On January 18, 2022 Scandion Oncology (Scandion), a biotech company developing first-in-class medicines aimed at treating cancer which is resistant to current treatment options, reported that it will have clinical data for its lead compound SCO-101 presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium taking place on January 20-22, 2022 (Press release, Scandion Oncology, JAN 18, 2022, View Source,c3486737 [SID1234605549]).

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Pharmacokinetic data from part 1 of the CORIST Phase II-trial in patients with metastatic colorectal cancer will be announced at a poster presentation. These data will provide details to the topline results communicated earlier documenting the unique mode of action for SCO-101 which provides a significant potentiation of the chemotherapy Irinotecan when combined with the chemotherapy regimen FOLFIRI (which includes Irinotecan).

"We are very pleased to have these data accepted for presentation at the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium, making it available to a large number of physicians and other stakeholders. We are excited by the data that confirms the potential of SCO-101 to significantly improve treatment options for patients", says Bo Rode Hansen, President & CEO of Scandion.

The combination of SCO-101 and FOLFIRI resulted in a dramatically increased exposure and half-life of SN-38, the active metabolite of Irinotecan. In other words, SCO-101 has the potential to make the chemotherapy much more effective without increasing side effects for patients.

Further to these findings, part 1 of the CORIST trial has also, as previously announced, helped establish a well-tolerated dose of SCO-101 in combination with FOLFIRI. Further, the trial identified the oncogene RAS as a predictive biomarker, helping to optimize patient inclusion in the ongoing second part of the trial, which is expected to conclude in the second half of 2022.

The abstract titled "Evaluation of SN-38 PK profile in patients with RAS wild-type metastatic colorectal cancer treated with a combination of SCO-101 and FOLFIRI" will be published at the conference website later today: View Source

On January 18, 2022 Sema4 (Nasdaq: SMFR), an AI-driven genomic and clinical data intelligence platform company, and OPKO Health, Inc. (Nasdaq: OPK) ("OPKO"), a multinational biopharmaceutical and diagnostics company, reported that they have signed a definitive agreement for Sema4 to acquire OPKO’s wholly owned subsidiary, GeneDx, Inc. ("GeneDx"), a leader in genomic testing and analysis, from OPKO (Press release, Opko Health, JAN 18, 2022, View Source [SID1234605547]). The acquisition strengthens Sema4’s leadership, growth, and scale for its market-leading health intelligence and genomic screening offerings. Together, Sema4 and GeneDx will be one of the largest and most advanced providers of genomic clinical testing in the U.S., with a projected $350 million in pro forma 2022 revenue.

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Following completion of the acquisition, Sema4 will be optimally positioned to partner with health systems and biopharma companies to further transform the standard of care throughout the patient health journey. GeneDx’s leadership in rare disease diagnostic and exome sequencing services brings more than 300,000 clinical exomes and over 2.1 million expertly annotated phenotypes to strengthen Sema4’s 12 million de-identified clinical records for Centrellis, its proprietary integrated health intelligence platform, and Traversa, its comprehensive genomic analysis platform for optimizing health screenings. Sema4 plans to leverage this combined health information database to transform patient care and therapeutic development and enable precision medicine for all.

"This acquisition gives us the opportunity to accelerate the use of genomics as standard of care by providing a deeper menu of precision medicine solutions to our health system partners to better meet their clinical needs," said Eric Schadt, PhD, Founder and CEO of Sema4. "Adding GeneDx’s comprehensive dataset and capabilities to our offerings enables us to inform on an even broader range of diseases, further closing the gap between the practice of medicine and the availability of more clinically actionable guidance. GeneDx’s operational prowess and market-leading cost structure in exome and genome sequencing will also help accelerate our path to improved gross margins and profitability. I am also delighted to welcome Katherine to our leadership team. She and her team’s world-class expertise will be critical to our continued growth and success."

Katherine Stueland, President and CEO of GeneDx and former CCO of Invitae, will be appointed as Sema4 co-CEO and is expected to join the Sema4 Board of Directors upon completion of the acquisition. She brings significant commercial and operational experience and will lead overall operational excellence and business planning, and will focus on the diagnostics business. Dr. Schadt will continue to serve Sema4 as co-CEO and as a member of the Board of Directors focusing on leading R&D and the IT platform components of Sema4, the strategic development of Sema4’s health intelligence capabilities, and partnerships with health systems and biopharma companies. Together as co-CEOs, Dr. Schadt and Ms. Stueland will drive overall strategy and direction of the company.

"We are excited to join forces with Sema4, a market leader in using genomic and clinical data to deliver precision medicine," said Ms. Stueland. "The complementary fit between our teams, missions, and capabilities is strong. We are eager to put those strengths to work and to make it easier to use data-driven insights to improve healthcare for all. I’m looking forward to partnering with Eric to create an unrivaled family health and health intelligence company, supporting patients making healthcare decisions throughout their lives, from pregnancy and newborn health to adult rare disease, risk assessment, and cancer care."

As part of the transaction, Sema4 has also announced that it has entered into definitive agreements for a $200 million private placement of Sema4 Class A shares from a syndicate of institutional investors, including Pfizer. The acquisition and the private placement (together, the "Transaction") are expected to close concurrently in the first half of 2022, subject to a Sema4 stockholder vote and other conditions to closing set forth in the definitive Transaction documents.

Dr. Schadt added: "We are excited to announce this investment with the support of several key institutions, including Pfizer. We believe that genomics and data, when harnessed in partnership with health systems, can be a powerful tool to enable precision medicine by bringing novel therapies to patients faster and more effectively. We hope that this investment may serve as a foundation for potential future collaborations."

Phillip Frost, MD, Chairman and CEO of OPKO, added: "We believe the sale of GeneDx to Sema4 will unlock untapped value and maximize the value of GeneDx for the benefit of OPKO shareholders. In addition to bolstering our cash position, we will have a significant equity stake in Sema4 at closing, ensuring OPKO and our shareholders continue to participate in the rapidly growing genomics market through a continued investment in GeneDx, which we believe is well positioned to deliver long-term success."

In conjunction with the Transaction, Jason Ryan, a member of Sema4’s Board of Directors, former CFO of Foundation Medicine and most recently Chief Operating and Financial Officer of Magenta Therapeutics (Nasdaq: MGTA), will assume the role of Executive Chair of Sema4’s Board of Directors. Mr. Ryan has outstanding leadership experience in the life sciences and biotechnology sectors, and an impressive background in finance and scaling businesses. Mr. Ryan succeeds Joshua Ruch, who will continue to serve on Sema4’s Board of Directors as the Chairperson of its Compensation Committee.

Based on Sema4’s closing stock price as of January 14, 2022, the purchase price is approximately $623 million, including in total upfront cash, stock consideration, and contingent consideration upon commercial milestones.

Sema4 Standalone Fiscal Year 2022 Guidance

Sema4 expects total revenue for fiscal year 2022 to be in the range of $215 million to $225 million, implying 23-29% growth excluding revenue associated with COVID-19, and Sema4 also expects to result over 350,000 tests in 2022 excluding COVID-19 tests. On December 15, 2021, Sema4 announced that it has decided to discontinue COVID-19 testing services by March 31, 2022 and therefore the company expects an immaterial amount of revenue from COVID-19 in 2022.

Acquisition Terms

Under the terms of the agreement, Sema4 will acquire GeneDx for an upfront payment of $150 million in cash plus 80.0 million shares in Sema4, with up to an additional $150 million revenue-based milestones over the next two years (which will be payable in cash or Sema4 shares at Sema4’s discretion). Based on the closing stock price of Sema4 as of January 14, 2022, the total upfront consideration represents approximately $473 million, and the total aggregate consideration including potential milestones is approximately $623 million. The acquisition, which has been unanimously approved by the Boards of Directors of both Sema4 and OPKO, is expected to close in the second quarter of 2022, subject to customary closing conditions including approval by the stockholders of Sema4.

Private Placement

In connection with the acquisition, Sema4 has also entered into definitive agreements for a private placement financing to sell $200 million in Class A common stock at a price of $4.00 per share from a syndicate of institutional investors, including Pfizer.

The private placement is expected to substantially close concurrently with close of the acquisition, subject to the satisfaction of customary closing conditions.

Advisors

Goldman Sachs & Co. LLC. acted as financial advisor and Fenwick & West LLP served as legal counsel to Sema4 on the Transaction. J.P. Morgan acted as lead financial advisor and Cowen acted as financial advisor to OPKO on the Transaction. Greenberg Traurig, P.A. served as OPKO’s legal counsel. Goldman Sachs & Co. LLC. served as lead placement agent on the private placement. Jefferies LLC, Cowen, and BTIG, LLC also served as placement agents.

Conference Call and Webcast Details

Management will host a conference call and webcast today at 8:00 a.m. ET to discuss the transaction. Following prepared remarks, management will respond to questions from analysts, subject to time limitations.

The live webcast of the call and slide deck may be accessed by visiting the investors section of Sema4’s website at ir.sema4.com. A replay of the webcast and conference call will be available shortly after the conclusion of the call and will be archived on Sema4’s website.

On January 18, 2022 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported its interim results (N=63) in its metastatic pancreatic cancer trial (QUILT 88) showing that the overall survival rate for patients doubled compared to historical survival rate of three months after two prior lines of therapy (Manax ASCO (Free ASCO Whitepaper) GI 2019) (Press release, ImmunityBio, JAN 18, 2022, View Source [SID1234605546]). The data of the Phase 2 trial studying a combination immunotherapy (Nant Cancer Vaccine) also show treatment-related serious adverse events were uncommon (8%) and no treatment-related deaths were reported. Based on these findings, ImmunityBio plans to meet with the FDA in 2022 to discuss the path for the approval of combination therapies for pancreatic cancer.

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The results were presented today at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal conference, which is being held virtually.

Pancreatic cancer is the fourth leading cause of cancer-related death in the United States and has one of the highest mortality rates of all major cancers, taking nearly 50,000 lives in the U.S. every year. Surgery and subsequent adjuvant chemotherapy are the preferred treatment options for pancreatic cancer today, but the five-year survival rate for late-stage cases is just 3%. For the majority of patients who present with more advanced disease, treatment typically consists of chemotherapy alone or supportive care for metastatic patients, and chemotherapy with or without radiation for those with locally advanced disease, leaving patients seeking new options.

"There are thousands of patients in advanced stages of this disease and there are few, if any, treatment options for them," said Patrick Soon-Shiong, M.D., Founder and Global Chief Scientific and Medical Officer of ImmunityBio, Inc. "Based on this encouraging data from our QUILT 88 trial, we are hopeful that our Nant Cancer Vaccine can potentially address this unmet need. What’s more, we designed this therapeutic candidate to be administered in an outpatient setting making it more accessible to future patients than traditional immune checkpoint inhibitors."

To date, 27% of third-line or greater patients (17/63) remain on study. The median overall survival in this highly advanced group of patients, who failed two to six prior lines of treatment, is 5.8 months (95% CI: 3.9, 6.9 months) exceeding the approximately three-month historical median overall survival. Of the 63 patients, 30 (48%) had progressed after two prior lines of therapy. Median overall survival in this group was 6.3 months (95% CI: 5.0, 9.8 months), more than doubling the historical overall survival. On the strength of this early data and significant unmet medical need, the company has expanded enrollment in the third-line or greater cohort.

QUILT 88 is an open-label study to evaluate the safety and efficacy of the Nant Cancer Vaccine, comprising ImmunityBio’s IL-15 receptor agonist Anktiva (N-803), its off-the-shelf targeted natural killer cells (PD-L1 t-haNK), and aldoxorubicin, an albumin-modulated agent, plus low-dose chemotherapy. This combination Nant Cancer Vaccine is randomized against standard-of-care high-dose chemotherapy for first- and second-line treatment; the third-line or greater cohort, with an original target of 50 patients, is a single arm with the primary endpoint of overall survival.

QUILT-88 Study Details

This Phase 2, randomized, three-cohort, open-label study will evaluate the comparative efficacy and overall safety of standard-of-care chemotherapy versus low-dose chemotherapy in combination with PD-L1 t-haNK, Anktiva (N-803), and aldoxorubicin in subjects with locally advanced or metastatic pancreatic cancer (NCT04390399). Each treatment setting, as well as each first- and second-line or later maintenance treatment, will be evaluated independently as Cohorts A, B, and C, respectively, with Cohorts A and B having independent experimental and control arms. The study is expected to enroll 328 subjects across all three cohorts giving effect to the expanded enrollment of Cohort C (63 of 80 participants are currently enrolled in Cohort C, third-line or greater). The primary objective of Cohorts A and B is progression-free survival (PFS) per RECIST V1.1, and the objective of Cohort C is overall survival (OS). Secondary objectives include initial safety and additional efficacy measures, including overall response rate (ORR), complete response (CR) rate, durability of response (DoR), disease control rate (DCR), and overall survival (OS).

Currently, three trial sites have been activated: Hoag Memorial Hospital Presbyterian in Orange County, Calif., The Chan Soon-Shiong Institute for Medicine in Los Angeles County, Calif., and Avera McKennan Hospital and University Health Center in Sioux Falls, South Dakota, which serves patients in the tri-state area (Iowa, Nebraska and South Dakota).

On January 18, 2022 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today reported that it has received an updated independent safety review of certain preliminary data for the first 30 patients in its three Phase 1 clinical trials with Annamycin targeting relapsed or refractory acute myeloid leukemia (AML) and the metastases of soft tissue sarcoma to the lungs (STS Lung), which concluded there was no evidence of cardiotoxicity (Press release, Moleculin, JAN 18, 2022, View Source [SID1234605545]). The review included analysis of ejection fraction, echo strain and certain troponin levels intended to assess potential for both acute and chronic heart damage. Additionally, the Company reported evidence that Annamycin may have a substantially lower incidence of alopecia (hair loss) than currently prescribed anthracyclines such as doxorubicin. Although 65%-92% of patients treated with doxorubicin typically experience hair loss, the incidence to date in patients treated with Annamycin is less than 10%1. Alopecia is considered an important factor in quality of life for many cancer patients.

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Two of the three clinical trials, as described more fully below, are ongoing and the data from those trials remain preliminary and are subject to change and future updates. The ongoing trials are both in the dose escalation phase (Phase 1) with the goal of determining the recommended Phase 2 dose (RP2D). In the course of this dose escalation, 19 of the 30 patients that have been reviewed to date have now been dosed above the lifetime maximum anthracycline limit currently set by the US Food and Drug Administration (FDA), further underscoring the potential for Annamycin to improve patient safety.

"Annamycin was designed to produce little to no cardiotoxicity, so we are pleased to see that this updated report continues to support that objective," commented Walter Klemp, Chairman and CEO of Moleculin. "This is a critical safety improvement in the field of anthracyclines since the risk of cardiotoxicity is the primary limitation in the use of currently prescribed anthracyclines. The apparent reduction in the incidence of hair loss is an added benefit that has the potential to significantly improve the quality of life for patients needing anthracycline therapy. We are also encouraged by the pace of recruitment for the STS Lung clinical trial and look forward to hopefully continuing the current pace of cohort updates. While the European AML trial has been slow in recruiting, we expect to announce an update regarding the next cohort in the first quarter of 2022."

1 Gonzalez et al. 2018; DOXORUBICIN HYDROCHLORIDE [package insert]. New York, NJ: Pfizer Injectables; 2019

An expert in assessing cardiotoxicity associated with chemotherapy at the Cleveland Clinic, the author of the independent review of Annamycin’s cardiac safety data added, "Although anthracyclines continue to be a cornerstone of chemotherapy for many cancer indications, their use has been limited due to the threat of both acute and chronic cardiotoxicity. The availability of an anthracycline that eliminated this risk would be a major advancement in patient safety."

Dr. Sant Chawla, Director of the Sarcoma Oncology Center, Director of the Cancer Center of Southern California, and a Principal Investigator in Moleculin’s STS Lung clinical trial concluded, "Patients often face difficult choices in an effort to balance the objectives of cancer therapy with quality of life. Having the ability to offer my patients a treatment alternative that substantially reduces the risk of alopecia would make Annamycin a valuable new alternative for patients who until now have had limited options."

Summary of Annamycin Clinical Trials

STS Lung Clinical Trial

The Phase 1b/2 study is an ongoing U.S. multi-center, open-label, single-arm study, which in its Phase 1b stage, will determine the maximum tolerated dose (MTD) or the recommended Phase 2 dose (RP2D) and safety of Annamycin. The Phase 2 portion of the study will explore the efficacy of Annamycin as a single agent for the treatment of subjects with STS with lung metastases for whom prior chemotherapy has failed, and for whom new chemotherapy is considered appropriate. A minimum of three subjects will be enrolled in each cohort of the Phase 1b portion of the study until an MTD is identified, after which there will be a recommendation for the RP2D based on an assessment of both safety and efficacy. Up to 25 subjects will be enrolled at the RP2D in Phase 2 to further evaluate efficacy. The Company has now opened enrollment in the fourth cohort of the Phase 1b portion of the study with dosing increased to 390 mg/m2. Three subjects minimum (6 maximum) for this and each subsequent dosing cohort will be enrolled until a maximum tolerated dose is identified. Therefore, up to 36 subjects may be enrolled in the Phase 1b portion of the study.

AML Europe Phase 1/2 Clinical Trial

The Phase 1/2 AML trial in Poland remains ongoing and is currently dosing patients at 240 mg/m2. Under the previous protocol transient elevated liver enzymes (AST and ALT) observed in two patients were initially deemed to be a dose limiting toxicity (DLT), however investigators believed this would inappropriately limit the potential for continued dose escalation. An amendment to the Annamycin clinical trial protocol was therefore undertaken, which allows for a change in the DLT criteria as it relates to transient grade 3 elevations and allows for the dosing of three additional patients in the 240 mg/m2 cohort. If no DLT (as defined by the new criteria) is experienced with these next three patients, the Company plans to escalate dosing in new cohorts by 30 mg/m2 instead of the 60 mg/m2 previously planned, and with a de-escalation of 15 mg/m2 at the DLT dose if future patients experience a DLT.

AML US Phase 1/2 Clinical Trial

The results from the Phase 1 portion of the Company’s U.S. Phase 1/2 clinical trial of Annamycin for the treatment of AML met its primary endpoint and demonstrated a clean safety profile with no evidence of cardiotoxicity when delivered to patients at or below the lifetime maximum anthracycline dose established by the FDA. The Company is awaiting data from its European AML trial prior to continuing with the Phase 2 portion of this trial.

About Annamycin

Annamycin is the Company’s next-generation anthracycline that has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in multiple human clinical trials, including ongoing trials for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases, and the Company believes that the use of Annamycin may not face the same usage limitations imposed on doxorubicin, one of the most common currently prescribed anthracyclines. Annamycin is currently in development for the treatment of AML and STS lung metastases and the Company believes it may have the potential to treat a number of additional indications.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of STS lung metastases, in addition to Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia. For more information about the Phase 1b/2 study evaluating Annamycin for the treatment of STS lung metastases, please visit clinicaltrials.gov and reference identifier NCT04887298.

On January 17, 2022 Midatech Pharma PLC (AIM: MTPH; Nasdaq: MTP), a drug delivery technology company focused on improving the bio-delivery and biodistribution of medicines, reported that it is pleased to announce the extension of its R&D collaboration with Janssen Pharmaceutica NV (Janssen) originally announced on 21 July 2020 (Press release, Midatech Pharma, JAN 18, 2022, View Source [SID1234605544]).

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On 17 June 2021, the Company announced that, using its Q-Sphera technology, it had successfully encapsulated a proprietary Janssen experimental large molecule medicine and importantly preserved its functional integrity. The Company believes no other commercial or academic organisation has been able to successfully deliver any such experimental medicine over extended periods using methods capable of commercial scaling.

Building on this important discovery, the Company has extended its R&D collaboration with Janssen. Under the extended collaboration the Company will focus on maximizing drug loading and optimizing in vitro duration of release for this undisclosed Janssen experimental molecule using the Company’s Q-Sphera technology.

Dmitry Zamoryakhin, Chief Scientific Officer of Midatech, said:

"We are excited about the potential of our Q-Sphera technology in the delivery of API via long acting injectables. We look forward to our continued collaboration with Janssen and the application of our technology specific to these APIs."

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014 (MAR).