On February 4, 2020 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the fourth quarter and full-year ended December 31, 2019 and provided full-year 2020 financial expense guidance (Press release, Neurocrine Biosciences, FEB 4, 2020, View Source [SID1234553833]).

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"I am very pleased with the accomplishments of Neurocrine Biosciences this past year, including our quarterly and full year business results. We anticipate an exciting year ahead as we continue to build a leading global neuroscience-focused biopharmaceutical company," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "In 2020, we will continue to focus on educating healthcare providers, caregivers and patients to help even more people suffering from the debilitating movements caused by tardive dyskinesia. In addition, we look forward to helping patients with Parkinson’s disease with the anticipated approval of opicapone and the potential expanded use of elagolix to help women with uterine fibroids. By mid-2020, our activities position us to have three approved treatments in four indications and three additional ongoing pivotal studies."

Fourth Quarter and Full Year Net Product Sales Highlights:

INGREZZA net product sales for the fourth quarter and full year 2019 were $238 million and $753 million respectively, representing an increase of over 80% vs. prior period comparisons.

Continued strength in INGREZZA new patient additions in the fourth quarter.

End of fourth quarter 2019 days-on-hand channel inventory increased relative to the third quarter 2019 due to timing of quarter-end purchases resulting in an approximate $11 million benefit to net product sales.

Financial Highlights:

R&D investment increased in the fourth quarter of 2019 versus the fourth quarter of 2018 primarily as a result of the Company’s ongoing activities in Congenital Adrenal Hyperplasia studies and in gene therapy partially offset by prior year spending on the Tourette syndrome program.

SG&A investment increased in the fourth quarter of 2019 versus the fourth quarter of 2018, primarily as a result of the patient-focused disease state awareness campaign, "Talk About TD", and an increase in the Branded Pharmaceutical Drug, or BPD, fee expense.

IPR&D expense of $36 million in the fourth quarter of 2019 reflects the Company’s collaboration with Xenon Pharmaceuticals specific to NBI-921352 (XEN901) for epilepsy.

Fourth quarter of 2019 GAAP net income and diluted earnings per share (EPS) were $34 million and $0.35, respectively, compared to $18 million and $0.19, respectively, in the fourth quarter of 2018.

Fourth quarter of 2019 non-GAAP net income and diluted earnings per share (EPS) were $102 million and $1.05, respectively, compared to $38 million and $0.40, respectively, in the fourth quarter of 2018.

As of December 31, 2019, the Company had cash, cash equivalents and marketable securities totaling $970 million.

A reconciliation of GAAP to non-GAAP quarterly financial results can be found in Tables 3 through 5 at the end of this earnings release.

Recent Events

In December 2019, the Company entered into a license and collaboration agreement with Xenon, a clinical-stage biopharmaceutical company. Pursuant to the terms of the agreement, the Company gained an exclusive license to NBI-921352 (formerly referred to as XEN901), a clinical-stage selective Nav1.6 sodium channel inhibitor with potential in SCN8A developmental and epileptic encephalopathy (SCN8A-DEE) and other forms of epilepsy, including focal epilepsy. Upon filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in mid-2020, the Company intends to start a Phase II study in SCN8A-DEE patients in 2H 2020.

In January 2020, the Company announced an option agreement that was originally signed in 2019 with Idorsia granting the Company an option to license ACT-709478, a potent, selective, orally-active, and brain penetrating T-type calcium channel blocker, in clinical development for the treatment of a rare pediatric epilepsy. A Phase II study in a rare pediatric epilepsy is planned in 2H 2020 dependent upon IND application acceptance by the FDA in mid-2020.

Full-Year 2020 Financial Guidance

GAAP and Non-GAAP expense guidance range reflects increased investment in R&D programs including three registrational programs, meaningful investments across early stage programs including Voyager and Xenon collaborations, continued investment in INGREZZA and marketing costs associated with the anticipated launch of opicapone.

GAAP-only guidance includes approximately $100 million of share-based compensation and a $20 million expected milestone payment to BIAL connected with the expected approval of opicapone by the FDA during the second quarter. GAAP-only guidance does not include any other potential milestones or in-process research and development costs associated with current collaborations or future business development activities.

Conference Call and Webcast Today at 4:30 PM Eastern Time

Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 877-876-9173 (US) or 785-424-1667 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

About INGREZZA (valbenazine) Capsules

INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso, and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release from presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information

Contraindications

INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

Warnings & Precautions

Somnolence

INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation

INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Parkinsonism

INGREZZA may cause Parkinsonism in patients with tardive dyskinesia. Parkinsonism has also been observed with other VMAT2 inhibitors. Reduce the dose or discontinue INGREZZA treatment in patients who develop clinically significant parkinson-like signs or symptoms.

Adverse Reactions

The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.

On February 4, 2020 Gilead Sciences, Inc. (Nasdaq: GILD) reported its results of operations for the fourth quarter and full year 2019 (Press release, Gilead Sciences, FEB 4, 2020, View Source [SID1234553832]). Total revenues for the fourth quarter of 2019 were $5.9 billion compared to $5.8 billion for the same period in 2018. Net income for the fourth quarter of 2019 was $2.7 billion, or $2.12 per diluted share, compared to net income of $3 million, or $0.00 per diluted share, for the same period in 2018. Non-GAAP net income for the fourth quarter of 2019 was $1.7 billion, or $1.30 per diluted share, compared to $1.9 billion, or $1.44 per diluted share, for the same period in 2018.

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Full year 2019 total revenues were $22.4 billion, compared to $22.1 billion for 2018. Net income for 2019 was $5.4 billion, or $4.22 per diluted share, compared to $5.5 billion, or $4.17 per diluted share, for 2018. Non-GAAP net income for 2019 was $8.5 billion, or $6.63 per diluted share, compared to $8.7 billion, or $6.67 per diluted share, for

For the fourth quarter of 2019, compared to the same period in 2018, net income attributable to Gilead increased primarily due to the net favorable tax effects of intra-entity intangible asset transfers to different tax jurisdictions and an increase in net gains from equity securities. In addition, during the fourth quarter of 2019 and 2018, Gilead recorded pre-tax
________________________________
Non-GAAP financial information excludes acquisition-related, up-front collaboration and licensing, stock-based compensation and other expenses, fair value adjustments of equity securities and discrete tax charges or benefits associated with changes in tax related laws and guidelines. A reconciliation between GAAP and non-GAAP financial information is provided in the tables on pages 9 through 12.

impairment charges of $800 million and $820 million, respectively, related to in-process research and development (IPR&D) intangible assets acquired in connection with the acquisition of Kite Pharma, Inc. (Kite) and pre-tax write-downs of $500 million and $410 million, respectively, for slow moving and excess raw material and work in process inventory.
In addition to the factors noted above, the full year 2019, compared to the same period in 2018, was impacted by pre-tax up-front collaboration and licensing expenses of $3.92 billion related to Gilead’s global research and development collaboration agreement with Galapagos NV (Galapagos) in 2019.

Product Sales
Total product sales for the fourth quarter of 2019 were $5.8 billion, compared to $5.7 billion for the same period in 2018. Product sales for the fourth quarter of 2019 were $4.5 billion in the United States, $840 million in Europe and $440 million in other locations. Product sales for the fourth quarter of 2018 were $4.5 billion in the United States, $813 million in Europe and $398 million in other locations.
Total product sales in 2019 were $22.1 billion, compared to $21.7 billion in 2018. For 2019, product sales were $16.6 billion in the United States, $3.6 billion in Europe and $2.0 billion in other locations. For 2018, product sales were $16.2 billion in the United States, $3.7 billion in Europe and $1.8 billion in other locations.

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February 4, 2020

HIV product sales were $4.6 billion for the fourth quarter of 2019 compared to $4.1 billion for the same period in 2018. For 2019, HIV product sales were $16.4 billion compared to $14.6 billion in 2018. The increases were primarily driven by higher sales volume as a result of the continued uptake of Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg).

Chronic hepatitis C virus (HCV) product sales were $630 million for the fourth quarter of 2019 compared to $738 million for the same period in 2018. For 2019, HCV product sales were $2.9 billion compared to $3.7 billion in 2018. The declines were primarily due to lower average net selling price.

Yescarta (axicabtagene ciloleucel) generated $122 million in sales during the fourth quarter of 2019 compared to $81 million in 2018. For 2019, Yescarta sales were $456 million compared to $264 million in 2018. The increases were driven by a higher number of therapies provided to patients and the continued expansion in Europe.

Other product sales, which include products from chronic hepatitis B virus (HBV), cardiovascular, oncology and other categories, inclusive of Vemlidy (tenofovir alafenamide 25 mg), Viread (tenofovir disoproxil fumarate 300 mg), Letairis (ambrisentan 5 mg and 10 mg), Ranexa (ranolazine 500 mg and 1000 mg), Zydelig (idelalisib 150 mg), AmBisome (amphotericin B liposome for injection 50 mg/vial) and Cayston (aztreonam for inhalation solution 75 mg/vial), were $467 million for the fourth quarter of 2019 compared to $797 million for the same period in 2018. For 2019, other product sales were $2.3 billion compared to $3.1 billion in 2018. The decreases were expected and primarily due to declines in Ranexa and Letairis sales after generic entries in 2019.

For the fourth quarter of 2019, compared to the same period in 2018:

Cost of goods sold and non-GAAP cost of goods sold increased primarily due to higher inventory write-downs, partially offset by lower royalty expenses. During the fourth quarter of 2019 and 2018, Gilead recorded write-downs of $500 million and $410 million, respectively, for slow moving and excess raw material and work in process inventory primarily due to lower long-term demand for Gilead’s HCV products.

Product gross margin and non-GAAP product gross margin decreased primarily due to the factors noted above.
For the full year 2019, compared to the same period in 2018:

Cost of goods sold and non-GAAP cost of goods sold decreased primarily due to lower royalty expenses, partially offset by higher inventory write-downs. Costs of goods sold also decreased due to lower amortization expense related to intangible assets associated with Ranexa.

Product gross margin and non-GAAP product gross margin increased primarily due to changes in product mix and the factors noted above.

For the fourth quarter of 2019, compared to the same period in 2018:

R&D expenses decreased primarily due to lower up-front collaboration and licensing expenses, partially offset by higher personnel costs to support Gilead’s cell therapy business and increased investment in Gilead’s research projects. Gilead recorded impairment charges of $800 million in 2019 for the IPR&D intangible assets acquired in connection with the acquisition of Kite primarily related to the treatment of indolent non-Hodgkin lymphoma and $820 million in 2018 related to the KITE-585 program (an anti-B cell maturation antigen being evaluated for the treatment of multiple myeloma).

Non-GAAP R&D expenses increased primarily due to higher personnel costs to support Gilead’s cell therapy business and increased investment in Gilead’s research projects.

SG&A expenses and non-GAAP SG&A expenses increased primarily due to higher promotional expenses in the United States and expenses associated with the expansion of Gilead’s business in Japan.
For the full year 2019, compared to the same period in 2018:

R&D expenses increased primarily due to up-front collaboration and licensing expenses of $3.92 billion related to Gilead’s global research and development collaboration agreement with Galapagos, partially offset by lower stock-based compensation expense associated with Gilead’s acquisition of Kite. Furthermore, R&D expenses and non-GAAP R&D expenses increased primarily due to higher personnel costs to support Gilead’s cell therapy business.

SG&A expenses increased primarily due to promotional expenses in the United States and expenses associated with the expansion of Gilead’s business in Japan and China, partially offset by lower stock-based compensation expense associated with Gilead’s acquisition of Kite.

Non-GAAP SG&A increased primarily due to promotional expenses in the United States and expenses associated with the expansion of Gilead’s business in Japan and China.
Cash, Cash Equivalents and Marketable Debt Securities
As of December 31, 2019, Gilead had $25.8 billion of cash, cash equivalents and marketable debt securities compared to $31.5 billion as of December 31, 2018. During 2019, Gilead generated $9.1 billion in operating cash flow, paid $5.6 billion in connection with the global research and development collaboration agreement with Galapagos and equity investments in Galapagos, repaid $2.8 billion of principal amount of debt, paid cash dividends of $3.2 billion and utilized $1.7 billion on stock repurchases.

Full Year 2020 Guidance
Gilead provides its full year 2020 guidance below. Starting in 2020, Gilead will no longer regularly exclude stock-based compensation expense from its non-GAAP financial information. For comparability purposes, full year 2019 non-GAAP operating income and non-GAAP diluted earnings per share would have been $10.4 billion and $6.13, respectively, had stock-based compensation expense not been excluded.

Corporate, Product and Pipeline Updates for the Fourth Quarter, Including the Announcement of:
Viral Diseases

Licensing of The Rockefeller University’s portfolio of broadly neutralizing antibodies against HIV, including the two clinical-stage agents 3BNC117 and 10-1074.

Approval of Vosevi (sofosbuvir 400 mg/velpatasvir 100 mg/voxilaprevir 100 mg) by the China National Medical Products Administration for the treatment of chronic HCV infection in adults without cirrhosis or with compensated cirrhosis who have failed prior treatment with a direct-acting antiviral therapy.

Donation to the National AIDS Memorial to support relocation of The Aids Memorial Quilt to San Francisco, as well as related educational programs, under the stewardship of the National AIDS Memorial.

Presentation of data at The Liver Meeting, which included new data on Vemlidy evaluating its safety profile compared with tenofovir disoproxil fumarate in patients with chronic HBV infection.

Presentation of data at the 17th European AIDS Conference, which included:

96-week results from the DISCOVER trial, evaluating the safety and efficacy of Descovy (emtricitabine 200 mg/tenofovir alafenamide 25 mg) for HIV pre-exposure prophylaxis (PrEP), compared with Truvada for PrEP (emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg).

Data on investigational HIV-1 capsid inhibitor GS-6207 as a potential component of long-acting HIV therapy.

Findings from two Phase 3 studies evaluating the safety and efficacy of Biktarvy compared with dolutegravir-containing regimens for the treatment of HIV-1 infection in adults new to HIV therapy.
Inflammatory Diseases

Collaboration with Kyverna Therapeutics, Inc. to research and develop advanced cell therapies for the treatment of autoimmune disease.

Agreement with Eisai Co., Ltd. for the distribution and co-promotion of filgotinib in Japan, pending regulatory approval from the Japan Ministry of Health, Labor and Welfare (MHLW), for the treatment of rheumatoid arthritis (RA).

Submission of a New Drug Application under priority review to the U.S. Food and Drug Administration (FDA) for filgotinib for the treatment of adults with moderate-to-severe RA.

Presentation of data at the 2019 American College of Rheumatology/Association of Rheumatology Professionals Annual Meeting from the clinical research collaboration with Galapagos evaluating the efficacy and safety of filgotinib in adults with moderately-to-severely acute RA.
Oncology

European Medicines Agency’s validation of the marketing authorization application and submission of a Biologics License Application to the FDA for KTE-X19, an investigational chimeric antigen receptor (CAR) T cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL).

Collaboration with Kiniksa Pharmaceuticals, Ltd. to conduct a Phase 2, multicenter study of mavrilimumab, an investigational fully human monoclonal antibody that targets granulocyte macrophage colony stimulating factor receptor alpha, in combination with Yescarta in patients with relapsed or refractory large B-cell lymphoma.

The presentation of data at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, which included:

Long-term data from the ZUMA-1 trial of Yescarta in adult patients with refractory large B-cell lymphoma.

Positive results from ZUMA-2 Phase 2 study of KTE-X19, an investigational CD19 CAR T cell therapy, in adult patients with relapsed or refractory MCL.

Positive real-world data from ongoing post-marketing study evaluating the safety and efficacy of Yescarta in adult patients with relapsed or refractory large B-cell lymphoma.
Fibrotic Diseases

Topline results from the Phase 2 ATLAS study of combination and monotherapy investigational treatments in patients with bridging fibrosis (F3) and compensated cirrhosis (F4) due to nonalcoholic steatohepatitis (NASH).

Presentation of data at The Liver Meeting, which included new data showing potential for machine learning to advance understanding of NASH.

Collaboration with Glympse Bio, Inc. to determine clinical trial participants’ stage of disease at initial screening and to determine responses to study treatment in Gilead’s NASH clinical program.
Non-GAAP Financial Information
The information presented in this document has been prepared in accordance with U.S. generally accepted accounting principles (GAAP), unless otherwise noted as non-GAAP. Management believes non-GAAP information is useful for investors, when considered in conjunction with Gilead’s GAAP financial information, because management uses such information internally for its operating, budgeting and financial planning purposes. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of Gilead’s operating results as reported under GAAP. Non-GAAP measures may be defined and calculated differently by other companies in the same industry. A reconciliation between GAAP and non-GAAP financial information is provided in the tables on pages 9 through 12.

Conference Call
At 4:30 p.m. Eastern Time today, Gilead’s management will host a conference call and a simultaneous webcast to discuss the company’s fourth quarter and full year 2019 financial results and provide a business update. The live webcast of the call can be accessed at Gilead’s Investors page at View Source Please connect to the website at least 15 minutes prior to the start of the call to allow adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 877-359-9508 (U.S.) or 224-357-2393 (international) and dial the conference ID 9634129 to access the call. Telephone replay will be available approximately two hours after the call through 8:00 p.m. Eastern Time, February 6, 2020. To access the replay, please call 855-859-2056 (U.S.) or 404-537-3406 (international) and dial the conference ID 9634129. The webcast will be archived on www.gilead.com for one year.

On February 4, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) reported the initiation of an expanded access program (EAP) in the U.S. for tafasitamab, an investigational anti-CD19 antibody (Press release, MorphoSys, FEB 4, 2020, View Source [SID1234553831]). The EAP may provide access to tafasitamab for use in patients with relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) in combination with lenalidomide. Tafasitamab is an investigational medicine and its safety and efficacy have not been established.

According to the FDA, expanded access programs – sometimes called "compassionate use" – provide a pathway for a patient to receive an investigational medicine for a serious disease or condition. They are often made available when there are no comparable or satisfactory alternative therapies to treat the disease or condition; patient enrollment in clinical trials is not possible; potential patient benefit justifies the potential risk of treatment and providing the investigational medicine will not interfere with investigational trials that could support the medicine’s marketing approval for the treatment indication.

To qualify for the tafasitamab EAP, patients with r/r DLBCL need to meet the EAP inclusion/exclusion criteria that are aligned with the MorphoSys’ L-MIND study. Treatment of DLBCL patients in the EAP is recommended with tafasitamab in combination with lenalidomide according to the treatment schedule in L-MIND. The EAP will be available for a limited time while the FDA reviews MorphoSys’ Biologics License Application (BLA) for tafasitamab. Requests for expanded access to tafasitamab must be made by a U.S. licensed, treating physician.

The tafasitamab EAP will be administered by Clinigen Healthcare Ltd. Questions or inquiries regarding the tafasitamab EAP should be directed to [email protected].

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About Tafasitamab
Tafasitamab is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. MorphoSys is clinically investigating tafasitamab as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of tafasitamab in combination with lenalidomide in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). Based on interim data from L-MIND, in October 2017 the U.S. FDA granted Breakthrough Therapy Designation for tafasitamab plus lenalidomide in this patient population.

On February 4, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) reported the launch of an Expanded Access Program (EAP) in the USA for tafasitamab, an anti-CD19 antibody currently under development (Press release, MorphoSys, FEB 4, 2020, View Source [SID1234553831]). Under certain conditions, patients with relapsed or refractory diffuse large B-cell lymphoma (r / r DLBCL) can have access to tafasitamab in combination with lenalidomide via the EAP. Tafasitamab is an active ingredient in development (investigational product), the safety and efficacy of which have not been established.

According to the FDA, programs for expanded access , sometimes referred to as "compassionate use", offer patients an opportunity to gain access to an investigational medication for the treatment of a serious illness. They are often provided when there are no comparable or satisfactory alternative therapies to treat the disease, when the patient cannot be enrolled in a clinical trial, when the potential benefit to the patient justifies the potential treatment risk, and when it is made available of the investigational medicinal product does not affect studies that could support the marketing authorization of the drug for the corresponding treatment indication.

To be eligible for the Tafasitamab EAP, r / r DLBCL patients must meet the EAP inclusion / exclusion criteria that correspond to those in the MorphoSys L-MIND study. Treatment of DLBCL patients under the EAP is recommended with tamasitamab in combination with lenalidomide according to the L-MIND treatment plan. The EAP will be available for a limited time while the FDA is reviewing MorphoSys’s application for a Biologics License Application (BLA) for Tamasitamab. Applications for inclusion in the Tafasitamab EAP must be made by a medical doctor approved in the United States.

The Tafasitamab EAP is from Clinigen Healthcare Ltd. managed. Questions or inquiries regarding the Tafasitamab EAP should be directed to [email protected] .

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About tafasitamab
Tafasitamab is a humanized Fc-modified monoclonal antibody against CD19. In 2010, MorphoSys licensed Xencor, Inc.’s worldwide development and marketing rights for tafasitamab. Tafasitamab has an Fc part modified with the XmAb (R) technology, which is said to lead to a significant increase in antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), and thus to improve a key mechanism of tumor cell killing. MorphoSys is investigating tafasitamab as a therapeutic option for malignant B-cell disorders in a number of ongoing combination studies. An open phase 2 combination study (L-MIND study) examined the safety and efficacy of tafasitamab in combination with lenalidomide in patients with relapsed / refractory DLBCL who were not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) Question come. Based on preliminary data from L-MIND, the FDA granted therapeutic breakthrough status for tafasitamab plus lenalidomide in this patient population in October 2017.

On February 4, 2020 IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical stage biopharmaceutical company pioneering a novel class of immunotherapies, reported that clinical translational data supporting the mechanism of action of its lead compound, DPX-Survivac, will be presented during the 2020 ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium, being held on February 6 – 8, 2020 in Orlando, FL (Press release, IMV, FEB 4, 2020, View Source [SID1234553830]).

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"These translational data continue to validate the mechanism of action of our lead program in advanced ovarian cancer," said Frederic Ors, President and Chief Executive Officer at IMV. "We continue to believe DPX-Survivac may offer significant clinical utility and a potentially meaningful treatment option for patients in this setting, as well as in other hard-to-treat indications in which survivin is highly expressed. We look forward to reporting topline results from our Phase 1b/2 study evaluating DPX-Survivac in advanced ovarian cancer, in the first quarter of 2020."

As part of this analysis, the Company measured systemic immune responses, tumor immune infiltrates and clinical tumor response from pre- and post-treatment patient samples in connection with three Phase 1 and/or Phase 2 clinical studies, each evaluating DPX-Survivac alone or in a combination regimen in patients with platinum sensitive or resistant, advanced ovarian cancer. Highlights from these translational data include:

DPX-Survivac generated survivin-specific T cells in the blood of 80% of patients sampled

Clinical anti-tumor responses were correlated with increased infiltration of T cells into tumors following treatment with DPX-Survivac

DPX-Survivac induced enrichment in T cell, cytotoxic lymphocytes and B cell-specific signatures which correlate with clinical response

Antigen-specific T cells retained their functionality throughout the duration of treatment

DPX-Survivac is currently being evaluated in three Phase 2 studies in advanced ovarian cancer, relapsed/refractory diffuse large B-cell lymphoma and a basket trial of five solid tumors, all of which are expected to report topline results in the first half of 2020.

Poster Presentation Details:

Poster Title: DPX-Survivac, a novel T cell immunotherapy, induces robust T cell responses in advanced ovarian cancer with significant anti-tumor efficacy Presenter: Oliver Dorigo, M.D., Ph.D., Associate Professor of Obstetrics and Gynecology (Oncology), Stanford University Medical Center

Abstract Number: 6 – Poster Session A

Date and Time: Poster will be displayed all day on February 6, 2020

ASCO-SITC has published the official abstracts on its meeting website in advance of the Clinical Immuno-Oncology Symposium on February 3rd, 2020 at 5:00PM EST.

The final conference poster presentation will include additional data collected between the abstract submission on October 15, 2019 and the presentation itself. The poster will be available under Events, Webcasts and Presentations in the investors section of IMV’s website on the day of presentation.

About DPX-Survivac

DPX-Survivac is the lead candidate in IMV’s new class of targeted immunotherapies designed to elicit antigen-specific functional, robust and sustained de novo T cell response. IMV believes this mechanism of action (MOA) is key to generating durable solid tumor regressions. DPX-Survivac consists of five unique HLA-restricted survivin peptides formulated in IMV’s proprietary DPX drug delivery platform and known to induce a cytotoxic CD8+ T cell response against survivin expressing cancer cells.

Survivin, recognized by the National Cancer Institute (NCI) as a promising tumor-associated antigen, is broadly over-expressed in most cancer types and plays an essential role in antagonizing cell death, supporting tumor-associated angiogenesis and promoting resistance to chemotherapies. IMV has identified over 20 cancer indications in which survivin can be targeted by DPX-Survivac.

DPX-Survivac has received Fast Track designation from the U.S. Food and Drug Administration (FDA) as maintenance therapy in advanced ovarian cancer, as well as orphan drug designation status from the U.S. FDA and the European Medicines Agency (EMA) in the ovarian cancer indication.