On October 29, 2020 HOOKIPA Pharma Inc. (NASDAQ: HOOK), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported the first patient dosed with HB-202, a replicating arenavirus therapy based on the Pichinde Virus (PICV) (Press release, Hookipa Pharma, OCT 29, 2020, View Source [SID1234569371]). HB-202 is part of a sequential alternating regimen of HB-202/HB-201 for the treatment of Human Papillomavirus 16-positive (HPV16+) cancers in the ongoing HB-201 Phase 1/2 trial (NCT04180215).

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"We are excited to expand our ongoing Phase 1/2 trial in HPV to explore the potential of combining HB-202 and HB-201 as an alternating two-vector therapy to enhance and focus the immune response against HPV16+ cancers," said Joern Aldag, Chief Executive Officer of HOOKIPA. "HPV-associated cancers remain an area of unmet need and a key focus for our oncology efforts. The start of this HB-202/HB-201 arm of our trial is an important achievement in our clinical development of improved therapeutics for people with HPV16+ cancers."

HB-202 and HB-201 are engineered using HOOKIPA’s replicating arenaviral vector platform. They are designed to use different arenavirus backbones (PICV for HB-202 and LCMV for HB- 201), while expressing the same antigen, an E7/E6 fusion protein derived from HPV16. In pre-clinical studies, alternating administration of HB-202 and HB-201 resulted in a ten-fold increase in immune response and better disease control than either compound alone.

The alternating, sequential HB-202/HB-201 two-vector therapy has been incorporated into the ongoing Phase 1/2 trial (NCT04180215). As such, the first patient dosed in the alternating HB-202/HB-201 arm received Dose Level 1 of HB-202 and is scheduled to receive Dose Level 2 of HB-201. This Phase 1/2 clinical trial is an open-label dose-escalation and dose-expansion trial in patients with treatment-refractory HPV16+ cancers. In addition to the newly added HB-202/HB-201 therapy option, patients will continue to be enrolled into the HB-201 dose groups.

The primary endpoint of the Phase 1 is a recommended Phase 2 dose based on safety and tolerability. Secondary endpoints include anti-tumor activity as defined by RECIST 1.1 and immunogenicity. Patients will receive HB-202/HB-201 via intravenous dosing or, for patients with an accessible lesion, the first dose of HB-201 will be delivered via intratumoral injection and the remaining doses of HB-202 and HB-201 will be administered intravenously.

The Phase 2 portion of the trial will investigate the efficacy of our arenaviral regimens alone and also in combination with a PD-1 inhibitor. While HOOKIPA intends to release preliminary data for the HB-201 arm of the trial in late 2020 or early 2021, the Company expects to provide interim safety, dose escalation, and efficacy data on the HB-202/HB-201 arm in mid-2021.

About Human Papillomavirus
Human Papillomavirus, or HPV, is estimated to cause about 5% of the worldwide burden of cancers. This includes approximately 99% of cases in cervical, up to 60% of head and neck, 70% of vaginal, and 88% of anal cancers.

The majority of these cancers are caused by the HPV serotype 16. Most infections with HPV are cleared from the body with no lasting consequences. However, in some cases, HPV DNA becomes integrated into chromosomal DNA. When host cells take up this DNA, they express the HPV E6 and E7 proteins. This uptake can potentially lead to cancer since expression of these proteins leads to alterations in cell cycle control, which in turn predisposes these cells to become cancerous.

On October 29, 2020 ERYTECH Pharma (Euronext Paris: ERYP – Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported that it will host a Third Quarter 2020 conference call and webcast on Friday, November 6, 2020, at 2:30 PM CET/8:30 AM EST to discuss operational highlights (Press release, ERYtech Pharma, OCT 29, 2020, View Source,ET%20%7C%20Source%3A%20Erytech%20Pharma%20S.A.&text=ERYTECH%20is%20a%20clinical%2Dstage,of%20cancer%20and%20orphan%20diseases. [SID1234569370]).

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The call is accessible via the below teleconferencing numbers, followed by the Conference ID#: 2943988#

USA/Canada: +1 (833) 818-6807 France: +33 1 70 80 71 53
International Dial-In Number: +1 (409) 350-3501 United-Kingdom: +44 2031070289
The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing + 1 855 859 2056, Conference ID: 2943988#.

An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

On October 29, 2020 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported interim data from its BPX-601 dose-escalation clinical trial in patients with relapsed/refractory metastatic pancreatic cancer (Press release, Bellicum Pharmaceuticals, OCT 29, 2020, View Source [SID1234569369]). Findings from the first four patients treated with BPX-601 followed by repeat rimiducid dosing showed evidence of rimiducid-mediated CAR-T cell activation. Clinically meaningful efficacy as measured by RECIST criteria was not observed.

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After an extensive review of its organization and programs, the company has implemented a restructuring plan, including a reduction in staff, to focus its efforts on its clinical GoCAR-T product candidates. For BPX-601, the first candidate incorporating iMC, the company expects to begin enrolling patients with metastatic castration-resistant prostate cancer (mCRPC) in the ongoing Phase 1/2 clinical trial before the end of the year and intends to review its plans in pancreatic cancer upon completion of the current safety cohort. For BPX-603, the company’s first dual-switch GoCAR-T candidate, Bellicum expects to initiate enrollment of patients with HER2+ solid tumors in a Phase 1/2 clinical trial also by the end of the year. In order to preserve operating capital for these clinical trials, the company plans to pause development of its BCMA GoCAR-NK program.

"The results we have observed in the BPX-601 study are encouraging in terms of safety and GoCAR-T cell activation, proliferation, and persistence. We are eager to investigate our technology further in new tumor types like mCRPC and against established target antigens like HER2," said Rick Fair, President and Chief Executive Officer of Bellicum. "We have concluded that Bellicum must reduce spending on preclinical programs and shift its resources to enable achievement of meaningful milestones in the clinic. We regret the impact this unavoidable decision will have on our departing employees and we sincerely thank them for their contributions and dedication."

Interim BPX-601 Data

As of July 9, 2020, four patients were treated with 5×106 BPX-601 cells/kg followed by 2-11 doses of rimiducid in cohort 5C of the Phase 1 dose escalation clinical trial. Interim results include the following observations:

Administration of BPX-601 and repeat doses of rimiducid was tolerated as follows:

No treatment-related adverse events ³Grade 2 were observed in these four patients; one treatment-related SAE (Grade 4 cytokine release syndrome) was reported in a patient treated after data cutoff

One genitourinary adverse event was reported (Grade 1 intermittent hematuria)

Four events of Grade 1 neurotoxicity (neurotoxicity, dysgraphia, and confusion x2) were reported in two patients

The safety profile observed was otherwise consistent with previous reports

Best Overall Response in these patients included 3 stable disease and 1 progressive disease

Evidence of repeat rimiducid-mediated CAR-T cell activation was observed as follows:

Rimiducid administration was associated with increased serum cytokine levels, including IL-5, TNF-α, and IFN-g

Rimiducid treatment was also associated with increased expression of activation markers (e.g. CD25) on peripheral CD4+ and CD8+ T cells, indicative of systemic immune modulation via BPX-601 iMC activation

In two evaluable subjects receiving >2 doses of rimiducid, repeat rimiducid dosing was not shown to increase the peak or AUC of circulating BPX-601 cells relative to single-dose rimiducid

Consistent with previous cohorts, rimiducid administration was associated with a transient decline followed by partial recovery in circulating BPX-601 cells

Corporate Restructuring

Under the restructuring program, the company will focus on the clinical development of BPX-601 and BPX-603, pause the BCMA GoCAR-NK program, and discontinue discovery research and new product development. Staff will be reduced by 79%, from 68 to 14 full-time employees by the end of 2020, and Bellicum expects to incur severance expenses of approximately $2.5 million. The company also intends to pay down all of its Oxford Finance debt obligations using cash on hand with payment of $27.4 million in principal plus applicable fees and accrued interest on or before October 30, 2020. These actions are expected to reduce the company’s expenses and extend its cash runway. The company now expects annual cash utilization of $25 to $30 million.

On October 29, 2020 Idera Pharmaceuticals, Inc. ("Idera" or the "Company") (Nasdaq: IDRA) reported its financial and operational results for the third quarter ended September 30, 2020 (Press release, Idera Pharmaceuticals, OCT 29, 2020, View Source [SID1234569368]).

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"Tilsotolimod is the most advanced TLR-9 agonist therapy in development, and we have made tremendous strides against our objectives for 2020," stated Vincent Milano, Idera’s Chief Executive Officer. "We continue to work diligently against our timelines for ILLUMINATE-301, which currently remain on track for data in the first quarter of 2021. In addition, our recent patent and allowed application for tilsotolimod method-of-use in colorectal and head and neck cancers as well as the continuation of ILLUMINATE-206 reinforce our ‘beyond melanoma’ strategy. We also have the financing to help our outstanding team continue to execute these key objectives."

Corporate Update

Since June 30, 2020, the following corporate updates were announced:

·The Company entered into a private placement of up to $20.0 million, with $5.1 million received in July 2020. The Company anticipates that its current cash, cash equivalents, and short-term investments will fund our operations through the second quarter of 2021. With this private placement, the Company has now entered into three financing vehicles since December 2019, which it believes could provide proceeds of up to $118.2 million to fund the potential NDA filing and commercial launch of tilsotolimod.
·The Company received a new U.S. Patent and allowed application for tilsotolimod, providing exclusivity through September 2037 when intratumoral tilsotolimod is used with certain immune checkpoint inhibitors in treating colorectal cancer (CRC) and head and neck squamous cell carcinoma (HNSCC).

ILLUMINATE (tilsotolimod) Clinical Development Updates

ILLUMINATE-301: Randomized phase 3 trial of tilsotolimod in combination with Yervoy* (ipilimumab) versus Yervoy alone in patients with anti-PD-1 refractory advanced melanoma:

•Primary endpoint family of overall response rate (ORR) by blinded independent central review using RECIST v1.1 and overall survival (OS);
•Trial initiated in March 2018;
•Enrollment completed in March 2020; and
•ORR and other preliminary data expected in the first quarter of 2021.

ILLUMINATE-206: Phase 2, open-label, multicohort, multicenter study to test the safety and effectiveness of tilsotolimod in combination with Yervoy and Opdivo* (nivolumab) for the treatment of solid tumors:

·Trial initiated in September 2019 with the microsatellite stable colorectal cancer (MSS-CRC) cohort;
·Initial safety run-in of 10 patients, which included Yervoy at 1 mg/kg every 8 weeks and Opdivo at 3 mg/kg every 2 weeks, showed that the regimen was generally well tolerated;
·Changes in the study design intended to improve potential outcomes in this patient population include increasing Yervoy dosing frequency to every 3 weeks and limiting the number of allowed prior lines of treatment to 2; and
·The Company has opened enrollment for the next 10 patients under the modified study design, with data anticipated in the second quarter of 2021.

ILLUMINATE-204: Phase 1/2 trial of tilsotolimod in combination with Yervoy or Keytruda± (pembrolizumab) in patients with anti-PD-1 refractory advanced melanoma:

·Final results from the recommended phase 2 dose (RP2D) of 8 mg of tilsotolimod in combination with Yervoy, which is the treatment regimen being evaluated in the Company’s registrational trial, ILLUMINATE-301, were shared in a Mini Oral presentation at the ESMO (Free ESMO Whitepaper) Virtual Congress in September 2020.

Third Quarter Financial Results

Research and development expenses for the three months ended September 30, 2020, totaled $4.8 million compared to $8.4 million for the same period in 2019. General and administrative expense for the three months ended September 30, 2020, totaled $2.7 million compared to $3.0 million for the same period in 2019. Additionally, during the three months ended September 30, 2020, we recorded $0.7 million and $12.4 million non-cash warrant revaluation loss and non-cash future tranche right revaluation loss, respectively, related to securities issued in connection with our December 2019 private placement transaction.

As a result of the factors above, net loss applicable to common stockholders for the three months ended September 30, 2020, was $20.6 million, or $0.59 per basic and diluted share, compared to net loss applicable to common stockholders of $11.1 million, or $0.39 per basic and diluted share, for the same period in 2019. Excluding the non-cash loss of approximately $13.1 million for the three months ended September 30, 2020, related to the securities issued in connection with the December 2019 private placement transaction, net loss applicable to common stockholders was $7.5 million, or $0.21 per basic and diluted share (calculated based upon the basic weighted-average number of common shares, due to the antidilutive effect of net loss).

As of September 30, 2020, our cash, cash equivalents, and short-term investments totaled $29.0 million. Based on our current operating plan, we anticipate that our current cash, cash equivalents, and short-term investments, will fund our operations through the second quarter of 2021.

On October 29, 2020 Illumina, Inc. (NASDAQ: ILMN) reported its financial results for the third quarter of fiscal year 2020 (Press release, Illumina, OCT 29, 2020, View Source [SID1234569367]).

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Third quarter 2020 results reflect strong sequential growth:

•Revenue of $794 million, an increase of 26% compared to the second quarter of 2020 and a 12% decrease compared to $907 million in the prior year period
•GAAP net income attributable to Illumina stockholders for the quarter of $179 million, or $1.21 per diluted share, compared to $234 million, or $1.58 per diluted share, for the prior year period
•Non-GAAP net income attributable to Illumina stockholders for the quarter of $150 million, or $1.02 per diluted share, compared to $286 million, or $1.93 per diluted share, for the prior year period. Non-GAAP net income excludes net gains from mark-to-market adjustments on our strategic investments, primarily from our marketable equity securities (see the "Reconciliation Between GAAP and Non-GAAP Net Income Attributable to Illumina Stockholders" table for a reconciliation of these GAAP and non-GAAP financial measures)
•Cash flow from operations of $153 million compared to $267 million in the prior year period
•Free cash flow (cash flow from operations less capital expenditures) of $105 million for the quarter compared to $218 million in the prior year period

"Our business accelerated in the third quarter with sequencing consumable revenue growing 29% from the second quarter," said Francis deSouza, Chief Executive Officer. "We’re also making progress incorporating genomics into the standard of care in non-invasive pre-natal testing, oncology therapy selection, and genetic disease diagnosis. Looking forward, we believe our planned acquisition of GRAIL will catalyze a new era of early cancer detection, transforming cancer survivability and opening up the largest clinical application of genomics we’ve seen."

Gross margin in the third quarter of 2020 was 66.2% compared to 71.5% in the prior year period. Excluding amortization of acquired intangible assets and the net impact from expenses and payroll credits related to COVID-19, non-GAAP gross margin was 67.4% for the third quarter of 2020 compared to 72.5% in the prior year period.

Research and development (R&D) expenses for the third quarter of 2020 were $172 million compared to $151 million in the prior year period. Excluding expenses related to COVID-19, non-GAAP R&D expenses as a percentage of revenue were 21.2% compared to 16.4% in the prior year period.

Selling, general and administrative (SG&A) expenses for the third quarter of 2020 were $192 million compared to $189 million in the prior year period. Excluding amortization of acquired intangible assets, gain on litigation, acquisition-related expenses, and expenses related to COVID-19, non-GAAP SG&A expenses as a percentage of revenue were 24.8% compared to 20.0% in the prior year period.

Depreciation and amortization expenses were $46 million and capital expenditures for free cash flow purposes were $48 million during the third quarter of 2020. At the close of the quarter, the company held $3.3 billion in cash, cash equivalents and short-term investments, compared to $3.4 billion as of December 29, 2019.
Updates since our last earnings release:

•Announced a definitive agreement to acquire GRAIL to accelerate commercialization and adoption of transformative multi-cancer screening genetic tests
•Released the NovaSeqTM 6000 v1.5 reagents to unlock deeper genomic discoveries by making a $600 genome more broadly available and catalyzing the next wave of adoption in smaller core labs
•Updated guidelines from the American College of Obstetricians and Gynecologists (ACOG) recommended Non-Invasive Prenatal Testing (NIPT) be made available to all pregnant women, regardless of maternal age or baseline risk
•Repurchased approximately $125 million of common stock in the third quarter with $295 million remaining for repurchase under our current plan
•Appointed Kathryne Reeves as Chief Marketing Officer

Financial outlook and guidance

As previously announced, Illumina has withdrawn its fiscal 2020 full year revenue and earnings per share guidance due to the uncertainties around the severity and duration of the COVID-19 pandemic.

Quarterly conference call information

The conference call will begin at 2:00 pm Pacific Time (5:00 pm Eastern Time) on Thursday, October 29, 2020. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website under the "Company" tab at www.illumina.com. Alternatively, individuals can access the call by dialing 1 (866) 211-4597 or 1 (647) 689-6853 outside North America, both with conference ID 9488740.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

Statement regarding use of non-GAAP financial measures

The company reports non-GAAP results for diluted net income per share, net income, gross margins, operating expenses, operating margins, other income, and free cash flow in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The company’s financial measures under GAAP include substantial charges such as amortization of acquired intangible assets, non-cash interest expense associated with the company’s convertible debt instruments that may be settled in cash, and others that are listed in the itemized reconciliations between GAAP and non-GAAP financial measures included in this press release. Management has excluded the effects of these items in non-GAAP measures to assist investors in analyzing and assessing past and future operating performance. Additionally, non-GAAP net income attributable to Illumina stockholders and diluted earnings per share attributable to Illumina stockholders are key components of the financial metrics utilized by the company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

The company encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP information and the reconciliation between these presentations, to more fully understand its business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.