On February 11, 2022 Nykode Therapeutics AS (Euronext Growth (Oslo): NYKD), a clinical-stage biopharmaceutical company dedicated to the discovery and development of vaccines and novel immunotherapies, reported the completion of patient enrollment in the Phase 2 study of VB10.16, Nykode’s lead cancer vaccine candidate, in combination with Roche’s checkpoint inhibitor atezolizumab for the treatment of advanced cervical cancer (Press release, Nykode Therapeutics, FEB 11, 2022, View Source [SID1234608011]). VB10.16 is a potentially first-in-class off-the-shelf therapeutic HPV16 cancer vaccine designed to induce strong HPV16 specific T cell responses for the treatment of HPV16-positive cancers.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are pleased to report the completion of patient enrollment in our ongoing Phase 2 trial of VB10.16, our wholly owned product candidate for the treatment of cervical cancer," said Michael Engsig, CEO of Nykode Therapeutics. "This is an important inflection point for Nykode as we advance our lead candidate through the clinic. After successfully enrolling 50 patients across trial sites in six countries, we look forward to reporting interim efficacy and safety data from the first patients in the first half of 2022. In parallel, we are exploring the possibility of evaluating VB10.16 in other HPV-driven cancers, including head and neck cancer."

VB C-02 is a multi-center, open-label Phase 2 trial of patients with advanced or recurrent, non-resectable HPV16-positive cervical cancer. Almost 20 European sites in six European countries were engaged in enrolling patients in the trial. Nykode has previously reported positive interim safety data from the trial.

Siri Torhaug, Chief Medical Officer of Nykode Therapeutics, commented: "Cervical cancer is a leading cause of death for women. It is an indication which continues to have a high unmet medical need with limited response to standard of care treatment in an advanced setting. We are very happy to have reached this important milestone for our lead cancer vaccine VB10.16 and look forward to continuing the development program. We are thankful for the patients, their families, the investigators and the trial site personnel who have made this trial possible."

Additional information about the Phase 2 trial is available at clinicaltrials.gov (NCT04405349).

On February 11, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the fourth quarter and fiscal year ended December 31, 2021 and provided financial guidance for 2022 (Press release, Neurocrine Biosciences, FEB 11, 2022, View Source [SID1234608010]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"As we exited last year with restored growth for INGREZZA, investments we are making this year will further accelerate our ability to help many more patients with tardive dyskinesia who remain undiagnosed and untreated. Additionally, we now have 12 clinical programs in mid-to-late-stage studies, many which will generate important data readouts over the next two years," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With a blockbuster product in INGREZZA, a novel and diverse pipeline, and a strong balance sheet, Neurocrine Biosciences is uniquely positioned to be a leading neuroscience-focused company."

Fourth Quarter and Fiscal 2021 INGREZZA Net Product Sales and Commercial Highlights:

INGREZZA fourth quarter and fiscal 2021 net product sales of $301 million and $1.1 billion, respectively
Fourth quarter 2021 INGREZZA net sales and total prescriptions grew 25% and 32%, respectively, vs. fourth quarter of 2020
Quarterly growth driven by record number of patients on therapy exiting 2021
Commercial expansion to better meet the needs of healthcare professionals across diverse sites of care on track for completion by the end of Q1 2022
Financial Highlights:

Fourth quarter 2021 GAAP net loss and loss per share of $7 million and $0.08, respectively, compared with fourth quarter 2020 GAAP net income and diluted earnings per share of $348 million and $3.58, respectively, primarily driven by a non-cash tax benefit of $296 million related to the release of substantially all of the Company’s valuation allowance against its deferred tax assets on December 31, 2020
Fourth quarter 2021 non-GAAP net income and diluted earnings per share of $4 million and $0.04, respectively, compared with $87 million and $0.89, respectively, for fourth quarter 2020
Differences in fourth quarter 2021 GAAP and non-GAAP financial results compared with fourth quarter 2020 driven by:
In-Process Research and Development (IPR&D) associated with a $100 million upfront fee paid to Sosei Heptares pursuant to our exclusive license agreement
Increased R&D expense in support of our expanded and advancing portfolio, including investment in in-licensed programs in epilepsy and psychiatry commencing at the end of 2019 and in mid-2020, respectively
Increased SG&A expense primarily due to increased investment in commercial initiatives including the launch of our INGREZZA direct-to-consumer advertising campaign, "TD Spotlight"
No cash payments for federal income tax were made in 2021 as the Company offset pre-tax income against previously benefited Federal net operating losses (NOLs)
At December 31, 2021, the Company had cash, cash equivalents and marketable securities of $1.3 billion
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release.

Recent Events:

In December 2021, the Company completed a strategic partnership with Sosei Heptares to expand its clinical psychiatry pipeline. The $100 million upfront fee paid to Sosei Heptares pursuant to our exclusive license agreement was expensed as IPR&D in fiscal 2021.
In the fourth quarter of 2021, the Company announced positive top-line data from the Phase III KINECT-HD study evaluating the efficacy, safety and tolerability of valbenazine in 120 adult patients with chorea in Huntington disease, also known as Huntington chorea. The study met the primary endpoint of reduction in severity of chorea. The Company plans to submit a supplemental new drug application for valbenazine for the treatment of Huntington chorea with the FDA in the second half of 2022.
In February 2022, the Company entered into a lease agreement for a four-building campus facility to be constructed in San Diego, California, pursuant to which a 6-year option for the construction of a fifth building and an option to purchase the entire campus facility in the future were also secured. Upon completion of construction, the Company expects to utilize the campus facility as its new corporate headquarters, which will consist of office space and R&D laboratories. The Company expects to begin subleasing its existing leased facilities beginning in 2023.

Based upon Federal NOL’s and tax credits, the Company expects to make minimal cash payments for Federal income tax beginning in the fourth quarter of 2022.

Key: VMAT2 = Vesicular Monoamine Transporter 2; CaV = Calcium Channel, Voltage-Gated; CSWS = Epileptic Encephalopathy with Continuous Spike and Wave During Sleep; M4= M4 Muscarinic Receptor; CFR1 = Corticotropin-Releasing Factor Type 1; AMPA = Alpha-Amino-3-Hydroxy-5-Methyl-4-Isoxazole Propionic Acid; GPR = Orphan G Protein Coupled Receptor; NaV1.6 = Sodium Channel, Voltage-Gated
Neurocrine Bioscience Partners: * Mitsubishi Tanabe Pharma Corporation has commercialization rights in East Asia;
** In-Licensed from Idorsia Pharmaceuticals; † In-Licensed from Sosei Group Corporation; ‡ Partnered with Takeda Pharmaceutical Company Limited; ∝ In-Licensed from Xenon Pharmaceuticals

Conference Call and Webcast Today at 8:00 AM Eastern Time
Neurocrine Biosciences will hold a live conference call and webcast today at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time). Participants can access the live conference call by dialing 800-895-3361 (US) or 785-424-1062 (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.

On February 11, 2022 Bio-Techne Corporation (NASDAQ: TECH) reported that Chuck Kummeth, President and Chief Executive Officer, will present at the 11th Annual SVB Leerink Global Healthcare Conference on Thursday, February 17, 2022, at 9:20 a.m. EST (Press release, Bio-Techne, FEB 11, 2022, View Source [SID1234608009]). A live webcast of the presentation can be accessed via the IR Calendar page of Bio-Techne’s Investor Relations website at View Source

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

On February 11, 2022 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biopharmaceutical company, reported its financial results for FY 2021 and provides an update on the strategic review of its CHC business (Press release, Ipsen, FEB 11, 2022, View Source [SID1234608007]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

David Loew, Chief Executive Officer, commented:

"The strong results in 2021 are aligned with our strategic intent, with improving levels of commercial execution reflecting in excellent performances from every major brand. As the replenishment of our pipeline gathered pace, it was an exciting year for business development and our key clinical programs. Furthermore, sharpening our focus on Specialty Care, I am pleased that we have entered into exclusive negotiations to divest our Consumer Healthcare business.

We will continue to grow our business in 2022, and beyond, through our core and innovative brands as we manage the gradual erosion of Somatuline while in addition, supporting growth through external innovation. Based on our strategy and our improved execution, we are pleased to update our mid-term outlook, underpinning the strength of Ipsen’s growth story built on our culture and an unrelenting focus on patients."

Exclusive negotiations to divest the Consumer Healthcare business
Following the decision of its Board of Directors held on 10 February 2022, Ipsen has entered into exclusive negotiations with Mayoly Spindler for the divestment of its global CHC business. This is a major step forward in the Company’s execution of its strategic roadmap presented in December 2020 towards building a more-focused Ipsen, centring on Specialty Care.

The combination of Ipsen’s and Mayoly Spindler’s respective CHC businesses will create a global consumerhealthcare platform with a critical size and the capacity to support its growth. The consideration for Ipsen’s CHC business represents an enterprise value of €350m, including an earnout contingent payment of €50m.

The proposed transaction will be submitted to the relevant employee-representation bodies and is expected to close before the end of Q3 2022, subject to regulatory approvals and customary closing conditions.

Delivering on strategy
Ipsen delivered successfully on its first year of the implementation of its strategy: Focus. Together. For patients and society. The key Specialty Care brands grew across all geographies, and alongside its expanding footprint in 2021, Ipsen began to invest in, and prepare for, a number of future launches.

It was a strong year of advancement of key R&D programs in Oncology, Rare Disease and Neuroscience, enhanced by the completion of seven external-innovation agreements. Ipsen also initiated a global program to drive efficiencies across the entire cost base, which started to deliver savings in 2021.

Further accomplishments were made in the Company Social Responsibility agenda, across the pillars of Employees, Communities and Environment. Ipsen increased the proportion of female leaders, now representing 42% of the full Global Leadership Team. Ipsen also announced the halving of absolute greenhouse-gas emissions of facilities and fleet (Scopes 1 and 2) by 2030, and will work closely with partners to deliver science-based Scope 3 emissions reductions by 2030.

Those achievements were the result of Ipsen’s evolving culture, underpinned by the strengthening of the Executive Leadership Team and the establishment of an asset-centric model. These successes provide excellent platforms for a culture of high performance, improved execution and a better and faster decision-making process.

Comparison of 2021 performance to guidance
Ipsen exceeded its full-year 2021 guidance, upgraded in October 2021:

FY 2021 guidance FY 2021 actuals
Total-sales growth Greater than 11.0%3 12.3%6
Core operating margin Around 34% 35.2%

Full-year 2022 guidance
Ipsen has set its financial guidance for FY 2022, excluding any contribution from the CHC business4:

Total-sales growth greater than 2.0%, at constant currency. Based on the level of exchange rates in January 2022, Ipsen anticipates an additional favorable impact of 2% from currencies
Core operating margin greater than 35.0% of total sales, excluding any potential impact of incremental investments from future external-innovation transaction
This guidance incorporates expectations for Somatuline of further launches of generic lanreotide in other countries in the E.U., as well as increased competition in the U.S.

Update of mid-term 2020-24 outlook
Ipsen today updates its outlook for 2020-24 to exclude any contribution from the CHC business5 and based on the strong performance delivered in 2021:

Total-sales 2020-24 compound annual growth rate between +4% and +6%6 at constant currency and assuming risk-adjusted potential additional indications
Continued commitment to invest in R&D supported by SG&A efficiencies:
Reduced SG&A expenses as a percentage of total sales, driven by further focus and optimization
Higher R&D expenses as a percentage of total sales, driven by the external-innovation strategy
To support the external-innovation strategy, Ipsen anticipates cumulative remaining firepower of €3.5bn by 2024, including the divestment of the CHC business. The calculation is based on net debt at 2.0 x EBITDA.

Palovarotene
In January 2022, Ipsen announced the first regulatory approval for palovarotene worldwide with Health Canada approving SohonosTM (palovarotene capsules). Ipsen plans to make a resubmission for palovarotene in fibrodysplasia ossificans progressiva to the U.S. FDA in H1 2022 and to continue the review process with the European Medicines Agency after the ‘clock-stop’ period. Discussions are progressing with other regulatory authorities around the world.

Basis of preparation for reported Financial Statements including the CHC business as continuing operations. 5 Earnings per share.
At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.
At CER, which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.
Assuming presentation of the CHC business as discontinued operations starting in 2022 and comparing to the FY 2021 operating performance excluding the contribution of the CHC business (as presented in the section ‘Core Measures excluding contribution from CHC’ thereafter).
Assuming presentation of the CHC business as discontinued operations starting in 2022 and comparing to the FY 2020 operating performance, excluding the contribution from the CHC business (as presented in the section ‘Core Measures excluding contribution from the CHC business’ thereafter).
Prior outlook, outlined in December 2020, included a total-sales 2020-24 compound annual growth rate of 2% to 5% at CER.

On February 11, 2022 Ichnos Sciences reported that innovation in medicine by developing potentially transformative biologic treatments in immuno-oncology (Press release, Ichnos Sciences, FEB 11, 2022, View Source [SID1234608006]). The company, currently a subsidiary of Glenmark Holding, SA, plans to pursue external financing following achievement of clinical proof of concept for its lead assets.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Headquartered in New York City, Ichnos has discovery and manufacturing operations at two sites in Switzerland. As a fully integrated biotechnology company with approximately 225 employees, Ichnos has strong capabilities in research, antibody engineering, CMC and clinical development of biotechnologies.

Ichnos is guided by an accomplished management team with experience developing immune cell engagers within the biopharmaceuticals industry, and is led by Cyril Konto, M.D., President and Chief Executive Officer.

The proprietary BEAT technology platform1 is the basis for Ichnos’ clinical-stage oncology pipeline. Using this technology coupled with the proprietary common light chain library, the company is developing novel multispecific immune cell engagers and modulators, with the goal of realizing its mission to provide breakthrough, potentially curative therapies that may extend and improve lives, writing a new chapter in healthcare.

ONCOLOGY PIPELINE
The first wave of Ichnos’ multispecific antibody pipeline consists of five programs targeting a range of hematologic malignancies and solid tumor indications through engagement of a broad spectrum of immune cells. The most advanced program is ISB 1342, a clinical-stage, potentially first-in-class bispecific antibody targeting CD38 and CD3, which is in Phase 1 for the treatment of relapsed/refractory multiple myeloma. OVERVIEW OF SELECT ONCOLOGY DRUG PRODUCT CANDIDATES ISB 1342 (CD38 X CD3 BISPECIFIC ANTIBODY)

• A Phase 1, open-label, dose-escalation, first-in-human study of ISB 1342 in patients with relapsed/refractory multiple myeloma is ongoing. + Enrollment of patients receiving a weekly dosing regimen is ongoing. + Number of sites participating in the study was expanded in the end of 2021 to enhance enrollment. New locations in the U.S. were added and 11 sites have opened for enrollment in France and are now recruiting subjects. + Clinical proof of concept in the ongoing study is anticipated in the middle of calendar year 2022.
• The primary objectives of the study are to: + Determine maximum tolerated dose and/or recommended Phase 2 dose of ISB 1342 (Part 1 dose escalation). Assess anti-myeloma activity of ISB 1342 according to the International Myeloma Working Group response criteria (Part 2 dose expansion).
• Preclinical data on ISB 1342 were presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting and EHA (Free EHA Whitepaper) 2021 Virtual Congress.
• ISB 1342 was granted Orphan Drug Designation for multiple myeloma by the FDA.
• The bulk drug substance is manufactured at the Ichnos site in La Chaux-de-Fonds, Switzerland. ISB 1442 (CD38 X CD47 BISPECIFIC ANTIBODY)
• This first-in-class 2+1 biparatopic bispecific antibody targeting CD38 x CD47 was generated using the BEAT 2.0 technology developed by scientists in Ichnos’ laboratories in Lausanne at the Biopole life sciences campus.
• ISB 1442 is designed to kill CD38-expressing tumor cells through inhibition of the CD47-SIRPα axis to increase antibody-dependent cellular phagocytosis (ADCP) and enhance antibody-dependent cellular cytotoxicity through complement dependent cytotoxicity (CDC) and antibody-dependent cell cytotoxicity (ADCC), enabled by the architecture and engineered Fc of the molecules.
• IND-enabling studies are proceeding, and IND filing is planned for second quarter of calendar year 2022. A Phase 1/2 first-in-human dose-finding study of ISB 1442 in relapsed/refractory multiple myeloma and other select hematologic malignancies is currently planned to start in the middle of 2022.
• Preclinical data on ISB 1442 were shared in an oral presentation at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting on December 11, 2021. These data, which may be viewed at this link, show: + Higher potency in vitro for ISB 1442 relative to daratumumab in CD38 high/low tumor models as measured by a multiple antibody-dependent mechanisms of action killing assay + Higher tumor growth inhibition for ISB 1442 than daratumumab in CD38 high preclinical in vivo models + Low on-target off-tumor binding with ISB 1442 compared to anti-CD47 mAb (5F9), resulting in lower red blood cell depletion and potentially a better therapeutic index than anti-CD47 bivalent monoclonal antibodies
• The first bulk drug substance batches to support IND filing and early clinical studies were manufactured at the Ichnos site in La Chaux-de-Fonds, Switzerland in 2021.Based on BEAT 2.0 technology, ISB 2001 trispecific antibody (TREATTM) represents a first-in-class potential treatment for hematologic malignancies and is designed to extend therapeutic durability.
• Identification and amino acid sequence lock of the top two candidates was achieved in 2021. Preclinical evaluation of in vivo efficacy, PK/PD correlation, additional biophysical properties description, late pharmacology studies and other attribute-defining studies are ongoing this quarter, and the results will inform the selection of the drug product candidate in the first half of calendar year 2022.
• Process development is ongoing at the Ichnos site in La Chaux-de-Fonds, Switzerland. AUTOIMMUNE DISEASES Ichnos has two monoclonal antibody drug product candidates addressing autoimmune diseases in the pipeline. The first, ISB 880, an anti-IL-1RAP antagonist, was licensed to Almirall, S.A. in December 2021, and the second, ISB 830 (telazorlimab), an OX40 antagonist that completed a Phase 2b study in moderate to severe atopic dermatitis in calendar year 2021, is in out-licensing discussions.

Both compounds have potential across a range of autoimmune diseases and are being out-licensed to enable a greater focus on oncology. Ichnos entered an exclusive global licensing agreement for ISB 880 in autoimmune diseases with Almirall in December 2021. Within the terms of the agreement, Almirall will assume full cost and responsibility for the global development and commercialization of the compound. Ichnos received an upfront payment of €20.8 million and the deal also includes development and commercial milestone payments and tiered royalties based upon future global sales.

• ISB 880, a fully human, high-affinity, monoclonal antibody blocking IL-1RAP signaling, has completed IND-enabling studies for patients with autoimmune diseases. The optimal antibody profile, the strong in vitro and in vivo data package, as well as toxicology, CMC, and clinical pharmacology plans will enable U.S. IND filing by Almirall in the first half of calendar year 2022.
• Blockade of IL-1RAP simultaneously abrogates multiple disease drivers among the IL-1 family of proinflammatory cytokine receptors, including IL-1R, IL-33R, and IL-36R, differentiating ISB 880 from single cytokine blockade therapies. These cytokines have been implicated in numerous autoimmune conditions, opening opportunities for ISB 880 to be positioned across broad disease indications.
• To date there is no IL-1RAP antagonist approved or under clinical development for autoimmune disease, positioning ISB 880 as a potential first-in-class therapeutic.
• Ichnos will retain rights for antibodies acting on the IL-1RAP pathway for oncology indications.The database for the ISB 830-204 Phase 2b clinical study in atopic dermatitis was locked in October 2021. This study, which was conducted in the U.S., Canada, Germany, Czech Republic, and Poland, had a randomized, controlled, multicenter design and assessed three doses and two dosing schedules of telazorlimab versus placebo in adults with moderate-to-severe atopic dermatitis (AD).
• Results from the double-blind portion of the study are summarized below. + Efficacy: The primary endpoint of EASI score, % change from baseline to Week 16, was achieved for the two highest doses of telazorlimab tested (300 mg and 600 mg q 2 weeks) versus placebo. Numerical improvements were also seen for the two higher dose arms of telazorlimab compared to placebo in the secondary endpoints of EASI75 and Investigator Global Assessment, but most of the differences were not statistically significantSafety: Telazorlimab was well tolerated.

The most commonly reported adverse events (>5%) were atopic dermatitis, nasopharyngitis, upper respiratory tract infection, and headache. One patient with pre-existing hypertension in the telazorlimab group died due to a presumed cardiovascular event during the treatment period. The investigator considered the death to be unrelated to the study drug.
• In addition to data from the 16-week primary analysis period, preliminary results from the open-label extension and follow-up period of this study, which was ongoing at the time, were presented at the 2021 Society for Investigative Dermatology Virtual Meeting and are accessible here. Of note: Clinical efficacy continued to improve after Week 16, with maximal impact achieved several weeks later + Reduction in AD disease activity was maintained after discontinuation of telazorlimab, through three months of follow-up
• A U.S. IND to conduct studies of telazorlimab in autoimmune diseases, including Rheumatoid Arthritis (RA), is active. • Licensing discussions are ongoing.