On January 11, 2021 Exact Sciences Corp. (Nasdaq: EXAS) reported that the company expects to report revenue between $464.5 million and $467.5 million for the fourth quarter ended Dec. 31, 2020 (Press release, Exact Sciences, JAN 11, 2021, View Source [SID1234573753]).

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"I’m incredibly proud of how the Exact Sciences team came together during 2020 to make progress towards our mission of eradicating cancer and the suffering it causes," said Kevin Conroy, chairman and CEO of Exact Sciences. "Following our combination with Thrive, we are confident in making blood-based, multi-cancer screening a reality and being a leading provider of earlier, smarter cancer tests for patients at every step of their journey. We’ve never been more excited about the impact Exact Sciences will have on patient lives with Cologuard, Oncotype, and our future tests."

Preliminary, Unaudited Fourth Quarter 2020 Financial Results

For the three-month period ended December 31, 2020, as compared to the same period of 2019 (where applicable):

Expected total revenue between $464.5 million and $467.5 million
Expected Screening revenue between $249 million and $250 million, an increase of 9 percent
Expected Precision Oncology revenue between $117 million and $118 million
Expected COVID-19 testing revenue between $98.5 million and $99.5 million
Preliminary, Unaudited 2020 Financial Results

For the twelve-month period ended December 31, 2020, as compared to the same period of 2019 (where applicable):

Expected total revenue between $1.490 billion and $1.493 billion
Expected Screening revenue between $814.5 million and $815.5 million, an increase of 1 percent
Expected Precision Oncology revenue between $440 million and $441 million
Expected COVID-19 testing revenue between $235 million and $236 million
For the fourth quarter and 2020, Screening includes laboratory service revenue from Cologuard and revenue from Biomatrica products. Precision Oncology includes laboratory service revenue from global Oncotype products.

Exact Sciences has not completed preparation of its financial statements for the fourth quarter or full year of 2020. The revenue ranges presented in this news release for the fourth quarter of 2020 and for the year ended Dec. 31, 2020 are preliminary and unaudited and are thus inherently uncertain and subject to change as we complete our financial results for the fourth quarter of 2020. Exact Sciences is in the process of completing its customary year-end close and review procedures as of and for the year ended Dec. 31, 2020, and there can be no assurance that final results for this period will not differ from these estimates. During the course of the preparation of Exact Sciences’ consolidated financial statements and related notes as of and for the year ended Dec. 31, 2020, the company’s independent registered public accountants may identify items that could cause final reported results to be materially different from the preliminary financial estimates presented herein.

Exact Sciences plans to report 2020 financial results during its February 2021 earnings call.

About Cologuard
Cologuard was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. Cologuard is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2016) and National Comprehensive Cancer Network (2016). Cologuard is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use Cologuard if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. Cologuard is not a replacement for colonoscopy in high risk patients. Cologuard performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. Cologuard performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again.

Medicare and most major insurers cover Cologuard. For more information about Cologuard, visit www.cologuardtest.com. Rx only.

About Oncotype DX
The Oncotype DX portfolio of breast, colon and prostate cancer tests applies advanced genomic science to reveal the unique biology of a tumor in order to optimize cancer treatment decisions. In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer. Additionally, the Oncotype DX Breast DCIS Score test predicts the likelihood of recurrence in a pre-invasive form of breast cancer called DCIS. In prostate cancer, the Oncotype DX Genomic Prostate Score test predicts disease aggressiveness and further clarifies the current and future risk of the cancer prior to treatment intervention, and the Oncotype DX AR-V7 Nucleus Detect test helps determine which patients with metastatic castration-resistant prostate cancer (mCRPC) are resistant to androgen receptor (AR)-targeted therapies. The Oncotype DX AR-V7 Nucleus Detect test is performed by Epic Sciences at its centralized, CLIA-certified laboratory in San Diego and offered exclusively by Exact Sciences. With more than 1 million patients tested in more than 90 countries, the Oncotype DX tests have redefined personalized medicine by making genomics a critical part of cancer diagnosis and treatment. To learn more about Oncotype DX tests, visit www.OncotypeIQ.com, www.MyBreastCancerTreatment.org or www.MyProstateCancerTreatment.org.

On October 1, 2021 Window Therapeutics, Inc. (Window), a Massachusetts-based therapeutics company, reported a foundational partnership and seed funding in collaboration with the National Foundation for Cancer Research (NFCR) through its AIM-HI Accelerator Fund (AIM-HI) biotechnology innovation award program (Press release, National Foundation for Cancer Research, JAN 10, 2021, View Source [SID1234609539]). Terms of the seed-funding round were not disclosed.

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Founded in 2020, Window is creating next-generation cancer therapeutics designed to overcome limited efficacy and prohibitive toxicity which often confound successful drug development and as well as favorable patient outcomes. The company’s revolutionary technology platform derives from research that began over ten years ago by Dr. Jeremiah Johnson at the Massachusetts Institute of Technology (MIT). Window’s Bottle-brush Analogue Macromolecule (BAM) platform modifies the therapeutic window of small molecule, immune-based, and combination drug treatments to maximize efficacy specifically within disease sites. AIM-HI identified Window, its technology, and lead drug programs as possessing significant potential for transforming cancer drug development and advancing promising targeted drugs and immunotherapeutic to the clinic in an accelerated manner. AIM-HI’s lead role in the seed-funding of Window is intended to catalyze the progression of its lead candidates to the clinic, based on BAM technology that alleviates the pharmacokinetics, bioavailability, and off-target effects that so often lead to drug candidate failure in clinical trials.

"The Window Therapeutics’ approach embodies our passion to urgently address the limitations of current cancer therapies," said Sujuan Ba, Ph.D., co-Founder of AIM-HI and Chief Executive Officer of NFCR. "Our support for translating promising research from academia to the clinic by selectively identifying and nurturing early-stage companies accelerates treatment breakthroughs for cancer patients worldwide."

"AIM-HI’s support in creating a path forward to the clinic for our novel, bottle-brush macromolecule-based drug programs to benefit cancer patients is truly transformational," remarked Jeremiah Johnson, Ph.D., Window Therapeutics co-Founder, and Scientific Advisor. "Following a decade of progressive development to reconfigure drug therapeutic windows, we aspire to help clinicians and patients achieve and sustain successful outcomes."

On January 10, 2021 Istari Oncology, Inc., a clinical-stage biotechnology company, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for PVSRIPO for the treatment of advanced melanoma (stage IIB-IV) (Press release, Istari Oncology, JAN 10, 2021, View Source [SID1234577675]). PVSRIPO is a novel viral immunotherapy, based on the Sabin type 1 polio vaccine, that activates a patient’s innate and adaptive immune system to facilitate an anti-tumor response and establish long-term immunologic memory to help prevent the cancer’s return.

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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

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"We are happy to kick off the new year with the announcement that our request for an orphan drug designation has been granted to PVSRIPO for the treatment of advanced melanoma," said Matt Stober, President and Chief Executive Officer at Istari Oncology. "This is just one of many milestones to come in 2021 as we continue to drive the clinical development of PVSRIPO across multiple indications."

Currently, Istari is recruiting for LUMINOS-102, a Phase 2 open-label, randomized trial (clinicaltrials.gov NCT04577807) in patients with advanced, unresectable melanoma who previously failed anti-PD1 therapy. This study will characterize the safety, tolerability, and initial efficacy of PVSRIPO intratumoral injection alone and in combination with a PD-1 inhibitor. The first patient is expected to be dosed this quarter.

LUMINOS-102 follows a successful Phase 1 monotherapy study of PVSRIPO in anti-PD1 refractory advanced melanoma in which patients who received 3 injections (6/12) had an overall response rate of 67% (4/6).

"We are encouraged by the data from our Phase 1 trial presented at last year’s Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2020 annual meeting" said Garrett Nichols, MD, MS, Chief Medical Officer at Istari Oncology. "We plan to build upon that success with LUMINOS-102. As those data and the data from our other solid tumor trials emerge, we will continue to work closely with the FDA toward the goal of bringing PVSRIPO to market."

Melanoma is the deadliest type of skin cancer today with approximately 7,000 deaths in the U.S. each year. Treatment-resistant, advanced melanoma patients have very poor survival rates, with less than 28% of metastatic melanoma patients surviving 5 years1. PVSRIPO aims to address the significant unmet need in advanced melanoma, since most patients are treated with checkpoint inhibitors, and many do not respond or later become resistant and require other options, which are limited.

Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to drugs or biological products intended for the treatment of rare diseases or conditions that impact fewer than 200,000 people in the U.S. This designation acts as a stimulus for the development of drugs for rare diseases through several incentives, including eligibility for federal grants, research and development tax credits, waiver of filing fees, and the potential for a seven-year marketing exclusivity period after FDA approval.

The granting of an orphan drug designation does not alter the standard regulatory requirements and process for obtaining marketing approval. More information about orphan drug designation can be found on the OOPD website.

For more information about Istari Oncology and their ongoing clinical trials, visit istarioncology.com.

About PVSRIPO
PVSRIPO is a virus based on the live attenuated Sabin type 1 polio vaccine that has been genetically modified for safety. Unlike other viral immunotherapies, PVSRIPO has a distinct target (the poliovirus receptor CD155), which is widely expressed in neoplastic cells of most solid tumors. Via CD155, PVSRIPO targets tumors with two primary mechanisms: 1) direct damage to and killing of cancerous cells; and 2) engaging innate and adaptive antitumor immune responses via sublethal infection of antigen presenting cells in the tumor, which unleashes an inflammatory cascade resulting in sustained systemic antitumor immunity. PVSRIPO has been granted Breakthrough Therapy Designation and Orphan Status by the FDA in recurrent glioblastoma. PVSRIPO has also been granted Orphan Status by the FDA for advanced melanoma.

About Melanoma
There are estimated to be over 12,000 new and recurrent cases of advanced, unresectable melanoma diagnosed in the U.S. each year, and around 7,000 deaths. While immune checkpoint inhibitors have dramatically improved the outlook for advanced melanoma patients today, most patients treated with these immunotherapies are either primary non-responders or eventually develop immune-refractory progressive disease and require additional therapy.

On January 10, 2021 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its new 5-year strategy "Alnylam P5x25" focused on the Company’s planned transition to a top-5 biotech (measured by market capitalization) in the next 5 years through: sustainable innovation yielding transformative medicines for rare and common diseases for patients around the world and delivery of exceptional financial performance (Press release, Alnylam, JAN 10, 2021, View Source [SID1234573944]). Alnylam P5x25 extends the Company’s decade-long heritage of providing longer term, 5-year business strategy guidance, the most recent of which was known as Alnylam 2020. In addition, Alnylam today reported preliminary fourth quarter and full year 2020 global net product revenues for ONPATTRO and GIVLAARI and provided additional updates on the Company’s commercial launches, including initial OXLUMO demand.

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New 5-Year Strategy: P5x25
Alnylam ended last year exceeding all metrics for its Alnylam 2020 strategy, with 4 marketed products (versus 3), 12 clinical programs (versus 10), 6 of which are in late-stage development (versus 4), across 4 strategic therapeutic areas (versus 3).

The Company’s Alnylam P5x25 strategy is aimed at Alnylam’s transition to a top 5 biotech company, as measured by market capitalization, over the next 5 years.

Specifically, the Company intends to end 2025 with the following profile**:

Patients: Over 0.5 million on Alnylam RNAi therapeutics globally
Products: 6 or more marketed products in rare and prevalent diseases
Pipeline: Over 20 clinical programs, with 10 or more in late stages and 4 or more INDs per year
Performance: ≥40% revenue CAGR through YE 2025
Profitability: Achieve sustainable non-GAAP profitability within the period
"We executed well on our Alnylam 2020 strategy, exceeding all pre-set metrics and transitioning into a global, multi-product commercial company with a robust clinical pipeline and an organic product engine delivering sustainable innovation, a profile that has rarely been achieved in biotech history. It was especially gratifying to cap 2020 with positive Phase 3 HELIOS-A results for vutrisiran, which is set to become our 5th RNAi therapeutic to reach the market, if approved," said John Maraganore, Ph.D., CEO of Alnylam Pharmaceuticals. "We are now thrilled to launch our new chapter with Alnylam P5x25, which is aimed at Alnylam’s planned transition to a top 5 biotech in market capitalization based on a proven and high-yielding technology for disruptive medical innovation and a foundational track record of commercial execution. Indeed, with Alnylam P5x25, we expect to sustainably and organically create and commercialize transformative rare and common disease medicines benefiting hundreds of thousands of patients around the world while delivering strong financial performance and profitability, resulting in a leading biotech profile."

2020 Preliminary Commercial and Financial Performance*
ONPATTRO (patisiran), a commercial-stage RNAi therapeutic targeting transthyretin (TTR) for the treatment of polyneuropathy in adult patients with hATTR amyloidosis.

Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $90 million and $306 million, respectively.
Q4 results represent approximately 10 percent growth compared to Q3 and include 10 percent growth in the U.S. market segment driven by new patient demand.
Further, the full year ONPATTRO revenues reached the high end of the previously shared guidance range of $295 million – $310 million and represent over 80 percent growth from full year 2019.
As of year-end 2020, about 1,350 patients worldwide were receiving commercial ONPATTRO.
GIVLAARI (givosiran), a commercial-stage RNAi therapeutic for the treatment of adults with acute hepatic porphyria (AHP).

Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $22 million and $55 million, respectively.
These results represent greater than 30 percent quarter over quarter growth.
As of year-end 2020, the product’s first full year of launch, over 200 patients are receiving commercial drug.
OXLUMO (lumasiran), a commercial-stage RNAi therapeutic for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients.

For the period following European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) approval of OXLUMO, in late November 2020, strong initial U.S. demand was observed with 8 Start Forms received through year-end.
Preliminary global net product revenues for the fourth quarter were approximately $0.3 million representing initial patient demand in Europe
Finally, the Company today reported that it expects its full year 2020 non-GAAP operating loss to be substantially improved relative to the prior year, marking 2019 as Alnylam’s peak non-GAAP operating loss year as the Company transitions towards a self-sustainable financial profile.

Further, at December 31, 2020, Alnylam had preliminary cash, cash equivalents, and marketable securities of approximately $1.9 billion, as compared to $1.5 billion at December 31, 2019. The Company balance sheet was strengthened by the 2020 strategic financing collaboration with Blackstone.

"We are extremely pleased with ONPATTRO and GIVLAARI performance in the fourth quarter and cumulatively for the year, reflecting strong commercial execution, a substantial increase in demand and new patient adds, despite the ongoing COVID-19 pandemic. Additionally, we’re pleased to report that strong top-line revenue growth at the upper end of our guidance range and disciplined R&D and SG&A investments have delivered on a lower non-GAAP operating loss for 2020 compared with the prior year. We believe we’re now firmly on our way toward a self-sustainable financial profile with significant growth from four revenue generating assets and moderated operating expenses, and a balance sheet that supports achievement of profitability without the need to access the equity markets," said Jeff Poulton, Chief Financial Officer of Alnylam. "As we now launch our Alnylam P5x25 strategy, we’re committed to delivering consistently strong financial performance driven by 40% or greater revenue CAGR over the next five years with non-GAAP profitability achieved within the period."

Alnylam management will discuss these preliminary selected financial results and commercial updates during a webcast presentation at the 39th Annual J.P. Morgan Healthcare Conference tomorrow, Monday, January 11, 2021, at 8:20 a.m. ET.

About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

On January 10, 2021 Istari Oncology, Inc., a clinical-stage biotechnology company, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for PVSRIPO for the treatment of advanced melanoma (stage IIB-IV) (Press release, Istari Oncology, JAN 10, 2021, View Source [SID1234573789]). PVSRIPO is a novel viral immunotherapy, based on the Sabin type 1 polio vaccine, that activates a patient’s innate and adaptive immune system to facilitate an anti-tumor response and establish long-term immunologic memory to help prevent the cancer’s return.

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 happy to kick off the new year with the announcement that our request for an orphan drug designation has been granted to PVSRIPO for the treatment of advanced melanoma," said Matt Stober, President and Chief Executive Officer at Istari Oncology. "This is just one of many milestones to come in 2021 as we continue to drive the clinical development of PVSRIPO across multiple indications."

Currently, Istari is recruiting for LUMINOS-102, a Phase 2 open-label, randomized trial (clinicaltrials.gov NCT04577807) in patients with advanced, unresectable melanoma who previously failed anti-PD1 therapy. This study will characterize the safety, tolerability, and initial efficacy of PVSRIPO intratumoral injection alone and in combination with a PD-1 inhibitor. The first patient is expected to be dosed this quarter.

LUMINOS-102 follows a successful Phase 1 monotherapy study of PVSRIPO in anti-PD1 refractory advanced melanoma in which patients who received 3 injections (6/12) had an overall response rate of 67% (4/6).

"We are encouraged by the data from our Phase 1 trial presented at last year’s Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2020 annual meeting" said Garrett Nichols, MD, MS, Chief Medical Officer at Istari Oncology. "We plan to build upon that success with LUMINOS-102. As those data and the data from our other solid tumor trials emerge, we will continue to work closely with the FDA toward the goal of bringing PVSRIPO to market."

Melanoma is the deadliest type of skin cancer today with approximately 7,000 deaths in the U.S. each year. Treatment-resistant, advanced melanoma patients have very poor survival rates, with less than 28% of metastatic melanoma patients surviving 5 years1. PVSRIPO aims to address the significant unmet need in advanced melanoma, since most patients are treated with checkpoint inhibitors, and many do not respond or later become resistant and require other options, which are limited.

Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to drugs or biological products intended for the treatment of rare diseases or conditions that impact fewer than 200,000 people in the U.S. This designation acts as a stimulus for the development of drugs for rare diseases through several incentives, including eligibility for federal grants, research and development tax credits, waiver of filing fees, and the potential for a seven-year marketing exclusivity period after FDA approval.

The granting of an orphan drug designation does not alter the standard regulatory requirements and process for obtaining marketing approval. More information about orphan drug designation can be found on the OOPD website.

For more information about Istari Oncology and their ongoing clinical trials, visit istarioncology.com.

About PVSRIPO
PVSRIPO is a virus based on the live attenuated Sabin type 1 polio vaccine that has been genetically modified for safety. Unlike other viral immunotherapies, PVSRIPO has a distinct target (the poliovirus receptor CD155), which is widely expressed in neoplastic cells of most solid tumors. Via CD155, PVSRIPO targets tumors with two primary mechanisms: 1) direct damage to and killing of cancerous cells; and 2) engaging innate and adaptive antitumor immune responses via sublethal infection of antigen presenting cells in the tumor, which unleashes an inflammatory cascade resulting in sustained systemic antitumor immunity. PVSRIPO has been granted Breakthrough Therapy Designation and Orphan Status by the FDA in recurrent glioblastoma. PVSRIPO has also been granted Orphan Status by the FDA for advanced melanoma.

About Melanoma
There are estimated to be over 12,000 new and recurrent cases of advanced, unresectable melanoma diagnosed in the U.S. each year, and around 7,000 deaths. While immune checkpoint inhibitors have dramatically improved the outlook for advanced melanoma patients today, most patients treated with these immunotherapies are either primary non-responders or eventually develop immune-refractory progressive disease and require additional therapy.