On January 5, 2023 QIAGEN (NYSE:QGEN; Frankfurt Prime Standard: QIA) reported an exclusive strategic partnership with California-based population genomics leader Helix to advance companion diagnostics for hereditary diseases (Press release, Qiagen, JAN 5, 2023, View Source [SID1234625909]).

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As the development of precision medicines accelerates, so does the need for companion diagnostics devices and tests detecting clinically relevant genetic abnormalities. These diagnostics help guide clinical decision-making by identifying patients most likely to benefit – or be at increased risk – from a particular therapeutic product. Principally used in oncology to date, companion diagnostics that employ whole exome sequencing are widely believed to have great potential in hereditary disease areas such as cardiovascular, metabolic, neuro-degenerative, and auto-immune diseases.

Under the agreement announced today, QIAGEN will be the exclusive marketing and contracting partner in the U.S. for Helix’s companion diagnostic services. The partnership will leverage the Helix Laboratory Platform, which was granted the first-ever U.S. Food & Drug Administration de novo class II authorization for a whole exome sequencing platform. This brings a new innovative solution to biopharmaceutical customers in support of hereditary disease therapies, complementing solutions both companies already provide such as Helix’s population genomics programs and QIAGEN’s diagnostic expertise, QIAseq Human Exome Kits and global reach supporting research initiatives outside the U.S.

A pioneer in precision medicine, QIAGEN has more than 30 master collaboration agreements with global pharmaceutical and biotechnology companies to develop and commercialize companion diagnostic tests for their drug candidates. QIAGEN’s companion diagnostic offerings encompass technologies from next-generation sequencing (NGS) to polymerase chain reaction (PCR) and digital PCR (dPCR), sample types from liquid biopsy to tissue, and disease areas from cancer to Parkinson’s – including 11 FDA-approved PCR based companion diagnostics and a collaboration with Neuron23 announced in September 2022 to develop an NGS-based companion diagnostic for a novel Parkinson’s disease drug.

"This partnership represents another step toward bringing the power of companion diagnostics to hereditary diseases by powering Helix’s leading products with QIAGEN’s extensive pharma and biopharma relationships, NGS capabilities, and global regulatory expertise," said Thierry Bernard, Chief Executive Officer of QIAGEN. "Access to a genomic database can help researchers find patients with particular biomarker signatures almost instantaneously, making trial recruitment a matter of months instead of years."

"Helix’s technology and regulatory capabilities coupled with QIAGEN’s well-established worldwide companion diagnostic development capabilities results in a powerful partnership that will allow us to expand critical access to genomic testing for both patients and providers working daily to fight life-threatening conditions," said Helix CEO and Co-Founder, James Lu, MD, PhD. "QIAGEN’s mission to fill the clinical gap for patient access and provide technology platform options to address the needs of physicians to target treatment with genomic medicine is well in line with our mission."

Helix has built an end-to-end platform that enables health systems, life sciences companies, and payers to advance genomic research and accelerate the integration of genomic data into clinical care. Helix has partnered with leading health systems to enable population genomics programs of at least 100,000 patients each across the U.S. These programs dramatically accelerate patient identification and recruitment for clinical trials in hereditary diseases such as Parkinson’s, cardiovascular or inflammatory disease like non-alcoholic steatohepatitis (NASH), power real-world data (RWD) or real-world evidence (RWE) services and insights, and bring deep genetic expertise and methodologies to examine sub-cohorts within both drug discovery and a clinical trial.

On January 5, 2023 Nouscom, a clinical stage immuno-oncology company developing off-the-shelf and personalized immunotherapies, reported that it has entered into a clinical trial collaboration and supply agreement with MSD (Merck & Co., Inc., Rahway, NJ, USA), to evaluate Nouscom’s wholly-owned lead candidate, NOUS-209, in combination with
MSD’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) versus KEYTRUDA alone in randomized Phase 2 trials in patients with Mismatch Repair/Microsatellite Instable High (dMMR/MSI-H) Metastatic Colorectal Cancer (CRC) (Press release, NousCom, JAN 5, 2023, View Source;utm_medium=rss&utm_campaign=nouscom-announces-clinical-trial-collaboration-and-supply-agreement-with-msd-to-evaluate-nous-209-in-combination-with-keytruda-pembrolizumab-in-a-phase-2-randomized-trials-in-dmmr-msi-high-met [SID1234625906]). NOUS-209 is an off-the-shelf immunotherapy targeting 209 specific, shared neoantigens found in dMMR/MSI-H unresectable or metastatic gastric, colorectal and gastro-esophageal junction tumors.

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The Phase 2 study includes two cohorts in dMMR/MSI-H unresectable and metastatic CRC assessing the efficacy and safety of NOUS-209 in combination with pembrolizumab at multiple active sites across Europe and the US (NCT04041310).

1. A randomized cohort enrolling patients who are eligible for first line treatment, randomized

2:1 to NOUS-209 plus pembrolizumab versus pembrolizumab alone;

2. A single arm cohort enrolling patients who have stopped responding to previous anti-PD1 treatment.

Dr Marina Udier, Chief Executive Officer of Nouscom, added: "We are thrilled to collaborate with MSD, a global leader in immuno-oncology, and to work with their highly experienced and talented clinical development team, allowing us to accelerate the ongoing US and EU Phase 2 trial with NOUS-209. Based on our published and presented Phase 1 clinical data1,2 we see great potential of this combination approach in addressing the unmet medical need in these patients.
We look forward to presenting preliminary data in 2023."

Under the terms of the agreement, MSD will supply KEYTRUDA. Nouscom retains all worldwide commercial rights to NOUS-209.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

References

1. ASCO (Free ASCO Whitepaper) Presentation: First clinical and immunogenicity results including all subjects enrolled in a phase I study of NOUS-209, an off-the-shelf immunotherapy, with pembrolizumab, for the treatment of tumors with a deficiency in mismatch repair/microsatellite instability (dMMR), Professor Marwan G. Fakih, M.D.

2. A.M. D’Alise et al. Adenoviral Based-Vaccine Promotes Neoantigen Specific CD8+ T Cell Stemness And Tumor Rejection, Science Translational Medicine; 14, 657, 2022

About NOUS-209

NOUS-209 is an off-the-shelf cancer immunotherapy for Microsatellite Instable High (MSI-H) tumors. MSI-H tumors are characterized by a defective DNA mismatch repair system, which generates highly immunogenic neoantigens called frame shift peptides (FSP) that are not present in healthy tissue.

NOUS-209 encodes for 209 shared FSP neoantigens, selected by Nouscom’s proprietary GENESIS (GE(netic)NE(oantigen)S(election)I(n)S(ilico)) algorithm. In published prospective validation studies, approximately 50 of the 209 neoantigens are expressed in any one patient’s tumor. These FSPs are cloned into Nouscom’s heterologous prime / boost viral vector platform of a Great Ape Adenoviral (GAd) and Modified Vaccinia Ankara (MVA) and potently generate FSP neoantigen specific CD8+ T cells, which have been shown to successfully infiltrate tumor microenvironments to exert anti-tumor activity.

NOUS-209 is being investigated in multi-center EU and US Phase 2 randomized clinical trials in patients with dMMR/MSI-H unresectable and metastatic colorectal cancer (CRC) (NCT04041310) in combination with checkpoint inhibitors (CPI) versus CPI alone and in patients who have stopped responding to previous anti-PD1 and other approved CPI therapies.

On January 5, 2023 Protara Therapeutics presented its corporate presentation (Press release, Protara Therapeutics, JAN 5, 2023, View Source [SID1234625908]).

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On January 5, 2023 PDX Pharma reported that its proprietary platform (Pdx-NP) utilizes polymer-modified mesoporous silica nanoparticles and can effectively co-deliver a plethora of therapeutic modalities while maintaining a small size, suitable for infusion and tumor accumulation (Press release, PDX Pharmaceuticals, JAN 5, 2023, View Source [SID1234625907]). ARAC-02, one of our leading candidates, targets PD-L1, PLK1, and TLR9, and has the ability to simultaneously kill cancer while also inducing a potent anti-tumor immune response. ARAC-02 development won a $2.3M fast track SBIR award from the NCI with a rare perfect score in September 2021. In December 2022, ARAC-02 was selected into the NCL’s Assay Cascade Characterization Program, during which its physicochemical properties and in vitro and in vivo compatibility (immunology, pharmacology, and toxicology) will be characterized at no cost using a standardized assay cascade developed in collaboration with the FDA and NIST. This program will directly support our IND enabling studies toward the clinical adoption of ARAC-02.

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On January 5, 2023 MorphoSys AG (FSE: MOR; NASDAQ: MOR) t reported preliminary Monjuvi U.S. net product sales for the full year of 2022 and reported its financial guidance for 2023 (Press release, MorphoSys, JAN 5, 2023, View Source [SID1234625905]). In this context, MorphoSys reduced its financial liability from the collaboration with Incyte and credited finance income accordingly.

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Preliminary Monjuvi (tafasitamab-cxix) U.S. net product sales are US$ 25.3 million (€ 24.0 million) for the fourth quarter and US$ 89.4 million (€ 84.9 million) for the full year of 2022. Fourth quarter and full year 2022 results will be published on March 15, 2023. For the full year of 2023, MorphoSys expects Monjuvi U.S. net product sales in the range of US$ 80 to 95 million.

As a result of changes in Monjuvi product sales expectations, the balance sheet item "Financial Liabilities from Collaborations" is reduced from € 580 million (balance as of September 30, 2022) to approximately € 220 million (balance as of December 31, 2022; unaudited). Finance income is credited with the difference between the two amounts. The balance in "Financial Liabilities from Collaborations" reflects an accounting view of expected future profits from the net product sales of Monjuvi in the U.S. in relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) owed to our partner Incyte. The reduction in Financial Liabilities from Collaborations has no impact on cash.

"Patients living with relapsed or refractory diffuse large B-cell lymphoma continued to benefit from Monjuvi in 2022, as we saw an increase in sales of our medicine in the fourth quarter and a preliminary final sales total in line with our updated guidance," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "As we enter into the third year post-launch, we set our sales guidance for 2023 and longer-term projections for Monjuvi in its approved indication to reflect the ongoing and future impact of competitive activity. The team remains highly engaged to ensure continued awareness and use of Monjuvi as the only NCCN-preferred in-practice, out-patient immunotherapy for all appropriate patients, while preparing for our longer-term opportunities for pelabresib in first-line myelofibrosis and Monjuvi for first-line DLBCL."

Full Year 2023 Financial Guidance:

2023 Financial Guidance 2023 Guidance Insights
Monjuvi U.S. net product sales US$ 80m to 95m 100% of Monjuvi U.S. net 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 are recorded on MorphoSys’ income statement and related profit/loss is split 50/50 between MorphoSys and Incyte.
R&D expenses € 290m to 315m 2023 anticipated to be incrementally higher than 2022 due to the expansion of the pelabresib development program.
SG&A expenses € 140m to 155m 45% to 50% of mid-point of SG&A expenses represent Monjuvi U.S. selling costs of which 100% are recorded in MorphoSys’ income statement. Incyte reimburses MorphoSys for half of these selling expenses.

Additional information related to 2023 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 sales outside of the U.S.

MorphoSys does not anticipate any significant cash-accretive revenues from the achievement of milestones in 2023.

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.