On February 6, 2020 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported that the company is scheduled to participate in a fireside chat at the Guggenheim Healthcare Talks Oncology Day on Thursday, February 13th, 2020, at 11:00 a.m. ET at the St. Regis Hotel in New York, NY (Press release, Magenta Therapeutics, FEB 6, 2020, View Source [SID1234553950]).

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A live webcast of the fireside chat can be accessed on the Magenta Therapeutics website at View Source The webcast replay will be available for 90 days following the event.

On February 6, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported the presentation of preclinical data from its ROR1 chimeric antigen receptor T cell (CAR-T) program at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) – Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting in Orlando, Florida (Press release, Oncternal Therapeutics, FEB 6, 2020, View Source [SID1234553949]). A copy of the poster presentation is available online at www.oncternal.com.

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In preclinical studies, anti-ROR1 CAR-T constructs were evaluated in an animal model of human leukemia. A single dose of anti-ROR1 CAR T-cells expanded in treated animals and the chimeric T cells trafficked to the disease sites. By week four, leukemia cells were cleared from major tissue reservoirs, including bone marrow, kidneys and spleen. CAR-T cell-treated animals survived longer than 90 days compared to 21 days for animals in control groups. The CAR-T cells were highly active and detected in mouse tissues more than two months after injection.

This research effort was led by Professor Thomas J. Kipps, M.D., Ph.D., and Charles Prussack, Pharm.D., Ph.D., at the University of California San Diego (UC San Diego) under a research grant from the California Institute of Regenerative Medicine (CIRM).

"It is exciting to see the potent preclinical activity of the ROR1 CAR-T cell therapy and its selectivity in targeting tumors," said Thomas Kipps, M.D., Ph.D., Professor of Medicine, Evelyn and Edwin Tasch Chair in Cancer Research, and Deputy Director of Research Operations at the UC San Diego Moores Cancer Center. "This CAR-T cell product utilizes the ROR1 binding domain derived from cirmtuzumab (UC-961), a clinical stage antibody that is currently being evaluated in patients with hematological malignancies and solid tumors. The challenges of CAR-T therapies include patient relapses due to the loss of target antigen and safety issues due to targeting of normal cells expressing the antigen. Harnessing cirmtuzumab’s specificity for ROR1 expressed on cancer cells has the potential to improve CAR-T efficacy and safety, and address the high unmet medical need for treating patients with aggressive cancers."

"We are encouraged by the preclinical results of this ROR1 CAR-T program and look forward to advancing it to clinical testing, initially for treating patients with hematological cancers, potentially in the fourth quarter of this year," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About ROR1 CAR-T

Oncternal Therapeutics is developing a ROR1-targeting (Receptor tyrosine kinase-like Orphan Receptor 1) chimeric antigen receptor T cell therapy (CAR-T) as a potential treatment for patients with aggressive hematological malignancies or solid tumors, in collaboration with the UC San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). The Company’s CAR-T program is based on the binding domain of cirmtuzumab, which is an investigational, potentially first-in-class monoclonal antibody targeting ROR1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of patients with chronic lymphocytic leukemia, or CLL, or mantle cell lymphoma, or MCL, and in an investigator-initiated Phase 1 clinical trial in combination with paclitaxel for the treatment of women with metastatic or unresectable breast cancer.

ROR1 is a potentially attractive target for cancer therapy because it is an onco-embryonic antigen – not usually expressed on adult cells, and its expression confers a survival and fitness advantage when reactivated and expressed by tumor cells. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab, that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Because the epitope of ROR1 recognized by cirmtuzumab appears to be restricted to tumor cells, a cirmtuzumab-based CAR-T may be selective in distinguishing cancer from normal tissues. Cirmtuzumab and ROR1 CAR-T cell therapy have not been approved by the U.S. Food and Drug Administration for any indication.

On February 6, 2020 Molecular Partners AG (SIX: MOLN), a clinical-stage biotech company that is developing a new class of drugs known as DARPin therapies, reported its unaudited financial results for 2019 and corporate highlights for the fourth quarter 2019 (Press release, Molecular Partners, FEB 6, 2020, View Source [SID1234553948]). The fourth quarter was marked by positive updated data on MP0250, refinement of the MP0250 development strategy, and noteworthy progress on the company’s pipeline of novel therapeutic designs.

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"We have delivered on our commitment to constructing groundbreaking therapeutic designs and advancing them rapidly to patients. Both the initiation of our phase 1 trial for MP0310 and the nomination of MP0317 as our second immuno-oncology candidate have underscored the potential of our novel therapeutic designs," said Patrick Amstutz, Ph.D., Chief Executive Officer of Molecular Partners. "As we embark on this new year, we look forward to FDA and EMA review of the regulatory submissions for abicipar, and working with our partner Allergan to deliver the first commercialized DARPin therapeutic to patients with neovascular AMD."

Abicipar: AAO Data highlight that vision gains were maintained throughout second year
In Q4 2019, two-year data from the CEDAR and SEQUOIA clinical studies of investigational Abicipar in patients with neovascular (wet) age-related macular degeneration (nAMD) were presented as a late-breaking oral presentation during Retina Subspecialty Day at the Annual Meeting of the American Academy of Ophthalmology (AAO). In the second year of these studies, four injections of Abicipar resulted in the maintenance of visual gains comparable to monthly ranibizumab.

Through week 104, patients received Abicipar 2 mg every 8-weeks or every 12-weeks or ranibizumab 0.5 mg every 4 weeks. At week 104 in the pooled Phase 3 data, the proportion of patients with stable vision was 93%, 90% and 94% in 8-week Abicipar; 12-week Abicipar and 4-week ranibizumab treatment regimens, respectively. This continuation of stable vision in year 2 further reinforces the ability of Abicipar to deliver consistent quarterly dosing for the majority of patients.

The pooled rate of new cases of intraocular inflammation in year two for patients who received Abicipar in the 8-and 12-week arms was 1.9%, which is similar to the ranibizumab arm of 1%.

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) are currently reviewing regulatory applications for Abicipar in patients with nAMD. The FDA is expected to take action on the BLA in mid-2020. A decision from the European Commission is expected in the second half of 2020.

Oncology: Updated data and Orphan Drug Designation for MP0250 in multiple myeloma
Data presented at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2019 indicate that MP0250 continues to show long-lasting and deepening responses across a variety of patients with multiple myeloma in the relapsed/refractory setting. MP0250 is a multi-DARPin candidate that targets hepatocyte growth factor (HGF) and vascular endothelial growth factor (VEGF), two prominent tumor escape pathways, and has the potential to improve sensitivity, or re-sensitize patients, to existing and emerging treatments.

The phase 2 trial for MP0250 in combination with bortezomib (Velcade) and dexamethasone in patients with multiple myeloma who have failed standard therapies is ongoing. The company announced at its R&D Day in December 2019 its intent to evaluate partnering opportunities for MP0250. In conjunction with this endeavor, the company announced it will not start the previously planned clinical trial investigating MP0250 in combination with an IMiD. This is aligned with the company’s corporate strategy to pursue combination data for the most relevant clinical combinations of MP0250, which would be more appropriately determined in collaboration with a partner. Additionally, the company announced in December 2019 that MP0250 has received Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).

Immuno-oncology: Phase 1 trial of MP0310 continues dose escalation
For MP0310 (AMG 506), the phase 1 MP0310-CP101 trial is ongoing and dose escalation is underway. The trial expects to enroll up to 54 patients at three sites in France to evaluate the safety, tolerability and pharmacokinetics of MP0310 in patients with locally advanced or metastatic solid tumors. Current clinical timelines are on track; the trial is expected to expand into additional combination cohorts in H2 2020. These combination trials will be conducted by Amgen.

Immuno-oncology: MP0317 (FAP x CD40) nominated as next IND candidate stemming from the company’s immuno-oncology DARPin toolbox
In Q4 2019, Molecular Partners nominated tumor-localized immune agonist MP0317 as the second DARPin protein in the company’s immuno-oncology pipeline. MP0317 comprises localizer (FAP) and stimulator (CD40) DARPin domains. It is designed to activate immune cells specifically in the tumor and not in the rest of the body, potentially delivering greater efficacy with fewer side effects.

Preclinical data demonstrated that the company’s multi-specific FAP x CD40 DARPin molecule induced FAP-dependent activation of B cells, dendritic cells and macrophages.

Oncology: Dosing ongoing in trial for MP0274 in HER2-positive solid tumors
Recruitment for the phase 1 trial for MP0274 and the dose escalation phase continues. MP0274 is a multi-DARPin product candidate being developed for the treatment of HER2-positive solid tumors. In preclinical trials MP0274 inhibits downstream signaling pathways, and directly kills HER2-addicted tumor cells through the induction of apoptosis. This represents a new and differentiated mode of action as compared to current standard of care antibodies.

Financial highlights: Net result and cash position on previous year’s level
In the financial year 2019, Molecular Partners recognized total revenues of CHF 20.4 million (2018: CHF 10.4 million) and incurred total expenses of CHF 57.6 million (2018: CHF 47.8 million). This led to an operating loss of CHF 37.2 million for 2019 (2018: Operating loss of CHF 37.4 million). The net financial result of CHF 0.4 million recorded in 2019 remained on the same level as in 2018. This resulted in a 2019 net loss of CHF 36.8 million (2018: Net loss of CHF 37.0 million).

The net cash used for operating activities in 2019 was CHF 1.2 million (2018: net cash used of CHF 42.5 million). Including time deposits, the cash and cash equivalents position decreased by CHF 3.9 million vs. year-end 2018 to CHF 95.1 million as of December 31, 2019 (December 31, 2018: CHF 99.0 million). Total shareholders’ equity stood at CHF 53.6 million as of December 31, 2019, a decrease of CHF 38.1 million (December 31, 2018: CHF 91.7 million).

As of December 31, 2019, the company employed 135 FTE, up 15% compared to year-end 2018. Approximately 85% of the employees are employed in R&D-related functions.

Business outlook and priorities
In 2020, Molecular Partners anticipates regulatory decisions by the FDA and EMA regarding the market launch of abicipar for patients with nAMD. The FDA is expected to take action on the BLA in mid-2020, and a decision from the European Commission is expected in the second half of 2020. Molecular Partners continues to work closely with its partner Allergan in the preparation and education of the market for the expected launch.

In immuno-oncology, recruitment of patients will continue in the phase 1 trial of MP0310 (AMG 506). Molecular Partners and Amgen expect to collect initial data from this trial in H2 2020.

In oncology, the company intends to continue to advance its phase 2 trial of MP0250 in patients with multiple myeloma in combination with Velcade and will pursue partnership opportunities for the MP0250 program. The company further plans in 2020 to establish dosing and clinical strategy for MP0274, as that therapeutic candidate concludes its phase 1 dose escalation.

Additionally, Molecular Partners will continue to advance its immuno-oncology research pipeline, specifically the MP0317, the CD3 DARPin T cell-engager platform and peptide-MHC programs.

Financial outlook 2020
For the FY 2020, at constant exchange rates, the company expects total expenses of CHF 60-70 million, of which around CHF 6 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciations. The increase versus the previous year is driven by the progress of the company’s pipeline as well as the budgeted growth of the company’s workforce. Capital expenditures in FY 2020 are expected to be approximately CHF 3 million.

This guidance is subject to the progress of the pipeline, mainly driven by manufacturing costs, the speed of enrollment of patients in clinical trials and data from research and development projects. No guidance can be provided with regard to net cash flow projections. Timelines and potential milestone payments for existing and potentially new partnerships are not disclosed.

Investor documentation of FY 2019 results
This FY 2019 press release as well as the FY 2019 results presentation are available on the investors section of the company’s website.

FY 2019 results presentation, conference call and audio webcast
Molecular Partners will hold the FY 2019 results presentation in its headquarters in Zurich-Schlieren on February 06, 2020, 2:00pm CET (1:00pm GMT, 8:00am EST). For those who are unable to participate in the live event, the company provides conference call and audio webcast facilities.

In order to register for the FY 2019 conference call, please dial the following numbers approximately 10 minutes before the start of the presentation:

Switzerland / Europe +41 (0) 58 310 5000

UK +44 (0) 203 059 5862

USA +1 (1) 631 570 5613

Participants will have the opportunity to ask questions after the presentation.

Audio webcast
The FY19 results presentation will be webcast live and will be made available on the company’s website under the Investors section. The replay will be available for 90 days following the presentation.

Financial Calendar
March 20, 2020 Expected Publication of Annual Report 2019
April 29, 2020 Annual General Meeting
May 7, 2020 Interim Management Statement Q1 2020
August 26, 2020 Publication of Half-year Results 2020 (unaudited)
October 29, 2020 Interim Management Statement Q3 2020
View Source

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates can engage more than five targets, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics. The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapeutics have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology and is advancing a proprietary pipeline of DARPin drug candidates in oncology and immuno-oncology. The most advanced global product candidate in partnership with Allergan is abicipar, a molecule for which phase 3 data have been filed to the respective regulators in both the US and in Europe. Several DARPin molecules for various ophthalmic indications are also in preclinical development. The most advanced DARPin therapeutic candidate wholly owned by Molecular Partners, MP0250, is in phase 2 clinical development for the treatment of hematological tumors. MP0274, the second-most advanced DARPin candidate owned by Molecular Partners, binds to Her2 and inhibits downstream signaling, which leads to induction of apoptosis. MP0274 is currently in phase 1. The company’s lead immuno-oncology product candidate MP0310 is a FAP x 4-1BB multi-DARPin therapeutic candidate designed to locally activate immune cells in the tumor by binding to FAP on tumor stromal cells (localizer) and co-stimulating T cells via 4-1BB (immune modulator). Molecular Partners has closed a collaboration agreement with Amgen for the exclusive clinical development and commercialization of MP0310. The molecule has entered in phase 1 of clinical development in H2 2019. MP0317 (FAP x CD40), the second tumor-localized immune agonist stemming from the company’s "I/O toolbox", has been nominated as next DARPin protein in Molecular Partners’ immuno-oncology pipeline. Molecular Partners is further advancing a growing preclinical and research pipeline in immuno-oncology and additional development programs such as novel therapeutic designs to target peptide-MHC complexes. DARPin is a registered trademark owned by Molecular Partners AG.

On February 6, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three and 12 months ended December 31, 2019, and provided an operating forecast and program updates (Press release, Ligand, FEB 6, 2020, View Source [SID1234553947]). Ligand management will host a conference call and webcast with accompanying slides today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

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"Ligand made tremendous progress during 2019 in areas that will drive our future success, including new license agreements, outstanding revenue performance driven by record revenue for Captisol and Kyprolis royalties, expansion of our proprietary technology platforms and continued investment in internal programs," said John Higgins, Chief Executive Officer of Ligand. "Last year we entered into nine OmniAb licensing transactions, reported positive Phase 1 results with Captisol-enabled Iohexol, bolstered our technology assets with the acquisition of an antigen design company, and advanced five internal immuno-oncology programs."

"Partners secured regulatory approvals during the year and we now have 13 partnered products contributing to royalty revenues, with as many as eight more potential approvals over the next three years. The first of 12 OmniAb programs currently in human trials entered pivotal testing in 2019."

"From a financial perspective, revenues exceeded the guidance we introduced last March after we monetized our Promacta assets for $827 million. That transaction was transformative for Ligand and provided significant cash for M&A activities and share repurchases. Over the past 18 months we have retired close to 25% of our outstanding shares through open-market repurchases which, all other things being equal, would result in future cash flow and profits per share increasing more than 30% given the new lower share count."

Higgins concluded, "As we move into 2020, we believe Ligand is well-positioned as a financial growth company driven by innovative technologies that enable partners to develop drugs. We are optimistic about our outlook, specifically in terms of EBITDA margin expansion, earnings growth and cash flow. For 2020 we forecast 14% organic revenue growth and 35% organic growth in adjusted diluted EPS, after factoring in the divestiture of Promacta in early 2019."

Fourth Quarter 2019 Financial Results

Total revenues for the fourth quarter of 2019 were $27.0 million, compared with $59.6 million for the same period in 2018. Royalties in the fourth quarter of 2019 were $11.0 million and primarily consisted of royalties from Kyprolis and EVOMELA. Royalties in the fourth quarter of 2018 were $40.2 million and included $31.0 million in royalties from Promacta, which was sold to Royalty Pharma as of March 6, 2019 for $827 million. Ligand did not receive any Promacta royalties in the fourth quarter of 2019 and will not receive any Promacta royalties going forward. Material sales were $7.1 million, compared with $10.1 million for the same period in 2018 due to the timing of Captisol purchases for use in clinical trials and commercial products. License fees, milestones and other revenues were $8.8 million, compared with $9.3 million for the same period in 2018.

Cost of material sales was $1.9 million for the fourth quarter of 2019, compared with $3.0 million for the same period in 2018. Amortization of intangibles was $6.3 million, compared with $3.5 million for the same period in 2018, with the increase due to accelerated amortization of the glucose receptor antagonist (GRA) asset. Research and development expense was $18.7 million, compared with $8.8 million for the same period of 2018, with the increase due to non-cash amortization of the upfront investments in the Palvella and Novan programs. General and administrative expense was $10.3 million, compared with $11.2 million for the same period in 2018, which included Vernalis acquisition-related expenses.

Net loss for the fourth quarter of 2019 was $(7.4) million, or $(0.43) per share, compared with net loss of $(42.5) million, or $(2.02) per share, for the same period in 2018. The net loss for both periods was impacted by a non-cash unrealized change in the value of Ligand’s investment in Viking Therapeutics of $8.5 million and $(74.0) million, respectively. Adjusted net income for the fourth quarter of 2019 was $12.9 million, or $0.71 per diluted share, compared with adjusted net income of $39.0 million, or $1.70 per diluted share, for the same period in 2018. See the table below for a reconciliation of net income (loss) to adjusted net income.

As of December 31, 2019, Ligand had cash, cash equivalents and short-term investments of approximately $1.0 billion. During the fourth quarter of 2019 Ligand used approximately $82 million in cash to repurchase approximately 760,000 shares.

Full Year 2019 Financial Results

Total revenues for 2019 were $120.3 million, compared with $251.5 million for 2018. Royalties were $47.0 million, compared with $128.6 million for 2018. Royalties for 2019 primarily consisted of royalties from Promacta, Kyprolis and EVOMELA and do not include contribution from Promacta after March 6, 2019, whereas 2018 royalties included the full year of Promacta royalties. Material sales were $31.5 million, compared with $29.1 million for 2018 due to the timing of Captisol purchases for use in clinical trials and commercial products. License fees, milestones and other revenues were $41.8 million, compared with $93.8 million for 2018, which included a $47 million payment from WuXi Biologics to amend its OmniAb platform license agreement as well as a $20 million upfront payment upon the licensing of Ligand’s GRA program.

Cost of material sales was $11.3 million for 2019, compared with $6.3 million for 2018, with the increase due primarily to higher sales and mix of Captisol sales in 2019. Amortization of intangibles was $16.9 million, compared with $15.8 million for 2018. Research and development expense was $55.9 million, compared with $27.9 million for 2018, with the increase due to costs associated with the VDP research team and non-cash amortization of the upfront investments in the Palvella and Novan programs. General and administrative expense was $41.9 million, compared with $37.7 million for 2018, with the increase due to costs associated with recent acquisitions and non-cash share-based compensation expense.

Net income for 2019 was $629.3 million, or $31.85 per diluted share, compared with net income of $143.3 million, or $5.96 per diluted share, for 2018. Net income for 2019 was impacted by an after-tax gain of approximately $642.6 million on the sale of Ligand’s Promacta license to Royalty Pharma. Adjusted net income for 2019 was $61.0 million, or $3.09 per diluted share, compared with adjusted net income of $166.9 million, or $7.15 per diluted share, for 2018.

2020 Financial Guidance

Ligand is providing guidance for 2020 with total revenues expected to be approximately $121 million, which includes royalties of approximately $38 million, material sales of approximately $35 million and license fees and milestones of approximately $48 million. Ligand notes that with total revenues of $121 million, adjusted earnings per diluted share would be approximately $3.40. This compares to 2019 adjusted revenue of $106.1 million and adjusted EPS of $2.52, excluding the impact of Promacta in 2019.

Fourth Quarter 2019 Highlights

Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

On December 10, Amgen announced additional results from the primary analysis of the Phase 3 CANDOR study evaluating Kyprolis in combination with dexamethasone and DARZALEX (daratumumab) compared to Kyprolis and dexamethasone alone in patients with relapsed or refractory multiple myeloma. The data were presented in a late-breaking abstract session at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (ASH) (Free ASH Whitepaper).
Additional Pipeline and Partner Developments

Viking Therapeutics, Inc. announced the initiation of a Phase 2b clinical trial of VK2809, its novel liver-selective thyroid hormone receptor beta agonist, in patients with biopsy-confirmed non-alcoholic steatohepatitis.
Palvella Therapeutics, Inc. announced that the Phase 3 pivotal portion of the seamless Phase 2/3 VALO study of PTX-022 (QTORIN 3.9% rapamycin anhydrous gel) for the treatment of patients with Pachyonychia congenita had commenced.
Sage Therapeutics launched ZULRESSO (brexanolone) Injection, the first and only treatment specifically indicated for postpartum depression. ZULRESSO utilizes Captisol in its formulation.
Sage announced the investigational new drug (IND) application for SAGE-689, a potential therapy for disorders associated with GABA hypofunction, was cleared by U.S. FDA and Sage expects to commence dosing in a Phase 1 clinical trial in healthy volunteers in 2020.
Retrophin, Inc. announced new data from the Phase 2 DUET study examining the impact of sparsentan on quality of life in patients with focal segmental glomerulosclerosis, at the American Society of Nephrology Kidney Week 2019.
Marinus Pharmaceuticals, Inc. announced that results from its Phase 2 trial of ganaxolone in refractory status epilepticus were presented at the Neurocritical Care Society 17th annual meeting.
Verona Pharma plc announced that it has randomized the last patient in its Phase 2b dose-ranging study evaluating the effect of nebulized ensifentrine as an add-on to treatment with a long-acting bronchodilator in patients with moderate-to-severe chronic obstructive pulmonary disease.
Aptevo announced that OmniAb-derived APVO436 is being evaluated in a Phase 1/1b clinical study in patients with acute myeloid leukemia and high-grade myelodysplastic syndrome. Aptevo expects to report ongoing progress from this study over the next several quarters as clinical data emerge.
Daiichi Sankyo announced positive results from the ESAX-DN Phase 3 study in Japan of esaxerenone in patients with diabetic nephropathy in a late-breaking poster presentation at the American Society of Nephrology Kidney Week 2019.
Immunovant announced that it initiated dosing in ASCEND-GO 2, a multicenter, randomized, masked, placebo-controlled Phase 2b clinical trial evaluating IMVT-1401 in patients with moderate-to-severe active Graves’ ophthalmopathy. IMVT-1401 is a fully human monoclonal antibody that selectively binds to and inhibits the neonatal Fc receptor, and is designed to be delivered by subcutaneous injection.
Aldeyra Therapeutics announced positive topline results from Part 1 of its Adaptive Phase 3 RENEW trial of topical ocular reproxalap in patients with dry eye disease.
Marinus Pharmaceuticals, Inc. announced additional data from its open-label, dose-finding Phase 2 study evaluating intravenous ganaxolone in patients with refractory status epilepticus. The results were presented at the American Epilepsy Society annual meeting.
CStone Pharmaceuticals announced that CS1001, its anti-PD-L1 antibody, demonstrated promising antitumor activity with a complete response rate of 33.3% and a good safety profile in patients with relapsed or refractory extranodal natural killer/T-cell lymphoma.
Sanofi presented pivotal data from its Phase 3 study of sutimlimab in patients with cold agglutinin disease at ASH (Free ASH Whitepaper) in a late-breaker session.
Business Development and Corporate Highlights

Ligand presented positive results from its Phase 1 clinical trial of its Captisol-enabled (CE) Iohexol program at American Society of Nephrology Kidney Week 2019. The CE-Iohexol program was established in January 2018 to develop a Captisol-enabled, next-generation contrast agent for diagnostic imaging with a reduced risk of renal toxicity.
Ligand announced a worldwide OmniAb license agreement with Sanofi under which Sanofi will be able to use Ligand’s full OmniAb antibody discovery platform including OmniRat, OmniFlic, OmniMouse, OmniChicken and OmniClic, in addition to Ligand’s patented antigen technology.
Ligand acquired Ab Initio for $12 million. Ab Initio is an antigen-discovery company based in South San Francisco, California. Antigen design and preparation are the first steps necessary for the discovery of therapeutic antibodies.
Ligand announced that two members of its Board of Directors, Nancy Gray and Sarah Boyce, had been named to WomenInc. magazine’s 2019 Most Influential Corporate Directors list.
Ligand focused on adopting and implementing policies and practices aimed at improving its environmental sustainability, positively impacting its social community and maintaining and cultivating good corporate governance. By focusing on such environmental, social and governance (ESG) policies and practices, Ligand believes it can effect a meaningful, positive change in its community and maintain its open, collaborative corporate culture. Ligand expects to continue its proactive shareholder engagement and to refine its ESG policies and practices in 2020.
Use of Non-GAAP 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 share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, amortization of commercial license and other economic rights, changes in contingent liabilities, acquisition and integration costs, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, unissued shares relating to its Senior Convertible Notes, gain on the sale of Promacta and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release.

However, other than with respect to total revenues, the Company only provides financial 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, changes in the market value of its investments in public companies, share-based compensation expense and effects of any discrete income tax items. 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.

Conference Call and Webcast

Ligand management will host a conference call and webcast with accompanying slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate in the call via telephone, please dial (833) 591-4752 from the U.S. or (720) 405-1612 from outside the U.S., using the conference ID 1150048. To participate in the call via live or replay webcast, a link is available at www.ligand.com. The conference call slides are available here.

On February 6, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported that it will report fourth quarter and full year 2019 financial results on Thursday, February 13, 2020 (Press release, Karyopharm, FEB 6, 2020, View Source [SID1234553946]). Karyopharm’s management team will host a conference call and audio webcast at 8:30 a.m. ET on Thursday, February 13, 2020, to discuss the financial results and other company updates.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 4367549. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.