On November 4, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the quarter ended September 30, 2019 and provided an update on the launch of INGREZZA (valbenazine) and its clinical development programs (Press release, Neurocrine Biosciences, NOV 4, 2019, View Source [SID1234550240]).

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 pleased that a record number of new patients initiated treatment with INGREZZA as healthcare providers continue to recognize and treat the involuntary movements associated with tardive dyskinesia," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Our development programs continue to progress including engagement with regulatory agencies on the adult CAH pivotal trial design. We remain focused on providing patients with access to INGREZZA and preparing for the approval of opicapone in the U.S., while investing strategically to position the company as a leading global biopharmaceutical organization."

Financial Results

Total revenues for the three and nine months ended September 30, 2019, were $222.1 million and $544.1 million, respectively, compared to $151.8 million and $319.7 million for the same periods in 2018.

Total revenues were comprised of the following (unaudited):

Collaboration revenue reflects event-based milestones and royalties earned under the Company’s collaboration agreement with AbbVie. During the third quarter of 2019, the Company recognized a $20 million event-based milestone as revenue upon the U.S. Food & Drug Administration (FDA) acceptance of AbbVie’s New Drug Application (NDA) submission of elagolix for the treatment of uterine fibroids. During the third quarter of 2018, the Company recognized a $40 million event-based milestone as revenue upon the FDA approval of ORILISSA (elagolix) for the management of endometriosis with associated moderate to severe pain.

For the third quarter of 2019, the Company reported net income of $53.8 million, or $0.56 diluted earnings per share, compared to net income of $50.8 million, or $0.52 diluted earnings per share, for the same period in 2018. The increase in net income is due to increased INGREZZA net product sales partially offset by continued INGREZZA investment and a $28.5 million unrealized loss on the Company’s Voyager Therapeutics equity investment. For the nine months ended September 30, 2019, the Company reported net income of $3.0 million, or $0.03 diluted earnings per share, compared to net income of $3.0 million, or $0.03 diluted earnings per share, for the same period in 2018. Net income for the first nine months of 2019 reflects increased INGREZZA net product sales offset by $118.1 million of in-process research and development (IPR&D) in connection with the strategic collaboration with Voyager.

Research and development (R&D) expenses for the three and nine months ended September 30, 2019, were $45.3 million and $144.6 million, respectively, compared to $35.5 million and $121.4 million for the same periods in 2018. The increase in R&D expenses for both periods is primarily due to funding of development activities in connection with the Voyager transaction.

In further connection with the Voyager collaboration, the Company recognized IPR&D of $118.1 million during the first nine months of 2019. In addition, the Company made an equity investment in Voyager which is required to be marked to market each quarter, resulting in an unrealized loss of $28.5 million and $5.8 million for the third quarter and first nine months of 2019, respectively, and is reflected in Other Expense.

Sales, general and administrative (SG&A) expenses for the three and nine months ended September 30, 2019, were $84.5 million and $252.9 million, respectively, compared to $60.4 million and $180.0 million for the same periods in 2018. The increase in SG&A expenses for both periods is primarily due to the sales force expansion completed in the third quarter of 2018, the national launch of a patient-focused disease state awareness campaign, Talk About TD, and an increase in the Branded Pharmaceutical Drug fee expense.

As of September 30, 2019, the Company’s cash and available-for-sale investments was $875.0 million.

Updated 2019 SG&A and R&D Expense Guidance

SG&A, IPR&D, and R&D expenses for 2019 are expected to be $658 million to $668 million. Ongoing SG&A and R&D expenses for 2019, excluding IPR&D, are now expected to approximate $540 million to $550 million, which compares to the prior SG&A and R&D expense guidance of $540 million to $570 million.

Pipeline Highlights

Valbenazine Update – Chorea Associated with Huntington’s Disease

In September 2019, the Company initiated KINECT-HD, a Phase III study of valbenazine for the treatment of chorea associated with Huntington’s disease. This is a multicenter, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of once-daily valbenazine in up to 120 adult patients over 12 weeks of treatment. The primary endpoint of this study is the comparison of the change from baseline of the Total Maximal Chorea sub-score of the Unified Huntington’s Disease Rating Scale between placebo and active treatment groups using the average of week 10 and week 12 scoring. Top-line data are expected in 2021.

Opicapone Update

In February 2017, the Company entered into an exclusive licensing agreement with

BIAL – Portela & CA, S.A. (BIAL) for the development and commercialization of opicapone in

the United States and Canada. Opicapone is a once-daily, oral, selective catechol-O-methyltransferase inhibitor, being developed as an adjunctive therapy to levodopa/carbidopa in patients with Parkinson’s disease experiencing OFF episodes. The Company met with the FDA in January 2018 and based upon the BIPARK-1 and BIPARK-2 pivotal Phase III studies conducted by BIAL, the FDA did not require additional Phase III trials to form an NDA submission. The NDA for opicapone was submitted to the FDA during the second quarter of 2019. The NDA was accepted by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 26, 2020.

Elagolix Update

On July 24, 2018, AbbVie, in collaboration with Neurocrine Biosciences, announced FDA approval and in October 2018 Health Canada approval for ORILISSA for the management of endometriosis with associated moderate to severe pain.

AbbVie provided positive top-line efficacy data from two Phase III studies in women with uterine fibroids in the first quarter of 2018 and from the associated six-month safety extension study during the third quarter of 2018. The ELARIS UF-I and UF-II studies of elagolix met all primary and ranked secondary endpoints at month six. The NDA for uterine fibroids was submitted to the FDA and accepted during the third quarter of 2019 with a PDUFA target action date in the second quarter of 2020. With the FDA acceptance of the NDA, a $20 million event-based milestone was recognized as revenue in the third quarter with a payment to be made by AbbVie during the fourth quarter of 2019.

AbbVie initiated a Phase II study of elagolix in women with polycystic ovary syndrome (PCOS) during the third quarter of 2019.

Congenital Adrenal Hyperplasia (CAH) Program (NBI-74788) Update

The Company began an adaptive, Phase II proof-of-concept study examining the pharmacokinetics, pharmacodynamics, and safety of NBI-74788 in adults with classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH) in November 2017. This study evaluates the safety and tolerability of NBI-74788 in patients with CAH together with the relationship between exposure and specific steroid hormone levels in these patients. In March 2019, positive interim results from this ongoing study demonstrated a clinically meaningful reduction in key biomarkers associated with the management of CAH. NBI-74788 was shown to be well tolerated with no serious adverse events reported to date.

In July 2019, the Company initiated an adaptive, Phase II proof-of-concept study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NBI-74788 in pediatric patients with classic CAH. In Q3 2019, the Company engaged with the FDA and the European Medicines Agency (EMA) to discuss the registrational trial design for the adult program.

Voyager Collaboration and VY-AADC Program

During the first quarter of 2019, Neurocrine Biosciences formed a strategic collaboration with Voyager Therapeutics focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine

Biosciences’ expertise in neuroscience, drug development and commercialization with Voyager’s innovative gene therapy programs targeting severe neurological diseases.

Based on the results from the VY-AADC Phase I programs in Parkinson’s disease, RESTORE-1, a Phase II, randomized, placebo-surgery controlled, double-blinded, multi-center, clinical trial was initiated to evaluate the safety and efficacy of VY-AADC in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary.

Conference Call and Webcast Today at 4:30 PM Eastern Time

Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 800-894-5910 (US) or 785-424-1052 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

About INGREZZA (valbenazine) Capsules
INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso, and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release from presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information

Contraindications

INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

Warnings & Precautions
Somnolence
INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation
INGREZZA may prolong the QT interval, although the degree of QT prolongation is not

clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Parkinsonism

INGREZZA may cause Parkinsonism in patients with tardive dyskinesia. Parkinsonism has also been observed with other VMAT2 inhibitors. Reduce the dose or discontinue INGREZZA treatment in patients who develop clinically significant parkinson-like signs or symptoms.

Adverse Reactions
The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.

On November 4, 2019 Myriad Genetics, Inc. (NASDAQ: MYGN, "Myriad" or the "Company"), a global leader in molecular diagnostics and precision medicine, reported financial results for its fiscal first-quarter 2020, provided an update on recent business highlights and provided revised fiscal year and second-quarter 2020 financial guidance (Press release, Myriad Genetics, NOV 4, 2019, View Source [SID1234550239]).

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 had a challenging start to fiscal year 2020 as hereditary cancer revenue accrual from small payers was impacted by the deletion of the historical hereditary cancer CPT codes. We had assumed this administrative change would have a minor impact to cash collections, but unfortunately, that has not proven to be the case. While the hereditary cancer business has returned to strong double-digit volume growth, the revenue accrual impact from these changes have led us to lower our financial outlook for the year," said Mark C. Capone, president and CEO, Myriad Genetics. "Despite this setback, we expect earnings to be significantly higher in the second half of the fiscal year and believe that a number of important upsides will materialize during the fiscal year generating momentum as we transition into fiscal year 2021."

Recent Business Highlights

Hereditary Cancer

In the fiscal first-quarter, Myriad made approximately an $11 million reserve adjustment for hereditary cancer revenue due to lower cash collections from small payers as a result of the deletion of the current procedural terminology (CPT) codes 81211 and 81213. As a result, the company has revised its revenue accrual rate, and is forecasting lower hereditary cancer rates for fiscal year 2020. Myriad will provide a detailed summary of the changes on its fiscal first-quarter 2020 earnings call.

Hereditary cancer volumes grew at a double-digit growth rate on a year-over-year basis for both the company’s oncology and women’s health business units.

GeneSight

Announced coverage decision from UnitedHealthcare, the largest commercial payer in the United States, covering GeneSight for patients that have a diagnosis of major depressive disorder or anxiety and have failed at least one prior medication.

Signed master service agreement with a large pharmacy benefit manager in the United States to offer GeneSight to its commercial payer and self-funded employer customers. A Fortune 50 company has already opted into the master service agreement.

Published the precision medicine analysis of the GUIDED study in the Journal of Clinical Psychiatry. The study evaluated 787 patients at baseline who were on medications with known gene drug interactions. The analysis showed that patients who had their treatment guided by GeneSight saw a 70 percent improvement in remission, 42 percent improvement in response, and a 23 percent improvement in symptoms, all of which were statistically significant.

Prenatal

Received acceptance for publication for new data in Prenatal Diagnosis demonstrating that Prequel is the only non-invasive prenatal screening (NIPS) test that outperforms traditional measures of aneuploidy detection across all classes of obesity. Other NIPS testing methodologies can have failure rates up to 24 percent in obese patients leading the American College of Gynecology to recommend against using NIPS in patients with significant obesity.

Prolaris

blished a clinical outcomes study in Personalized Medicine that demonstrated the Prolaris test can identify men with low-risk prostate cancer who can safely select active surveillance (AS) and defer the need for costly treatments such as radiation therapy or surgery. In the study of 664 men with low risk prostate cancer, 82.4 percent selected AS for their initial treatment and only 0.4 percent experienced disease progression. Additionally, the AS decision was durable with 91.2 percent of men remaining on AS at year one and 65.2 percent at year four.

Companion Diagnostics

Filed a supplementary Premarket Approval Application with the U.S. Food and Drug Administration (FDA) to authorize BRACAnalysis CDx as a companion diagnostic test for olaparib in metastatic, castrate-resistant, prostate cancer patients with germline BRCA mutations.

Received the first FDA approval for myChoice CDx as a companion diagnostic to identify women with ovarian cancer who are candidates for Zejula monotherapy in the late-line setting.

Submitted our myChoice CDx test for approval by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) in ovarian cancer.

Fiscal Year 2020 and Fiscal Second-Quarter 2020 Financial Guidance

Below is a table summarizing Myriad’s fiscal year 2020 and fiscal second-quarter 2020 financial guidance:

Revenue

Myriad’s fiscal year 2020 and second-quarter 2020 adjusted earnings per share guidance excludes the impact of stock based compensation expense, non-cash amortization associated with acquisitions and certain non-recurring expenses. These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The company will provide further details on its business outlook during the conference call today and discuss the fiscal first-quarter financial results and fiscal year 2020 financial guidance.

Conference Call and Webcast

A conference call will be held today, Monday, November 4, 2019, at 4:30 p.m. EDT to discuss Myriad’s financial results for the fiscal first-quarter, business developments and financial guidance. The dial-in number for domestic callers is 1-800-945-0427. International callers may dial 1-212-231-2918. All callers will be asked to reference reservation number 21931991. An archived replay of the call will be available for seven days by dialing (800) 633-8284 and entering the reservation number above. The conference call along with a slide presentation will also will be available through a live webcast at www.myriad.com.

On November 4, 2019 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported financial results for the third quarter ended September 30, 2019 (Press release, Mirati, NOV 4, 2019, View Source [SID1234550238]).

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!

Recent Corporate Updates:

Presented early clinical data from the Phase1/2 trial of MRTX849 on October 28, 2019 at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). At the highest dose (600 mg BID), three of five (3/5) evaluable patients with non-small cell lung cancer (NSCLC) and one of two (1/2) evaluable patients with colorectal cancer (CRC) achieved a partial response (PR); the remaining patients had stable disease (SD). Across all dose levels, three of six (3/6) patients with NSCLC and one of four (1/4) patients with CRC achieved a PR. Two responding patients (1 NSCLC and 1 CRC) achieved confirmed PRs, both with continuing tumor shrinkage following their first scan. The other two patients with PRs (both NSCLC) have not had confirmatory scans. Clinical PK data demonstrated that the dose of 600 mg BID results in drug levels that meet or exceed those likely to lead to full inhibition of KRAS G12C signaling. Treatment duration ranged from 6.7- 38.6 weeks as of the data cut-off, October 11, 2019.

Published data demonstrating the efficacy of MRTX849 in preclinical studies on October 28, 2019 simultaneously with the Company’s oral presentations at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), in Cancer Discovery, a journal of the American Association of Cancer Researchers.

Announced that the Company would have two presentations of interim Phase 2 data for sitravatinib in combination with nivolumab in urothelial carcinoma and oral cavity squamous cell carcinoma at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting on November 9, 2019.

Announced on July 9, 2019 that the Company had entered into a clinical collaboration agreement with Novartis to evaluate the combination of MRTX849, Mirati’s investigational KRAS G12C inhibitor and TNO155, Novartis’ investigational SHP2 inhibitor, in patients with advanced solid tumors with KRAS G12C mutations.

"The recent data presented demonstrate that MRTX849 treatment can result in clinical responses at well tolerated doses. We believe that MRTX849 clinical combinations, including the combination of MRTX849 with TNO155 in our collaboration with Novartis, will significantly increase the eligible patient population," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer at Mirati. "We continue to expand and accelerate our KRAS programs and expect to continue expanding our team, including the addition of commercial talent so that we are ready to deliver our novel cancer therapies to the patients most in need."

Financial Results for the Third Quarter 2019

Cash, cash equivalents, and short-term investments were $454.2 million at September 30, 2019, compared to $222.8 million at December 31, 2018. In January 2019, we completed a public offering of common stock that provided net cash proceeds of $107.9 million. In June 2019, we completed a public offering of common stock that provided net cash proceeds of $219.9 million.

License and collaboration revenues relate to the Collaboration and License Agreement between the Company and BeiGene, Ltd. ("BeiGene"), dated January 7, 2018. License and collaboration revenues for the three and nine months ended September 30, 2019 were $1.0 million and $2.8 million, respectively, compared to none and $9.5 million for the three and nine months ended September 30, 2018, respectively. The 2019 revenues relate to revenues earned in connection with a manufacturing supply services agreement with BeiGene and the 2018 revenues relate to the license the Company granted to BeiGene under the Collaboration and License Agreement.

Research and development expenses for the third quarter of 2019 were $47.4 million, compared to $23.6 million for the same period in 2018. Research and development expenses for the nine months ended September 30, 2019 were $119.9 million, compared to $67.1 million for the same period in 2018. The increase in research and development expenses is due to an increase in expense associated with the development of sitravatinib and MRTX849, as well as an increase in salaries and related expense, including an increase in share-based compensation expense. The Company recognized research and development-related share-based compensation expense of $8.6 million during the third quarter of 2019, compared to $1.8 million for the same period in 2018, and $20.4 million during the nine months ended September 30, 2019, compared to $5.1 million for the same period in 2018.

General and administrative expenses for the third quarter of 2019 were $10.7 million, compared to $5.3 million for the same period in 2018. General and administrative expenses for the nine months ended September 30, 2019 were $30.3 million, compared to $15.3 million for the same period in 2018. The increase is due primarily to an increase in share-based compensation expense and, to a lesser extent, an increase in employee related expense, professional services expense and facilities and insurance expense. The Company recognized general and administrative-related share-based compensation expense of $6.5 million during the third quarter of 2019, compared to $2.2 million for the same period in 2018, and $18.4 million during the nine months ended September 30, 2019, compared to $6.5 million for the same period in 2018.

Net loss for the third quarter of 2019 was $54.3 million, or $1.38 per share basic and diluted, compared to net loss of $27.6 million, or $0.85 per share basic and diluted for the same period in 2018. Net loss for the nine months ended September 30, 2019 was $140.9 million, or $3.83 per share basic and diluted, compared to net loss of $70.1 million, or $2.31 per share basic and diluted for the same period in 2018.

About Sitravatinib

Sitravatinib is an investigational spectrum-selective kinase inhibitor that potently inhibits receptor tyrosine kinases (RTKs), including TAM family receptors (TYRO3, Axl, Mer), split family receptors (VEGFR2, KIT) and RET. As an immuno-oncology agent, sitravatinib is being evaluated in combination with nivolumab (OPDIVO), an anti-PD-1 checkpoint inhibitor, in patients whose cancers have progressed despite treatment with a checkpoint inhibitor. Sitravatinib’s potent inhibition of TAM and split family RTKs may overcome resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment, enhancing antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation. Sitravatinib is being evaluated in multiple clinical trials to treat patients who are refractory to prior immune checkpoint inhibitor therapy, including the ongoing potentially registration-enabling Phase 3 trial of sitravatinib in combination with a checkpoint inhibitor in non-small

cell lung cancer (NSCLC). In addition, sitravatinib combinations with checkpoint inhibitors are being evaluated in selected checkpoint inhibitor naïve patients.

About MRTX849

MRTX849 is an investigational, orally-available small molecule that is designed to potently and selectively inhibit a form of KRAS which harbors a substitution mutation (G12C). KRAS G12C mutations are present in approximately 14% of non-small cell lung cancer adenocarcinoma patients, 4% of colorectal cancer patients, and subsets of other types of cancer. Tumors characterized by KRAS G12C mutations are commonly associated with poor prognosis and resistance to therapy, and patients with these mutations have few treatment options. MRTX849 is being evaluated in a Phase 1/2 trial treating patients with molecularly-identified, KRAS G12C-positive advanced solid tumors.

On November 4, 2019 MAX BioPharma, Inc. (www.maxbiopharma.com) reported that published two articles describing its anti-tumorigenic oxysterol lead compounds in the peer-reviewed journal, Cells (Press release, MAX BioPharma, NOV 4, 2019, View Source [SID1234550237]). Oxy186 (View Source) and Oxy210 (View Source) are derived from the Company’s Oxysterol Therapeutics platform of proprietary oxysterols. MAX BioPharma is currently optimizing the compounds for oral dosing and performing pre-clinical studies required by the FDA for clinical development and commercialization.

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!

MAX BioPharma’s anti-tumorigenic oxysterol, Oxy186, inhibits the Hedgehog signaling pathway, which is dysregulated and activated in many human tumors including pancreatic and lung cancers. Oxy210, an analogue of Oxy186, inhibits Hedgehog signaling as well as transforming growth factor (TGF)beta signaling. Oxy210 and Oxy186 have unique mechanisms of action compared to other Hedgehog and TGFbeta signaling inhibitors that are commercially available or under clinical development. MAX BioPharma is focusing its efforts to develop these compounds for lung and pancreatic cancers, which still have huge unmet medical needs. Oxy210 and Oxy186 have favorable pharmacokinetic and safety profiles, are scalable, and were found to inhibit tumor cell growth, invasive activity, and epithelial-mesenchymal transition, a hallmark of cancer metastasis. In addition, Oxy210 was found to inhibit development of chemoresistance by tumor cells and to enhance the cytotoxic effects of carboplatin on lung cancer cells in vitro.

MAX BioPharma has collaborated with Dr. Ying Zhang of the National Cancer Institute (NCI) under a Materials-Cooperative Research and Development Agreement (M-CRADA) and with academic investigators. These collaborations are evaluating the potential of the oxysterols to be potent, orally bioavailable, and safe therapies for cancer. "Given the important role of Hedgehog signaling in acute myeloid leukemia (AML) and the recent FDA approval of a Hedgehog pathway inhibitor, DaurismoTM (glasdegib) by Pfizer, for the treatment of AML, we are excited to begin examining the potential of oxysterols for targeting AML" explains Dr. William Matsui, co-founder of MAX BioPharma and Professor of Oncology and Deputy Director of LIVESTRONG Cancer Institutes at the University of Texas in Austin. MAX BioPharma is seeking strategic partnerships with biotechnology and pharmaceutical companies that have the expertise and resources to further the development of oxysterols as a cancer therapeutic towards FDA approval and commercialization. In addition, MAX BioPharma is in the process of raising a series A financing round to support the advancement of its therapeutic development programs. "We are extremely excited about the progress of our oncology program and we believe that our findings will be groundbreaking in finding more effective cures for cancer," says Dr. Farhad Parhami, President & CEO of MAX BioPharma.

On November 4, 2019 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, reported financial results from the third quarter and nine months ending September 30, 2019, and provided a corporate update (Press release, Iovance Biotherapeutics, NOV 4, 2019, View Source [SID1234550236]).

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 continue making great progress in developing tumor infiltrating lymphocyte (TIL) therapy, which could become the first approved cell therapy product for solid tumor indications," commented Maria Fardis, Ph.D., MBA, president and chief executive officer of Iovance Biotherapeutics. "Our pivotal studies in metastatic melanoma and advanced cervical cancer are on track to complete enrollment in early 2020. We expect to submit for regulatory approval for TIL therapies lifileucel and LN-145 in late 2020. These therapies have the potential to impact the lives of thousands of patients in the U.S. with melanoma or cervical cancer that have exhausted current treatment options. Furthermore, we are very pleased to have a new IND active in order to investigate the polyclonal blood-based T cell, or PBL therapy (IOV-2001), in chronic lymphocytic leukemia (CLL). This candidate was developed based on Iovance research efforts focused on the generation of novel cell therapy products. We anticipate the initiation of IOV-CLL-01, an Iovance-sponsored trial with IOV-2001 PBL product, before the end of 2019."

Recent Achievements and Upcoming Milestones

Clinical

·Completion of enrollment of registration-enabling Cohort 4 in the C-144-01 melanoma study is expected in the first quarter of 2020. A late-breaking abstract on Independent Review Committee (IRC)-read results from Cohort 2 will be presented at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting.
·To further expand on evaluating TIL for a broader cervical patient population, we have amended this protocol and added new cohorts. The pivotal cohort, cohort 1, will treat 75 patients, as planned, who are second line metastatic cervical cancer patients that have progressed during or following systemic therapy. Completion of enrollment of Cohort 1, the pivotal cohort, of the C-145-04 cervical cancer study is expected before mid-2020. Iovance has added new cohorts to the C-145-04 study in order to investigate TIL therapy in broader treatment settings. Enrollment in these additional cohorts will not impact the timing of the completion of the pivotal cohort nor the size of the registration program. The C-145-04 study has been amended to collect additional data on early-line patients as well as late-line patients. These additional cohorts also allow access to TIL therapy when enrollment in the registration Cohort 1 is completed.

·To further its strategy of the study of TIL therapy in additional solid tumors, Iovance and Yale University have initiated an Investigator Sponsored Trial with LN-145 in patients with metastatic triple negative breast cancer (TNBC). The IND has been accepted by the FDA and the trial is expected to begin enrollment in 2020.

Regulatory

·An IND application for IOV-2001, PBL therapy for patients with CLL, was accepted by the U.S. Food and Drug Administration (FDA) and the study has been cleared to proceed. IOV-CLL-01 is a company sponsored study currently active at one clinical site. Patient dosing is planned before the end of 2019. IOV-CLL-01 is a Phase 1/2 study evaluating safety and efficacy of IOV-2001 in patients with relapsed or refractory CLL or small lymphocytic lymphoma (SLL). The study is expected to enroll up to 70 patients.

Research

·Three preclinical abstracts highlighting Iovance TIL therapy will be presented at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting. These presentations will include three poster abstracts covering expansion of TIL from core biopsies, transient genetic knockdown of PD1 in TIL and Gen 2 TIL manufacturing results from several solid tumor types. SITC (Free SITC Whitepaper) meeting takes place from Nov. 6-10, 2019, at the Gaylord National Hotel and Convention Center in National Harbor, Maryland. The SITC (Free SITC Whitepaper) meeting abstract titles are listed at View Source
·Iovance entered into a collaboration with Lytix Biopharma, to study the activity of LTX-315, an oncolytic peptide, in conjuction with TIL therapy.
·The company intends to expand its hematologic research to include mantle cell lymphoma (MCL).

Manufacturing

·The Gen 2 TIL therapy manufacturing process continues to be robust with a demonstrated success rate, as measured from the receipt of the starting material to the shipment of TIL, of 93 percent. The manufacturing success rate is comparable with rates published for the approved cell therapy treatments.

Corporate

·The company has been granted a total of seven U.S. patents for compositions and methods of treatment in a broad range of cancers related to its 22-day Gen 2 manufacturing process.

SITC Late-Breaking Abstract Information

·Title: Safety and efficacy of lifileucel (LN-144) tumor infiltrating lymphocyte therapy in metastatic melanoma patients after progression on multiple therapies – independent review committee data update
·Author: Sarniak, A. et al.
·Abstract Number: P865
·Dates/Times: late-breaking abstract posters will be displayed Friday, Nov. 8, 2019, from 7 a.m. – 8 p.m. EST and Saturday, Nov. 9, 2019, from 7 a.m. – 8:30 p.m. EST

Third Quarter 2019 Financial Results

Net loss for the third quarter ended September 30, 2019, was $49.5 million, or $0.40 per share, compared to a net loss of $33.8 million, or $0.36 per share, for the third quarter ended September 30, 2018.

Research and development expenses were $41.6 million for the third quarter ended September 30, 2019, an increase of $13.7 million compared to $27.9 million for the third quarter ended September 30, 2018. The increase was primarily attributable to higher manufacturing costs resulting from increased capacity added to support enrollment in the pivotal and other clinical trials. In addition the increase is also due to higher personnel costs including stock-based compensation resulting from an increase in headcount as compared to the third quarter in 2018.

General and administrative expenses were $10.0 million for the third quarter 2019, an increase of $2.9 million compared to $7.1 million for the third quarter 2018. The increase was primarily attributable to increased personnel costs due to additional employees added in 2019 and additional legal fees to support the growing patent portfolio.

Nine Months Ended September 30, 2019, Financial Results

Net loss for the nine months ended September 30, 2019, was $134.0 million, or $1.08 per share, compared to a net loss of $91.0 million, or $1.01 per share, for the same period ended September 30, 2018.

Research and development expenses were $111.8 million for the nine months ended September 30, 2019, an increase of $39.4 million compared to $72.4 million for the same period ended September 30, 2018. The increase was primarily attributable to additional manufacturing and clinical trial costs resulting from higher enrollment in the clinical trials and increased personnel costs due to an increase in employees as compared to the same period in 2018.

General and administrative expenses were $30.0 million for the nine months ended September 30, 2019, an increase of $9.1 million compared to $20.9 million for the same period ended September 30, 2018. The increase was primarily attributable to higher personnel costs including stock-based compensation resulting from an increase in the number of employees and additional legal fees to support the patent portfolio.

Cash, cash equivalents, short term investments and restricted cash

At September 30, 2019, the company held $367.3 million in cash, cash equivalents, short-term investments and restricted cash compared to $468.5 million at December 31, 2018. The company anticipates that the year-end balance of cash, cash equivalents, short-term investments and restricted cash may be between $310 and $320 million.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss these third quarter 2019 results and provide a corporate update. The conference call dial-in numbers are 1-844-646-4465 (domestic) or 1-615-247-0257 (international). The conference ID access number for the call is 3482317. The live webcast can be accessed in the Investors section of the company’s website at View Source