On January 6, 2022 Tiziana Life Sciences (Nasdaq: TLSA) ("Tiziana"), a biotechnology company enabling breakthrough immunotherapies via novel routes of drug delivery, reported that senior management will present at the Biotech Showcase Conference and be available for one-on-one meetings throughout the conference (Press release, Tiziana Life Sciences, JAN 6, 2022, View Source [SID1234598400]). The conference is being held virtually from January 10th -12th and also during January 17th -19th.

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Conference details can be found below:

Biotech Showcase

Date: January 10-12 and 17-19, 2022
Format: On-demand presentation (available January 7, 2022) and one-on-meetings
Registration: Click here

On January 7, 2022 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that Søren Bregenholt, Chief Executive Officer, will participate and host one-on-one investor meetings at the following upcoming investor conferences (Press release, Alligator Bioscience, JAN 7, 2022, View Source [SID1234598399]):

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H.C. Wainwright BioConnect Conference, US
Location: Virtual Event

Date: January 10-13

The presentation will be available online – January 10-13, 2022.

Please visit the conference linkto register and view the event.

Redeye Fight Cancer Seminar – Outlook 2022
Location: Virtual Event
Date: Thursday, January 20, 2022
Time: 9:00 – 16:00, company presentation schedule will be updated by Redeye closer to the event and will be live broadcasted at Fight Cancer Seminar – Outlook 2022 | Redeye for Redeye members.

All presentations will be available via a digital library, which is accessible to event participants only. Please contact the organizers to if you wish to attend or Julie Silber if you wish to schedule a meeting with Alligator.

On January 7, 2022 Nuvalent, Inc. (Nasdaq: NUVL), a biotechnology company creating precisely targeted therapies for clinically proven kinase targets in cancer, reported that the first patient has been dosed in ARROS-1, its Phase 1/2 clinical trial evaluating NVL-520 in patients with advanced ROS1-positive non-small cell lung cancer (NSCLC) and other advanced solid tumors (Press release, Nuvalent, JAN 7, 2022, https://www.nuvalent.com/nuvalent-announces-first-patient-dosed-in-arros-1-phase-1-2-clinical-trial-of-nvl-520-its-novel-ros1-selective-inhibitor/ [SID1234598395]). NVL-520, Nuvalent’s lead product candidate, is a novel ROS1-selective inhibitor designed to address the clinical challenges of emergent treatment resistance, CNS adverse events, and brain metastases that may limit the use of currently available ROS1 kinase inhibitors.

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"The initiation of patient dosing with NVL-520 in ARROS-1 is a significant milestone for Nuvalent as we transition from a preclinical to clinical stage company," said Christopher Turner, M.D., Chief Medical Officer of Nuvalent. "We designed NVL-520 with the goal of fulfilling a specific target product profile developed in collaboration with leading physician-scientists who are actively treating patients with ROS1-driven cancers today. We are encouraged by the preclinical data generated to date, which provide evidence that NVL-520 represents a differentiated ROS1-selective inhibitor with the potential to overcome the limitations of current tyrosine kinase inhibitor therapies and provide a new therapeutic option for patients in need."

ARROS-1 is a Phase 1/2, multicenter, open-label, dose-escalation and expansion trial evaluating NVL-520 as an oral monotherapy. The Phase 1 dose-escalation portion of the study is open and enrolling patients with advanced ROS1-positive solid tumors who have been previously treated with at least one prior ROS1 tyrosine kinase inhibitor (TKI) therapy, and will evaluate the overall safety and tolerability of NVL-520 as well as determine the recommended Phase 2 dose (RP2D), characterize the pharmacokinetic profile, and evaluate preliminary anti-tumor activity.

Once a safe and tolerable dose is determined as the RP2D, the trial is designed to transition directly into the Phase 2 multiple cohort expansion portion, which will evaluate the overall activity of NVL-520 in patients with advanced ROS1-positive NSCLC and other advanced solid tumors. The Phase 2 portion will examine several cohorts of patients based on the number and type of prior anti-cancer therapies they have received. The Phase 2 cohorts are designed to support potential registration in ROS1-positive patients with NSCLC who are kinase inhibitor-naïve and in those who have been previously treated with ROS1 kinase inhibitors.

"Nuvalent thoughtfully designed the ARROS-1 trial to support the goal of seamless acceleration from first-in-human dose-exploration of NVL-520 into Phase 2 cohorts that are structured to evaluate multiple opportunities for potential registration," said Darlene Noci, A.L.M., Senior Vice President of Product Development & Regulatory Affairs for Nuvalent. "Through parallel evaluation of NVL-520 in TKI-naïve and clearly defined cohorts of pre-treated patients, we aim to generate data to comprehensively evaluate NVL-520 throughout the treatment paradigm for ROS1-driven cancers."

In addition to NVL-520, Nuvalent is advancing a robust pipeline including the development of NVL-655 as a parallel lead program for the potential treatment of patients with ALK-positive NSCLC, along with multiple discovery-stage research programs.

"Our focus in 2022 is on establishing Nuvalent as an operationally efficient, clinical-stage biotech company with an active in-house R&D pipeline. We are on track for the planned IND submission for NVL-655 which we expect to enable the opening of our second Phase 1/2 clinical trial for enrollment in the first half of the year, and continue to plan for portfolio expansion with multiple internally discovered novel drug candidates," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "Through the prioritization of a pipeline of novel small molecules designed to overcome the dual challenges of kinase resistance and selectivity, we aim to deliver new medicines that may not only provide additional therapeutic options, but have the potential to advance earlier in the treatment paradigm and become best-in-class treatments for patients with cancer."

Nuvalent ended 2021 with $288.4 million in cash, cash equivalents and marketable securities (unaudited), which, based on its current operating plans, is expected to fund its operations into 2024.

About NVL-520

NVL-520 is a brain-penetrant ROS1-selective inhibitor designed to remain active in tumors that have developed resistance to currently available ROS1 inhibitors, including tumors with the prevalent G2032R resistance mutation and those with the S1986Y/F, L2026M, or D2033N resistance mutations. NVL-520 has been optimized for brain penetrance to potentially improve treatment options for patients with brain metastases. NVL-520 has been observed in preclinical studies to selectively inhibit ROS1 over the structurally related tropomyosin receptor kinase (TRK) family to potentially avoid TRK-related CNS adverse events seen with dual TRK/ROS1 inhibitors and drive more durable responses for patients with ROS1-mutant variants. NVL-520 is currently being investigated in the ARROS-1 study, a first-in-human Phase 1/2 clinical trial for patients with advanced non-small cell lung cancer (NSCLC) and other solid tumors.

On January 7, 2022 MorphoSys AG (FSE: MOR; NASDAQ: MOR) reported that preliminary Monjuvi(R) U.S. net product sales for the full year of 2021 and provided financial guidance for 2022 (Press release, MorphoSys, JAN 7, 2022, View Source [SID1234598394]).

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Preliminary Monjuvi (tafasitamab-cxix) U.S. net product sales are US$ 23.6 million (€ 20.5 million) for the fourth quarter and US$ 79.1 million (€ 66.9 million) for the full year of 2021. Fourth quarter and full year 2021 results will be published on March 16, 2022.

"We are pleased that many patients have benefitted from Monjuvi since launch and we expect to see continued growth in 2022," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "The updated structure of our 2022 financial guidance better reflects our business model and provides greater transparency to the investment community."

Full Year 2022 Financial Guidance:

Amounts in million 2022 Financial Guidance 2022 Guidance Insights
Monjuvi U.S. Net Product Sales US$ 110m to 135m 100% of Monjuvi U.S. product sales are recorded on MorphoSys’ income statement and related profit/loss is split 50/50 between MorphoSys and Incyte.
Gross Margin for Monjuvi U.S. Net Product Sales 75% to 80% 100% of Monjuvi U.S. product cost of sales is recorded on MorphoSys’ income statement and related profit/loss is split 50/50 between MorphoSys and Incyte.
R&D expenses € 300m to 325m 2022 growth over 2021 will be driven primarily by investment in ongoing pivotal phase-3 studies, excluding transaction/restructuring/other charges related to Constellation acquisition recorded in 2021.
SG&A expenses € 155m to 170m 51% to 56% of mid-point of SG&A expenses represents Monjuvi U.S. selling costs of which 100% are recorded in MorphoSys’ income statement. Incyte reimburses MorphoSys for half of these selling expenses.
For 2022, we anticipate a year-over-year decline in SG&A, excluding transaction/restructuring/other charges related to Constellation acquisition recorded in 2021.

Additional information related to 2022 Financial Guidance:

– Tremfya royalties will continue to be recorded as revenue without any cost of sales in MorphoSys’ income statement. These royalties, however, will not contribute any cash to MorphoSys as 100% of the royalties will be passed on to Royalty Pharma.

– MorphoSys anticipates receiving royalties for Minjuvi(R) sales outside of the U.S. Guidance for these royalties is not being provided as MorphoSys does not receive any sales forecasts from its partner Incyte.

– MorphoSys does not anticipate any significant cash-accretive revenues from the achievement of milestones in 2022. Milestones for otilimab are passed on to Royalty Pharma. Milestones from all other programs remain with MorphoSys at 100%.

– MorphoSys anticipates sales of commercial and clinical supply of tafasitamab outside of the U.S. to its partner Incyte. Revenue from this supply is recorded in the "Licenses, milestones and other" category in MorphoSys’ income statement. These sales result in a zero gross profit/margin. As such, MorphoSys does not provide guidance for these sales.

– While R&D expense is anticipated to grow year-over-year due to investments in three pivotal studies, the growth is partially being offset by the consolidation of research/discovery activities.

– SG&A expense guidance range reflects savings from synergies following the acquisition of Constellation and streamlined commercialization efforts.

– Anticipated foreign exchange (USD/EUR) to impact operating expenses (R&D and SG&A) negatively by approximately 3%.

About Monjuvi(R) (tafasitamab)
Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).
In the United States, Monjuvi(R) (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In Europe, Minjuvi(R) (tafasitamab) received conditional approval, in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials.

Minjuvi(R) and Monjuvi(R) are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi(R) in the U.S., and marketed by Incyte under the brand name Minjuvi(R) in the EU.

XmAb(R) is a registered trademark of Xencor, Inc.

On January 7, 2022 Alexion, AstraZeneca’s Rare Disease group, reported that it has entered into an exclusive global collaboration and licence agreement with Neurimmune AG for NI006, an investigational human monoclonal antibody currently in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) (Press release, AstraZeneca, JAN 7, 2022, View Source [SID1234598393]). NI006 specifically targets misfolded transthyretin and is designed to directly address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart, with the potential to treat patients with advanced ATTR-CM.

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Under the agreement, Alexion will be granted an exclusive worldwide licence to develop, manufacture and commercialise NI006.

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and high rate of fatality within four years from diagnosis.1 It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.2

Marc Dunoyer, Chief Executive Officer, Alexion, said: "With 30 years of experience in developing medicines for people with rare diseases, Alexion is uniquely positioned to advance innovative science for small patient populations who are frequently underdiagnosed. We look forward to applying this expertise to the development of NI006, which is designed to clear cardiac amyloid fibril deposits with the potential to improve cardiac function for patients living with advanced ATTR-CM, who are currently underserved by existing treatment options."

There is a significant unmet medical need for patients with various types and levels of severity of amyloidosis that may require multiple mechanisms of action to address those needs. NI006, an ATTR depleter, adds a novel and complementary approach to AstraZeneca and Alexion’s pipeline of investigational therapies focused on amyloidosis and strengthens our broader commitment to addressing cardiomyopathies that can lead to heart failure.

Financial considerations
Alexion will pay Neurimmune an upfront payment of $30m with the potential for additional contingent milestone payments of up to $730m upon achievement of certain development, regulatory and commercial milestones, as well as low-to-mid teen royalties on net sales of any approved medicine resulting from the collaboration.

Neurimmune will continue to be responsible for completion of the current Phase Ib clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Alexion will be responsible for further clinical development, manufacturing and commercialisation.

The transaction is expected to close following satisfaction of customary closing conditions and regulatory clearances.

Notes

Heart failure
Heart failure (HF) is a life-threatening chronic disease that prevents the heart from pumping sufficient levels of blood around the body. HF affects approximately 64 million people worldwide. HF remains as fatal as some of the most common cancers in both men (prostate and bladder cancers) and women (breast cancer).5 Chronic HF is the leading cause of hospitalisation for those over the age of 65 and represents a significant clinical and economic burden.6

AstraZeneca’s ambition is to be the leading company in HF, expanding from Forxiga today in heart failure with reduced ejection fraction (HFrEF), to the full HF spectrum including cardiomyopathies. AstraZeneca is investing in multiple investigational therapies with diverse mechanisms of action to address the spectrum of patient need in this area.

ATTR-CM
Cardiomyopathy due to ATTR is caused by aging or genetic mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium. In patients with ATTR-CM, both the mutant and wild type TTR protein builds up as fibrils in tissues, including the heart. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death.

Worldwide, there are an estimated 300,000-500,000 patients with ATTR-CM3,4; however, many of those patients remain undiagnosed.

NI006
NI006 is an investigational human monoclonal antibody that specifically targets misfolded transthyretin and is designed to directly address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart.