 2017 annual meeting.
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HanAll Biopharma, an OmniAb partner, announced entering into a strategic collaboration with Harbour BioMed to develop novel biologic therapies in greater China.
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ARMO BioSciences, an OmniAb partner, announced a $67 million Series C-1 financing to fund their immunotherapy pipeline.
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Immunoprecise Antibodies, an OmniAb Contract Research Organization (CRO), announced recent success in conducting OmniAb antibody-generation projects for Aptevo Therapeutics and Tizona Therapeutics.
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A paper was published by Ligand scientists in the journal MAbs, entitled "Chickens with humanized immunoglobulin genes generate antibodies with high affinity and broad epitope coverage to conserved targets", highlighting the use of OmniChicken in antibody drug discovery.

Internal Glucagon Receptor Antagonist (GRA) Program

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In September 2017, Ligand presented positive top-line results from its Phase 2 clinical study evaluating the efficacy and safety of LGD-6972, as an adjunct to diet and exercise, in subjects with type 2 diabetes mellitus (T2DM) inadequately controlled on metformin monotherapy. The study achieved statistical significance (p < 0.0001) in the primary endpoint of change from baseline in hemoglobin A1c (HbA1c) after 12 weeks of treatment at all doses tested, demonstrating a robust, dose-dependent reduction in HbA1c of 0.90%, 0.92% and 1.20% with 5 mg, 10 mg and 15 mg of LGD-6972, respectively, compared to a 0.15% reduction with placebo. LGD-6972 was safe and well tolerated, with no drug-related serious adverse events and no dose-dependent changes in lipids (including total cholesterol, LDL cholesterol, HDL cholesterol and triglycerides), body weight or blood pressure after 12 weeks of treatment.

New Licensing Deals

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Ligand announced receipt of a $2 million payment from WuXi Biologics subsequent to their licensing of exclusive rights to the anti-PD-1 antibody GLS-010 to Arcus Biosciences in North America, Europe, Japan and certain other territories. Ligand is also entitled to future milestones and royalties from this antibody.
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Ligand announced a commercial license and supply agreement with Amgen granting rights to use Captisol in the formulation of AMG 330, an anti-CD33 x anti-CD3 (BiTE) bispecific antibody construct. Ligand is eligible to receive milestone payments, royalties and revenue from Captisol material sales related to AMG 330.
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Ligand entered into Captisol Clinical Use Agreements with both Syros Pharmaceuticals and Vaxxas Inc.

Adjusted Financial Measures

The Company reports adjusted net income and adjusted net income per diluted share, in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include stock-based compensation expense, amortization of debt-related costs, amortization related to acquisitions, changes in contingent liabilities, net losses of Viking Therapeutics, mark-to-market adjustment for amounts owed to licensors, fair value adjustments to Viking Therapeutics convertible note receivable and warrants, unissued shares relating to the Senior Convertible Note and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included in this press release. However, other than with respect to total revenue, the Company only provides guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, net losses of Viking Therapeutics, stock based compensation expenses, mark-to-market adjustments for amounts owed to licensors, effects of any discrete income tax items and fair value adjustments to Viking Therapeutics convertible note receivable. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

On November 9, 2017 Intrexon Corporation (NYSE: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported its third quarter financial results for 2017 (Press release, IntelGenx, NOV 9, 2017, View Source [SID1234521874]).

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Business Highlights and Recent Developments:

Xogenex, a majority-owned subsidiary of Intrexon, filed an Investigational New Drug application with the U.S. Food and Drug Administration for a Phase 1 trial of the gene therapy INXN-4001, the world’s first multigene therapeutic candidate expressing proteins from three cardiac effector genes for the treatment of heart disease;
Intrexon’s proprietary methanotroph bioconversion platform continued to increase yield across multiple products including 2,3 butanediol, which increased approximately 15% during the quarter, and isobutanol, which increased 78%;
Okanagan Specialty Fruits, a wholly-owned subsidiary of Intrexon, recently initiated the first commercial launch of its non-browning Arctic Golden fresh sliced apples in stores across the mid-West and other regions;
Collaborator ZIOPHARM Oncology, Inc. (Nasdaq: ZIOP) announced that the first patient has been dosed in a new Phase 1 study of its gene therapy Ad-RTS-hIL-12 + veledimex for the treatment of pediatric brain tumors;
Collaborator Oragenics, Inc. (NYSE MKT: OGEN) dosed the first patient in its Phase 2 clinical trial of AG013, an ActoBiotics therapeutic candidate, for the treatment of oral mucositis;
Collaborator Fibrocell Science, Inc. (NASDAQ: FCSC) reported interim results in its Phase 1/2 clinical trial of its gene therapy FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa with encouraging safety and positive early trends noted in wound healing and pharmacology signals;
Intrexon Crop Protection announced the achievement of a key milestone in its collaboration with a leading agricultural company on the development of an eco-friendly fall armyworm solution utilizing Oxitec’s self-limiting technology, the receipt of a milestone payment, and the continued advancement of the program. Native to the Americas, fall armyworm invaded Africa in 2016 and has rapidly spread to at least 28 countries causing an estimated $13.8 billion in losses of maize, sorghum, rice and sugarcane;
Oxitec’s innovative solution to suppress the diamondback moth (DBM), a major pest of brassica crops that costs farmers over $4 billion yearly in crop losses and control management, began field trials in the U.S. following the Finding of No Significant Impact issued by the U.S. Department of Agriculture. DBM is considered one of the most difficult pests to control because it has become resistant to dozens of chemical insecticides and has also evolved resistance in the field to Bacillus thuringiensis (Bt) proteins;
Intrexon announced a collaboration with Arch Pharmalabs, Ltd. to develop microbial strains for fermentation-based production of an active pharmaceutical ingredient currently sourced from animals; and
Intrexon entered into a Preferred Stock Equity Facility with an affiliate of Third Security, LLC, a venture capital firm founded by Randal J. Kirk, Intrexon’s Chairman and Chief Executive Officer, under which Intrexon may, from time to time at its discretion, sell to the investor up to $100 million of newly issued Series A Redeemable Preferred Stock.

Third Quarter 2017 Financial Highlights:

Total revenues of $46.0 million, a decrease of 6% from the third quarter of 2016;
Net loss of $39.7 million attributable to Intrexon, or $(0.33) per basic share, including non-cash charges of $24.0 million;
Adjusted EBITDA of $(16.4) million, or $(0.14) per basic share;
The net change in deferred revenue related to upfront and milestone payments, which represents the cash and stock received from collaborators less the amount of revenue recognized during the period, was a decrease of $8.6 million compared to a decrease of $1.8 million in the third quarter of 2016; and
Cash, cash equivalents, and short-term investments totaled $108.7 million, the value of preferred shares totaled $148.5 million, and the value of common equity securities totaled $26.6 million at September 30, 2017.

Year-to-Date 2017 Financial Highlights:

Total revenues of $154.0 million, an increase of 6% over the nine months ended September 30, 2016;
Net loss of $89.8 million attributable to Intrexon, or $(0.75) per basic share, including non-cash charges of $66.0 million;
Adjusted EBITDA of $(25.5) million, or $(0.21) per basic share; and
The net change in deferred revenue related to upfront and milestone payments, which represents the cash and stock received from collaborators less the amount of revenue recognized during the period, was a decrease of $28.2 million compared to a net increase of $127.8 million in the nine months ended September 30, 2016.
"I am proud of our team for its astonishing number of significant accomplishments. We continue our transition from a company with substantial potential to one that is realizing that promise scientifically and commercially," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon. "Four years ago, we were focused almost exclusively on early stage programs but now are engaged in partnering efforts to capitalize on certain of our mature programs, including Methane Bioconversion Platform and Intrexon Crop Protection. We believe that these efforts will allow us to increase our investments in ground-breaking new programs while realizing, sometimes with partners, the available potential of our earlier work.

"Today, for example, with earlier-developed therapeutic candidates moving into Phase 3 and Phase 2 clinical trials, we mark a historic technical achievement in the fight against heart failure with the filing of an IND for the world’s first multigenic gene therapy targeting the leading cause of human death. This candidate generated promising safety and efficacy data in large animal models, and we are hopeful that it will greatly improve the prospects for the many patients — 5.7M adults in the U.S. alone — with this grim diagnosis. We shall be introducing additional complex, multigenic therapies into the clinic, both through our partners and on behalf of Precigen, in the near future.

"We see a similar pattern in our Energy and Food portfolios and look forward to developing this in Environment and Consumer as well and so believe that the balance of this year and 2018 will be validating for our strategy and ambition," concluded Mr. Kirk.

Third Quarter 2017 Financial Results Compared to Prior Year Period

Total revenues decreased $3.0 million, or 6%, from the quarter ended September 30, 2016. Collaboration and licensing revenues decreased $2.4 million from the quarter ended September 30, 2016 due to a decrease in research and development services for certain of the Company’s collaborations as the Company temporarily redeployed certain resources towards supporting prospective new platforms and partnering opportunities. Product revenues decreased $1.6 million, or 17%, primarily due to lower customer demand for cows and live calves. Gross margin on products also decreased in the current period primarily due to customer demand. Service revenues increased $1.3 million, or 15%, due to an increase in the number of bovine in vitro fertilization cycles performed due to higher customer demand. Gross margin on these services was consistent period over period.

Research and development expenses increased $7.4 million, or 26%, due primarily to increases in (i) salaries, benefits and other personnel costs for research and development employees, (ii) depreciation and amortization, (iii) rent and utilities expenses, and (iv) lab supplies and consulting expenses. Salaries, benefits and other personnel costs increased $2.6 million due to an increase in research and development headcount necessary to invest in current or expanding platforms and to develop new prospective collaborations and other partnering opportunities. Depreciation and amortization increased $2.0 million primarily as a result of (i) the amortization of developed technology acquired from Oxitec, which began in November 2016 upon the completion of certain operational and regulatory events, and (ii) the amortization of developed technology acquired from GenVec in June 2017. Rent and utilities expenses increased $1.4 million primarily due to the expansion of certain facilities to support the Company’s increased headcount. Lab supplies and consulting expenses increased $1.1 million as a result of (i) the progression of certain programs into the preclinical and clinical phases with certain of Intrexon’s collaborators and (ii) the expansion or improvement of certain of the Company’s platform technologies. Selling, general and administrative (SG&A) expenses increased $5.5 million, or 16%. Salaries, benefits and other personnel costs increased $2.7 million primarily due to (i) increased headcount to support the Company’s expanding operations and (ii) increased stock-based compensation expense resulting from grants to certain of the Company’s officers in February 2017. Legal and professional fees increased $0.9 million primarily due to (i) increased legal fees to defend ongoing litigation and (ii) increased fees incurred for business development and prospective partnering efforts.

Total other income, net, increased $1.4 million, or 31%. This increase was primarily attributable to (i) increases in fair market value of the Company’s equity securities portfolio and (ii) dividend income from the Company’s investments in preferred stock.

Equity in net loss of affiliates, which includes the Company’s pro-rata share of the net losses of its investments accounted for using the equity method of accounting, decreased $3.3 million, or 52%. This decrease was primarily due to the temporary redeployment of certain of the Company’s resources away from these joint venture programs towards supporting prospective new platforms and additional collaborations.

Year-to-Date 2017 Financial Results Compared to Prior Year Period

Total revenues increased $9.0 million, or 6%, over the nine months ended September 30, 2016. Collaboration and licensing revenues increased $7.2 million, or 9%, over the nine months ended September 30, 2016, primarily due to the recognition of deferred revenue associated with the payment received in June 2016 from ZIOPHARM to amend the collaborations between the parties and increased revenues associated with collaborations entered into with the Harvest start-up entities in 2016. Product revenues decreased $2.9 million, or 10%, primarily due to lower customer demand for cows and live calves. Gross margin on products improved in the current period primarily due to a decline in the average cost of cows. Service revenues increased $4.6 million, or 14%, due to an increase in the number of bovine in vitro fertilization cycles performed due to higher customer demand. Gross margin on services decreased slightly in the current period primarily due to an increase in royalties and commissions due to vendors.

Research and development expenses increased $21.4 million, or 26%, due primarily to increases in (i) salaries, benefits and other personnel costs for research and development employees, (ii) lab supplies and consulting expenses, (iii) depreciation and amortization, and (iv) rent and utilities expenses. Salaries, benefits and other personnel costs increased $7.4 million due to an increase in research and development headcount necessary to invest in current or expanding platforms and to develop new prospective collaborations and other partnering opportunities. Lab supplies and consulting expenses increased $6.3 million as a result of (i) the progression of certain programs into the preclinical and clinical phases with certain of Intrexon’s collaborators and (ii) the expansion or improvement of certain of the Company’s platform technologies. Depreciation and amortization increased $4.3 million primarily as a result of (i) the amortization of developed technology acquired from Oxitec, which began in November 2016 upon the completion of certain operational and regulatory events, and (ii) the amortization of developed technology acquired from GenVec in June 2017. Rent and utilities expenses increased $2.5 million due to the expansion of certain facilities to support the Company’s increased headcount. SG&A expenses increased $6.3 million, or 6%. Salaries, benefits and other personnel costs increased $4.2 million primarily due to (i) increased headcount to support the Company’s expanding operations and (ii) increased stock-based compensation expense resulting from grants to certain of the Company’s officers in February 2017. Legal and professional fees increased $4.7 million primarily due to (i) increased legal fees to defend ongoing litigation, (ii) increased business development and public relations consulting expenses, and (iii) the Company’s acquisition of GenVec that was completed in June 2017. These increases were offset by $4.2 million in litigation expenses recorded in the prior period arising from the entrance of a court order in Trans Ova Genetics, L.C.’s trial with XY, LLC.

Total other income (expense), net, increased $66.8 million, or 171%. This increase was primarily attributable to (i) increases in fair market value of the Company’s equity securities portfolio, investments in preferred stock and other convertible instruments and (ii) dividend income from the Company’s investments in preferred stock.

Equity in net loss of affiliates, which includes the Company’s pro-rata share of the net losses of its investments accounted for using the equity method of accounting, decreased $5.7 million, or 33%. This decrease was primarily due to the temporary redeployment of certain of the Company’s resources away from these joint venture programs towards supporting prospective new platforms and additional collaborations.

Conference Call and Webcast

The Company will host a conference call today Thursday, November 9th, at 5:30 PM ET to discuss the third quarter 2017 financial results and provide a general business update. The conference call may be accessed by dialing 1-888-317-6003 (Domestic US), 1-866-284-3684 (Canada), and 1-412-317-6061 (International) and providing the number 7741944 to join the Intrexon Corporation Call. Participants may also access the live webcast through Intrexon’s website in the Investors section at View Source