On October 13, 2021 Veracyte, Inc., (Nasdaq: VCYT) reported the publication of new data demonstrating that the company’s Decipher Bladder genomic classifier accurately identifies bladder tumors that are most likely to respond to chemotherapy prior to radical cystectomy (Press release, Veracyte, OCT 13, 2021, View Source [SID1234591182]). These findings could ultimately help physicians optimize treatment planning for their patients with bladder cancer based on their tumor subtype biology. The peer-reviewed paper appears online today in The Journal of Urology.

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Patients diagnosed with non-metastatic muscle-invasive bladder cancer (MIBC) often undergo neoadjuvant chemotherapy (NAC) prior to standard-of-care radical cystectomy, even though the absolute survival benefit associated with the addition of NAC to radical cystectomy is 5-10%. Until recently, there was no reliable way to determine which MIBC tumors would – or would not – respond to chemotherapy. Molecular subtyping with the Decipher Bladder genomic classifier has shown that biological differences in MIBC are strongly associated with chemotherapy response.

"Our findings are among the first to show the clinical utility of implementing molecular subtyping in order to identify patients for whom NAC is most likely to confer significant benefit," said Yair Lotan, MD, professor of urology and chief of urologic oncology at UT Southwestern Medical Center, and the paper’s lead author. "This study represents meaningful progress in clinically validating biomarkers that are associated with chemotherapy response in patients with MIBC, and it offers hope that we will be able to better manage these patients in the future by accurately selecting those most likely to benefit from additional treatment."

In this multicenter retrospective study, scientists evaluated the use of the Decipher Bladder genomic classifier in a cohort of 601 patients. After three years, patients with classifier-identified luminal tumors (37% of the study population) experienced no additional benefit to overall survival (OS) from receiving NAC prior to radical cystectomy (63% OS with NAC and 65% OS without). However, those patients whose tumors were classified as non-luminal (63% of the study population) experienced a significant benefit from the addition of NAC, with 10% greater overall survival after three years as compared to patients who were treated with cystectomy alone (71% vs. 61%).

"We are pleased that our Decipher Bladder genomic classifier performed so well in this broad study," said Elai Davicioni, Ph.D., Veracyte’s senior vice president of scientific and clinical operations, Urologic Cancers. "We look forward to further validating the test to encourage its routine clinical use for patients diagnosed with MIBC."

The Decipher Bladder test is supported by multiple peer-reviewed clinical studies demonstrating its ability to identify which patients have a higher risk of upstaging to non-organ confined disease at surgery and which patients may benefit the most from neoadjuvant therapy. The test also can be used to identify neuroendocrine-like and immune-infiltrated subtypes, which may have implications for future therapeutic strategies. In June 2021, Veracyte announced that Decipher Bladder had become the first molecular subtyping test to gain Medicare coverage to inform treatment for individuals with bladder cancer.

About Decipher Bladder

Decipher Bladder is a genomic test that measures the molecular profile of bladder cancer using gene expression analysis from transurethral resected bladder tumor specimens. It was developed for bladder cancer patients with high-grade non-muscle-invasive disease who are being considered for treatment and patients with muscle-invasive disease who face the question of immediate cystectomy or systemic treatment in the neoadjuvant setting prior to cystectomy (NAC). Decipher Bladder reports the molecular subtype of the tumor specimen as Luminal or Non-Luminal (Luminal Infiltrated, Basal, Basal Claudin-Low or Neuroendocrine-like), with each subtype having distinct biological composition, clinical behavior and predicted benefit from NAC.

On October 13, 2021 LAVA Therapeutics Presented the Corporate Presentation (Presentation, Lava Therapeutics, OCT 13, 2021, View Source [SID1234591181]).

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On October 13, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported positive results from a pharmacoeconomic study evaluating the cost-effectiveness of using investigational drug Motixafortide as a primary stem cell mobilization (SCM) agent on top of granulocyte colony stimulating factor (G-CSF), versus G-CSF alone, in multiple myeloma patients undergoing autologous stem cell transplantation (ASCT) (Press release, BioLineRx, OCT 13, 2021, View Source [SID1234591180]). The study was performed by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA, and was a pre-planned study conducted in parallel with the GENESIS Phase 3 trial. These results, together with the highly significant and clinically meaningful data from the GENESIS trial, strongly support the potential use of Motixafortide, on top of G-CSF, as the standard of care in SCM for ASCT.

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The study concluded that the addition of Motixafortide to G-CSF (the current standard of care) is associated with a statistically significant decrease in health resource utilization (HRU) during the ASCT process, compared to G-CSF alone. Based on the significantly higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus G-CSF alone. The study findings, combined with model estimates, suggest that the use of Motixafortide, on top of G-CSF, as the standard of care in mobilization for ASCT, could be a cost-effective option in the US, based on accepted willingness-to-pay (WTP) values for healthcare payers.

"The compelling cost savings identified by this rigorously designed study strongly support the Company’s view that Motixafortide, in combination with G-CSF, can become the new standard of care as an upfront, or primary, therapy for all multiple myeloma patients undergoing autologous stem cell transplantation," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Based on data from the GENESIS trial showing that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration of Motixafortide and in only one apheresis session, versus less than 10% for G-CSF alone, the pharmacoeconomic study demonstrates that use of Motixafortide on top of G-CSF can save $17,000 per patient, not including the cost of Motixafortide. These cost savings should leave substantial room in the future to optimize our pricing strategy for Motixafortide at product launch and thereafter, if approved.

"It is also important to note that fewer administrations and apheresis sessions confer meaningful safety and time benefits to patients. In addition, the significantly higher median number of cells collected in one apheresis session – ~11 million using Motixafortide on top of G-CSF versus ~2 million for G-CSF alone – not only enables transplantation of an optimal number of cells, with the potential to significantly save on time to engraftment, it also permits the retention of enough cells for cryopreservation in the event that an additional transplantation is required in the future. Lastly, higher levels of certainty regarding the number of apheresis sessions required for mobilization could enable more efficient utilization of apheresis units at transplantation institutions, where there is often a shortage of available machines.

"We believe the data from the GENESIS study, together with the results from this pharmacoeconomic study, set Motixafortide apart from all other mobilization agents either currently available or in development. If approved, Motixafortide represents a significant advancement in SCM to the benefit of patients and payers alike, and, to that end, we remain on track to submit a New Drug Application (NDA) to the FDA in the first half of next year," Mr. Serlin concluded.

About the Pharmacoeconomic Study

The pharmacoeconomic study analyzed healthcare resource utilization (HRU) observed during the Phase 3 GENESIS trial, which randomized 122 patients into two arms: Motixafortide plus G-CSF (n=80) or placebo plus G-CSF (n=42). HRU data points collected include: (1) the number of Motixafortide and G-CSF doses, as well as the number of apheresis sessions performed, in primary mobilization; (2) the percentage of patients needing rescue mobilization due to poor primary mobilization, including the number of apheresis sessions needed and the number of G-CSF and plerixafor doses required; and (3) hospitalization costs related to conditioning and transplantation, including length of stay. Quality-adjusted life years gained (QALY) from published literature were also incorporated into the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard-of-care G-CSF alone. Given the higher number of mobilized cells and lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus the current standard of care.

About the GENESIS Phase 3 Trial

The GENESIS Phase 3 trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. The study successfully met all primary and secondary endpoints with an exceptionally high level of statistical significance (p<0.0001), including approximately 90% of patients who mobilized the target number of cells for transplantation with only one administration of Motixafortide and in only one apheresis session.

About Stem Cell Mobilization for Autologous Stem Cell Transplantation

Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma, non-Hodgkin’s lymphoma and other lymphomas. In eligible patients, ASCT is performed after initial (induction) therapy, and, in most cases, requires consecutive-day clinic visits for the mobilization and apheresis (harvesting) phases, and full hospitalization for the conditioning chemotherapy and transplantation phases until engraftment. The associated burden is therefore significant – patients experience clinically relevant deteriorations in their quality of life during ASCT, and healthcare resource use throughout the ASCT phases is particularly intense. Therefore, new interventions impacting the ASCT process have the potential for relieving some of the clinical burden for transplanted patients, the logistical burden for the apheresis units, and the financial burden for healthcare providers and payers.

Described simply, ASCT consists of: (1) mobilizing the patient’s own stem cells from his/ her bone marrow to the peripheral blood for removing (harvesting) via an apheresis procedure; (2) freezing and storing the harvested cells until they are needed for transplantation; (3) providing a conditioning treatment, such as high-dose chemotherapy or radiation, to kill the remaining cancer cells the day before transplant; and (4) infusing the stored stem cells back to the patient intravenously via a catheter.

To mobilize the patient’s stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.

On October 13, 2021 Incyte (Nasdaq:INCY) reported that it has scheduled its third quarter financial results conference call and webcast for 8:00 a.m. ET on Tuesday, November 2, 2021 (Press release, Incyte, OCT 13, 2021, View Source [SID1234591178]).

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The schedule for the press release and conference call/webcast is as follows:

• Q3 2021 Press Release:

November 2, 2021 at 7:00 a.m. ET
• Q3 2021 Conference Call: November 2, 2021 at 8:00 a.m. ET
• Domestic Dial-In Number: 877-407-3042
• International Dial-In Number: 201-389-0864
• Conference ID Number:
13724199

If you are unable to participate, a replay of the conference call will be available for thirty days. The replay dial-in number for the U.S. is 877-660-6853 and the dial-in number for international callers is 201-612-7415. To access the replay you will need the conference ID number 13724199.

The live webcast with slides can be accessed at Investor.Incyte.com and will be available for replay for 90 days.

On October 13, 2021 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, reported a strategic collaboration to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases (Press release, Intellia Therapeutics, OCT 13, 2021, View Source [SID1234591177]).

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As part of this collaboration, Intellia will grant SparingVision exclusive rights to Intellia’s proprietary in vivo CRISPR/Cas9-based genome editing technology for up to three ocular targets addressing diseases with significant unmet medical need. SparingVision will lead and fund the preclinical and clinical development for the genome editing product candidates pursued under the collaboration. In addition, the parties will research and develop novel self-inactivating AAV vectors and LNP-based approaches to address delivery of CRISPR/Cas9 genome editing reagents to the retina.

As part of the licensing agreement, Intellia will receive a 10% equity ownership stake in SparingVision. Intellia will also be eligible to receive certain development and commercial milestone payments (around $200 million per product) as well as royalties on potential future sales of products arising from the collaboration. In addition, Intellia may exercise an option to obtain the US commercialization rights for product candidates arising from two of three collaboration targets. For product candidates Intellia chooses to option, Intellia will pay an opt-in fee, reimburse certain costs, share in 50% of development costs and pay royalties to SparingVision on US sales. Intellia will also maintain the ability to leverage technology advances established under this collaboration for any targets outside the partnership.

Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: "SparingVision’s aim has always been to disrupt the ophthalmology field by using cutting-edge technologies to address areas of significant unmet need. This collaboration with Intellia marks a pivotal moment in this mission and is highly complementary to our already mature and growing pipeline of unique mutation-agnostic gene therapies. Intellia is the first company in history to present clinical data supporting precision editing of a disease-causing gene within the body following a single, systemic dose of CRISPR/Cas9 and we are honored to have been selected as a strategic partner. We look forward to working together with the shared goal of radically changing the treatment of blinding ocular diseases."

"Intellia has been a pioneer in utilizing CRISPR/Cas9 technology to develop potentially curative treatments for genetic diseases. Today’s announcement is another step forward in more fully leveraging the power of our genome editing technology to address diseases inadequately treated with existing medicines," said Intellia President and Chief Executive Officer John Leonard, M.D. "We have been thoroughly impressed with the team at SparingVision, particularly regarding their unparalleled understanding of retinal diseases and track-record for developing novel therapies for patients with ocular diseases. We believe SparingVision will be an excellent partner to expand our genome editing capabilities into the field of ophthalmology and we look forward to our new partnership."

The transaction is expected to close in the fourth quarter and is subject to certain closing conditions.

SparingVision’s management team will be holding a webcast to discuss the collaboration today (13th October) at 16:00 CEST / 15:00 BST / 10:00 ET, which will include a live Q&A session. Please find a link to join this webcast here: View Source