On May 5, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the first quarter ended March 31, 2021 and provided revised full-year 2021 financial expense guidance (Press release, Neurocrine Biosciences, MAY 5, 2021, View Source [SID1234579183]).

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"Our first quarter results reflect a lower than normal refill rate per patient due to the typical seasonal payor dynamics for INGREZZA that were exacerbated by COVID. Importantly, we did not see an increase in discontinuations and exited the quarter with more patients on INGREZZA versus the prior quarter. New patient starts did pick up late in the quarter and we are focused on restoring INGREZZA growth as the impact of the pandemic wanes," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We made meaningful progress advancing our large, growing and diverse pipeline, including initiating a registrational study for the treatment of pediatric patients with congenital adrenal hyperplasia and a Phase II study in essential tremor. With important commercial products addressing patient needs and on track to initiate nine mid-to-late stage clinical studies this year, we are executing our plan to build a leading neuroscience-based company."

First Quarter Net Product Sales and Commercial Highlights:

INGREZZA net product sales for the first quarter of 2021 were $230 million and $227 million on an inventory adjusted basis
Annual seasonal payor dynamics resulted in lower refill rates per existing patient as compared to historical norms due to the COVID-19 pandemic and related disruption on timing of patient insurance reauthorizations
Exiting the first quarter, commercial activity and weekly new prescriptions both achieved their highest levels since the start of the pandemic reflecting an improved environment for diagnosis of tardive dyskinesia (TD). We expect second quarter inventory adjusted sales sequential dollar growth to be similar to 2020.
INGREZZA direct-to-consumer advertising campaign, "TD Spotlight", to be launched in May to educate patients about tardive dyskinesia and the benefits of INGREZZA
Financial Highlights:

First quarter 2021 GAAP net income and diluted earnings per share were approximately $32 million and $0.33, respectively, compared with approximately $37 million and $0.39, respectively, in the first quarter of 2020
First quarter 2021 non-GAAP net income and diluted earnings per share were approximately $48 million and $0.49, respectively, compared with approximately $79 million and $0.82, respectively, in the first quarter of 2020
Difference between first quarter 2021 GAAP and non-GAAP net income and diluted earnings per share vs. the first quarter of 2020 were driven by:
Increased Research and Development (R&D) expense primarily due to increased investment across our expanded pipeline programs and higher headcount costs
Increased Selling, General and Administrative (SG&A) expense primarily due to increased investment in commercial initiatives
At March 31, 2021, the Company had cash, cash equivalents and debt securities available-for-sale of $1.1 billion
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release.

Provision for Income Taxes:

First quarter 2021 benefit from income taxes was $4.9 million, compared with a provision for income taxes of $1.5 million in the first quarter of 2020. Our effective tax rate for the first quarter of 2021 was lower than federal and state statutory rates primarily due to excess tax benefits related to stock compensation. On December 31, 2020, the Company released substantially all of its valuation allowance against its net operating losses and other deferred tax assets. Beginning in the first quarter of 2021, the Company began recording a provision for income taxes using an effective tax rate approximating federal and state statutory rates. Due to our ability to offset our pre-tax income against previously benefited federal net operating losses, no federal cash tax is expected in 2021.

Recent Events

In February 2021, the Mitsubishi Tanabe Pharma Corporation (MTPC) reported positive top-line results from the J-KINECT Phase III Study, designed to evaluate the efficacy and safety of valbenazine in tardive dyskinesia. Detailed results from this trial will be presented at a future medical conference. In April 2021, MTPC submitted a Marketing Authorization Application (MAA) with the Ministry of Health and Welfare in Japan for valbenazine for the treatment of tardive dyskinesia. MTPC submission of valbenazine triggered a milestone payment of $15 million, to be paid by MTPC to Neurocrine Biosciences and recognized as collaboration revenue in the second quarter of 2021.
In February 2021, the Company notified Voyager Therapeutics, Inc. (Voyager) of the Company’s termination of the NBIb-1817 (VY-AADC) development program in Parkinson’s disease (the Program). The Company will work to transfer the rights to the Program to Voyager by August 2, 2021.
On March 2, 2021, the Company announced that investigational drug luvadaxistat (NBI-1065844/TAK-831) did not meet its primary endpoint in the Phase II INTERACT study in adults with negative symptoms of schizophrenia. Luvadaxistat met both secondary endpoints of cognitive assessment. The Company plans to initiate a Phase II study for the treatment of cognitive impairment associated with schizophrenia (CIAS) by the end of 2021.
In April 2021, the U.S. Food and Drug Administration (FDA) approved a 60 mg INGREZZA capsule for the treatment of adults with tardive dyskinesia. The 60 mg capsule of INGREZZA is expected to be available to patients by late second quarter 2021.
Previously, the Company expected combined GAAP R&D and SG&A expenses in the range of $800 million to $850 million and combined non-GAAP R&D and SG&A expenses in the range of $675 million to $725 million
Increase to GAAP and Non-GAAP expense guidance range primarily driven by investment in INGREZZA direct-to-consumer marketing campaign, "TD Spotlight"
GAAP-only guidance includes approximately $130 million of share-based compensation. GAAP-only guidance does not include any potential milestones or in-process research and development costs associated with current collaborations or future business development activities.
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 800-895-3361 (US) or 785-424-1062 (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.

(Press release, Neurocrine Biosciences, MAY 5, 2021, View Source [SID1234579183])

On May 5, 2021 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported a corporate and financial update for the first quarter ended March 31, 2021 (Press release, G1 Therapeutics, MAY 5, 2021, View Source [SID1234579203]).

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"The first quarter of 2021 was a transformational period for G1 as the approval and availability of COSELA provided extensive-stage small cell lung cancer patients undergoing chemotherapy with the first proactive multilineage myeloprotection therapy to help prevent myelosuppression," said Jack Bailey, Chief Executive Officer of G1 Therapeutics. "COSELA represents an opportunity to shift the treatment paradigm away from reactive treatments with multiple different single lineage therapeutic interventions. Our commercial team has demonstrated that we can effectively launch and deliver the drug during a very challenging time. Further, though very early in the launch period, leading and lagging indicators from the first four weeks of availability are encouraging and suggest that the awareness and interest in COSELA is strong, that it is being accepted by oncologists and oncology nurses, and that it is being well covered by payers. Another important step during the quarter was the inclusion of COSELA in two sets of NCCN Guidelines, which are the standard resource for determining best course of treatment and supportive care for people living with cancer. We also maintained clinical momentum in our tumor-agnostic development program for COSELA, including the initiation of PRESERVE 2, our Phase 3 registrational trial in triple-negative breast cancer."

First Quarter 2021 and Recent Highlights

Commercial

COSELA Approved by U.S. Food and Drug Administration (FDA): On February 12, 2021, the FDA approved COSELA for injection to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer (ES-SCLC). (Press release here)
COSELA Now Available in the U.S.: On March 2, 2021, G1 and its commercial partner Boehringer Ingelheim announced that COSELA was available through G1’s specialty distributors Amerisource Specialty Distribution, Oncology Supply, McKesson Plasma and Biologics, McKesson Specialty and Cardinal Specialty. (Press release here)
Medical

COSELA Added to Two National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines): On March 25, 2021, G1 announced that COSELA had been added to the Treatment Guidelines for Small Cell Lung Cancer and to the Supportive Care Guidelines for Hematopoietic Growth Factors. These guidelines document evidence-based, consensus-driven management to ensure that all patients receive preventive, diagnostic, treatment, and supportive services that are most likely to lead to optimal outcomes. (Press release here)
Presented New Budget Impact Data for COSELA at the Academy of Managed Care Pharmacy (AMCP) Meeting: These data described a model that compared ES-SCLC treatment scenarios with and without COSELA. The incremental cost of COSELA to a third-party payer is projected to be entirely offset by a reduction in the costs of managing adverse events related to myelosuppression. Therefore, the use of COSELA is estimated to provide cost savings.
Clinical

Initiated Pivotal Trial of COSELA in Patients Receiving First or Second Line Gemcitabine and Carboplatin Chemotherapy for Locally Advanced Unresectable or Metastatic Triple-Negative Breast Cancer (mTNBC): Patient enrollment is underway in PRESERVE 2, a randomized, double-blind, placebo-controlled Phase 3 registrational trial of COSELA in patients receiving first- or second-line gemcitabine/carboplatin (GC) chemotherapy for locally advanced unresectable or metastatic TNBC. (Press release here)
Entered Clinical Trial Collaboration for Upcoming First Line Locally Advanced or Metastatic Bladder Cancer (mUC) Trial of COSELA: G1 entered a clinical trial collaboration with the alliance between Merck KGaA, Darmstadt, Germany and Pfizer whereby the alliance will contribute clinical supply of the checkpoint inhibitor avelumab to the G1-sponsored and funded first-line mUC trial.
On Track to Initiate Two Phase 2 Trials of COSELA in First Line Metastatic Bladder Cancer (mUC) and Second Line / Third Line Non-Small Cell Lung Cancer (NSCLC) in the Second Quarter of 2021: The Company expects to initiate Phase 2 trials of COSELA in first-line treatment of locally advanced or metastatic bladder cancer (locally advanced or metastatic urothelial carcinoma, or mUC) and second- and third-line treatment of NSCLC, both of which are known immunogenic tumors, in the second quarter of 2021. Both trials are designed to evaluate the anti-tumor efficacy of COSELA.
First Quarter 2021 Financial Results

As of March 31, 2021, cash and cash equivalents totaled $279.0 million, compared to $207.3 million as of December 31, 2020. This includes $86.4 million in net proceeds in the first quarter from the "At the Market" offering with Cowen and Company, LLC. This ATM offering is now closed. In addition, the Company drew the additional $10M available in the first tranche of debt on its debt financing facility with Hercules Capital. The Company has access to an additional $70 million through this facility.

Total revenue for the first quarter of 2021 was $14.2 million, including approximately $0.6 million in initial net product sales of COSELA and license revenue of $13.6 million, primarily related to development milestone payments from the Company’s license agreement with Simcere and revenue from EQRx related to delivery of clinical drug supply and manufacturing services and reimbursement of clinical trial costs with EQRx. In addition, the Company recognized revenue related to achievement of a development milestone by Genor.

Operating expenses for the first quarter of 2021 were $39.8 million, compared to $31.8 million for the first quarter of 2020. GAAP operating expenses include stock-based compensation expense of $5.9 million for the first quarter of 2021, compared to $4.7 million for the first quarter of 2020.

Cost of goods sold expense for the first quarter of 2021 were $0.2 million, compared to $0 for first quarter of 2020. The increase related to the Company’s period costs for the sales of COSELA.

Research and development (R&D) expenses for the first quarter of 2021 were $16.5 million, compared to $20.4 million for the first quarter of 2020. The decrease in R&D expenses was primarily due to a decrease in costs for manufacturing of active pharmaceutical ingredients and drug product to support clinical trials, as well as a decrease in external costs related to discovery and pre-clinical development.

Selling, general and administrative (SG&A) expenses for the first quarter of 2021 were $23.0 million, compared to $11.4 million for the first quarter of 2020. The increase in SG&A expenses was largely due to an increase in compensation due to increases in headcount and increased commercialization activities.

The net loss for the first quarter of 2021 was $26.4 million, compared to $31.0 million for the first quarter of 2020. The basic and diluted net loss per share for the first quarter of 2021 was $(0.65) compared to $(0.82) for the first quarter of 2020.

Financial Guidance

The Company expects its current financial position to be sufficient to fund its operations and capital expenditures into 2023.

Webcast and Conference Call

G1 will host a webcast and conference call at 4:30 p.m. ET today to provide a corporate and financial update for the first quarter 2021 ended March 31, 2021. The live call may be accessed by dialing (866) 763-6020 (domestic) or (210) 874-7713 (international) and entering the conference code: 2509548. A live and archived webcast will be available on the Events & Presentations page of the company’s website: www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

Indication
COSELA (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

Important Safety Information
COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.

Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.

The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.

Please click here for full Prescribing Information. View Source

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.

On May 5, 2021 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the Company has received centralized Institutional Review Board (IRB) approval of the clinical trial protocol for its upcoming Acclaim-1 clinical trial in non-small cell lung cancer (NSCLC) (Press release, Genprex, MAY 5, 2021, View Source [SID1234579219]). Acclaim-1 is an open-label, multi-center Phase 1/2 clinical trial that combines the Company’s lead drug candidate, REQORSA immunogene therapy, with AstraZeneca’s Tagrisso (osimertinib) in patients with late-stage NSCLC with mutated epidermal growth factor receptors (EGFRs), whose disease progressed after treatment with Tagrisso.

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"The purpose of IRB review is to assure that appropriate steps are taken to protect the rights and welfare of individuals participating as subjects in clinical research," said Rodney Varner, President and Chief Executive Officer of Genprex. "With this centralized IRB approval, we have achieved another significant clinical milestone. We remain focused on completing our preparations for the Acclaim-1 clinical trial, and look forward to its commencement."

An IRB is a U.S. Food and Drug Administration (FDA) registered constituted group of medical professionals that are responsible for reviewing and monitoring biomedical research involving human subjects. In accordance with FDA regulations, an IRB has the authority to approve, require modifications (to secure approval), or disapprove research. IRB group review serves an important role in the protection of the rights, safety and welfare of human research subjects.

A centralized IRB review process involves an agreement under which multiple study sites in a multicenter trial can rely on the review of a centralized IRB, other than the IRB affiliated with each individual research site. The goal of the centralized process is to increase efficiency and decrease duplicative efforts, while enabling the central IRB to take responsibility for all aspects of IRB review at each site participating in the centralized review process.

In January 2020, the Company received FDA Fast Track Designation for its Acclaim-1 patient population. Genprex has recently completed the manufacturing and scaled-up clinical grade production of REQORSA to supply drug product for its upcoming Acclaim clinical trials.

The Company expects to conduct the Acclaim-1 clinical trial at approximately 15 U.S. clinical sites with participation of approximately 92 patients (with up to 18 patients in the Phase 1 component and up to 74 patients in the Phase 2 component). An interim analysis will be performed after 25 clinical events (i.e., disease progression or death). Additional information on the Acclaim-1 clinical trial can be found by visiting ClinicalTrials.gov.

On May 5, 2021 Alkermes plc (Nasdaq: ALKS) reported that management will participate in a virtual fireside chat at the BofA Securities 2021 Healthcare Conference on Wednesday, May 12, 2021 at 2:45 p.m. ET (7:45 p.m. BST) (Press release, Alkermes, MAY 5, 2021, View Source [SID1234579236]). The presentation may be accessed under the Investors tab on www.alkermes.com and will be archived for 14 days.

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On May 5, 2021 InDex Pharmaceuticals reported that interim report January – March 2021 (Press release, InDex Pharmaceuticals, MAY 5, 2021, View Source [SID1234579151])

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Period January – March 2021
Net sales amounted to SEK 0.0 (0.0) million
Operating loss amounted to SEK –9.3 (–24.0) million
Result after tax amounted to SEK –9.3 (–24.0) million, corresponding to SEK –0.02 per share (–0.10) before and after dilution
Cash flow from operating activities amounted to SEK –8.5 (–21.9) million
Cash and cash equivalents at the end of the period amounted to SEK 532.5 (104.6) million
Number of employees at the end of the period was 7 (7)
Number of shares at the end of the period was 532,687,650
All comparative amounts in brackets refer to the outcome during the corresponding period 2020.

Significant events during January – March 2021
The Board resolved on a fully guaranteed rights issue of approximately SEK 533 million
InDex’s rights issue was oversubscribed and the company received approximately SEK 488 million net
Significant events after the reporting period
No significant events have occurred after the reporting period
Other events during the reporting period
InDex entered an agreement for services with global clinical research organization (CRO) Parexel Biotech for the phase III study CONCLUDE
CEO statement
It has been an intensive beginning of 2021 for InDex, where the crucial pieces of the puzzle for the start of phase III now have fallen into place. In February, we completed a successful rights issue of approximately SEK 533 million. The subscription ratio amounted to as much as 153 percent and more than 99 percent was subscribed for by exercise of subscription rights. HBM Healthcare Investments and Handelsbanken Funds came in as new large owners in the rights issue. These are two internationally recognized and successful life sciences specialists that chose to invest significant amounts, which not only strengthens the ownership base, but also constitutes a strong validation of the potential of InDex.

The rights issue will primarily fund the important initial induction study in a sequential phase III program with cobitolimod for left-sided moderate to severe ulcerative colitis. The results of this induction study will constitute a significant value inflection point and the remaining program can be optimised according to the outcome of the study. We estimate that the study will take 18 to 24 months to complete from initiation.

The study, which has been named CONCLUDE, will be a global study with approximately 400 patients at a few hundred clinics. The primary endpoint, clinical remission, will be measured at week 6. Apart from the dosing 250 mg x 2, which was the highest dose and the one that showed the best efficacy in the phase IIb study CONDUCT, cobitolimod’s excellent safety profile allows to also evaluate a higher dose, 500 mg x 2, in an adaptive study design. This higher dose has the potential to provide an even better efficacy than what was observed in the CONDUCT study.

With the financing in place, we entered into an agreement for services at the end of March with Parexel Biotech for the phase III study. After the successful collaboration in CONDUCT, we are very pleased to collaborate once again with them as our clinical development partner. They are a leading global CRO with considerable experience managing phase III studies in inflammatory bowel disease, which will ensure an efficient execution of CONCLUDE. The clinical study must now be approved by the authorities of each participating country. The goal is to start the study in the second quarter of 2021, but it is subject to the development of the Covid-19 pandemic if authorities and healthcare providers will be able to prioritize the start of new clinical studies in the near future.

On June 3 we have the annual general meeting in InDex, but unfortunately there will not be a physical meeting this year either due to the pandemic. I will present the company at Redeye Growth Day the day before the annual general meeting, on June 2, for those who want an update. I will also present the company at Erik Penser Bank tomorrow, on May 6. You can follow the presentations live or watch them afterwards on our updated website.

I would also like to highlight the recent annual report for 2020. It provides a good overview of ulcerative colitis, cobitolimod, the phase III design and the company in general. Strong stories like our patient interview with 23-year-old Felicia confirm the need for new effective and safe treatment options for ulcerative colitis.

I look forward to a continued eventful year with the start of the phase III study CONCLUDE as the next important milestone.