On April 28, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI) reported that it will report first quarter 2020 financial results on Tuesday, May 5, 2020 (Press release, Karyopharm, APR 28, 2020, View Source [SID1234556685]). Karyopharm’s management team will host a conference call and audio webcast at 8:30 a.m. ET on Tuesday, May 5, 2020, to discuss the financial results and other company updates.

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To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 9936655. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

On April 28, 2020 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for people living with kidney disease, reported plans to release its financial results for the first quarter ended March 31, 2020, on Tuesday, May 5, 2020 before the opening of the financial markets (Press release, Akebia, APR 28, 2020, View Source [SID1234556701]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Akebia will host a conference call at 8:30 a.m. Eastern Time on Tuesday, May 5th, to discuss its first quarter financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 8464788. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through May 11, 2020. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 8464788. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On April 28, 2020 Immunovia reported that first quarter interim report for January – March 2020 (Press release, Immunovia, APR 28, 2020, View Source;march-2020-301048485.html [SID1234556721]). It is available on Immunovia’s website.

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"During Q1 2020, Immunovia worked hard and continued to make great strides towards IMMray PanCan-d’s launch and the sales start of the first test for early diagnosis of pancreatic cancer, in spite of the COVID-19 global pandemic.

Work on the verification study proceeded as planned and we received all the blood samples from our KOL network. In January, our commercial team prepared detailed plans for the launch and in March and April these plans were adapted significantly to accommodate the COVID-19 situation. This included making in-person activities, such as conferences and the patient organization meetings that were canceled, into digital activities, etc.

In January, Immunovia welcomed a new Senior VP Sales NA (North America), Michael Pettigrew, to the team. Michael brings with him a very successful track record in product launches and sales growth in life science, along with unique leadership qualities. He is an important addition to Immunovia.

Following the end of Q1 2020, we provided an update on the measures the company has taken and continues to take in order to support patients, employees and public health initiatives in response to the COVID-19 pandemic. We also announced other crucial preparations that Immunovia has made in response to COVID-19, such as securing inventories of critical consumables, the successful collection of samples for the verification study including the reallocation of extra blood samples to the efforts of securing all samples for the verification study. All this done to facilitate the uninterrupted continuation towards commercialization start.

We reiterate that Immunovia’s R&D laboratories and clinical laboratory in Lund, along with the production facilities are fully operational. They all have plans in place to help sustain operations in these unusual times."

– Excerpt from the CEO Mats Grahn’s comment on the report

First Quarter Interim Report for January – March 2020 Call Details:

Immunovia invites to a teleconference (in English) for investors, analysts and media on Tuesday, April 28th, 2020 at 16:30 CET.

Mats Grahn, CEO will present Immunovia and comment on the first quarter interim report for the period January – March 2020 followed by a Q&A session.

There will be an MP3-file available at Immunovia’s webpage under Investors/Financial Reports (View Source) for those who want to listen to the telephone conference afterwards. The file will be available within two hours after the conference has ended.

This information is information that Immunovia AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 16:00 CET on April 28, 2020.

On April 28, 2020 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported an update on its ZENITH20 Phase 2 clinical trial evaluating poziotinib in non-small cell lung cancer (NSCLC) patients with EGFR and HER2 exon 20 insertion mutations (Press release, Spectrum Pharmaceuticals, APR 28, 2020, View Source [SID1234556666]). The protocol has been amended to explore additional twice daily dosing regimens as well as lower single daily dosage amounts. Earlier use of corticosteroids has also been added to the protocol in an effort to help patients better tolerate poziotinib and stay on therapy for a longer time. Previously announced results for Cohort 1 of the SPECTRUM20 trial were presented yesterday during the oral plenary of the Lung Cancer Targeted Therapy session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting. Although the results for Cohort 1 did not meet the primary endpoint, as previously announced, poziotinib demonstrated a positive treatment effect with a 68.7% disease control rate.

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"Based on our analysis of the data, we believe that dose reductions and interruptions may have negatively impacted the treatment effect of poziotinib in this trial, and we are moving forward to further test this hypothesis with additional dose exploration in various cohorts. Pharmacologic modeling suggests that BID dosing can significantly lower Cmax, potentially reducing toxicity, and at the same time, maintain serum concentration above the IC50 value, therefore retaining anti-tumor activity," said Francois Lebel, M.D., Chief Medical Officer of Spectrum Pharmaceuticals. "Cohort 5 already included lower daily doses of poziotinib and we now have added twice daily regimens with a goal of providing a greater therapeutic window. Additionally, we are incorporating earlier use of corticosteroids for better control of the observed treatment related rash."

"Our data clearly shows that poziotinib produces a positive treatment effect and we are exploring changes in its dosing to allow patients to stay on drug longer and therefore derive the full potential benefits of treatment," stated Joe Turgeon, CEO of Spectrum Pharmaceuticals. "These seriously ill cancer patients urgently need alternative treatment options."

ZENITH20 Trial Design and Results from Cohort 1

Cohort 1 of the ZENITH20 trial enrolled 115 patients who received 16 mg/day of poziotinib. The intent-to-treat analysis showed that 17 patients had a response (by RECIST) and 62 patients had stable disease for a 68.7% disease control rate (DCR). The confirmed objective response rate (ORR) was 14.8% (95% Confidence Interval (CI) 8.9%-22.6%). Based on the FDA reviewed protocol, an observed ORR of 30%, with 17% as the lower bound for 95% CI was considered to be the clinically meaningful efficacy in our study. The median duration of response was 7.4 months with progression free survival of 4.2 months.

The safety profile was in-line with the type of adverse events seen with other second-generation EGFR tyrosine kinase inhibitors. Approximately 63% of patients experienced a grade 3 or 4 adverse event, the most common of which was rash and diarrhea. Tolerability may have led to reduced drug exposure as 68% of patients were dose reduced and 88% of patients had a temporary drug/dosing interruption. The median relative dose intensity was 72%. The high incidence of dose reductions and interruptions may indicate that the once daily starting dose of 16 mg was high and the resulting reduced dose intensity could have impaired the efficacy results. The company believes that a reduction in dose interruptions and daily dosing could lead to a greater number of deeper and more durable responses.

Cohort 5 is evaluating patients using a range of different doses compared to Cohort 1. Patients are randomized to 10, 12 or 16 mg administered once daily, and with the amended protocol, the trial will also explore 6 and 8 mg administered twice daily. Cohorts 4, 6 and 7 have also been amended to use 8 mg twice daily dosing.

The ZENITH20 trial is made up of seven independent cohorts. Cohorts 1 – 4 are each independently powered for a pre-specified statistical hypothesis with a primary endpoint of ORR. Cohorts 5 – 7 are exploratory studies. Cohort 3 is now fully enrolled and the company expects to report results for Cohort 2 in mid-2020 and Cohort 3 by the end of the year.

Conference Call and Webcast

Spectrum’s management will host a webcast and conference call today, April 28, 2020 at 8:30 a.m. ET / 5:30 a.m. PT to review the data and program strategy. The live call may be accessed by dialing (877) 837-3910 for domestic callers and (973) 796-5077 for international callers and entering the conference ID#: 5536065. A live webcast of the call will be available from the Investors and Media section of the company’s website at View Source and will be archived there shortly after the live event.

On April 28, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that five abstracts were accepted for the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place virtually from May 12-15, 2020. Abstracts are available on the ASGCT (Free ASGCT Whitepaper) website (Press release, Intellia Therapeutics, APR 28, 2020, View Source [SID1234556686]).

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Intellia’s data include important updates about the company’s progress and platform development activities:

Oral Presentations

"Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering"
As a follow-on to data presented at Keystone Symposia’s Engineering the Genome Conference this past February, Intellia’s ASGCT (Free ASGCT Whitepaper) presentation will focus on process improvements in its CRISPR/Cas9-based engineering to deliver T cell therapies with high levels of editing, achieving robust levels of expansion, desirable memory phenotypes, improved function and reduced translocations. These platform advances support NTLA-5001, which is Intellia’s first wholly owned engineered T cell therapy development candidate. NTLA-5001 utilizes a T cell receptor (TCR)-directed approach to target the Wilms’ Tumor 1 (WT1) antigen, obtained through the ongoing research collaboration with IRCCS Ospedale San Raffaele, which is over-expressed in many hematologic and solid tumors. Intellia will focus its initial clinical investigation of NLTA-5001 on acute myeloid leukemia (AML), while exploring in ongoing preclinical studies its application in solid tumors. Intellia plans to submit an IND for NTLA-5001 for the treatment of AML in the first half of 2021.

Presenter: Aaron Prodeus, Ph.D., senior scientist, Cell Therapy
Abstract number: 35
Session: CAR T-Cell Therapies I
Presentation date/time: Tuesday, May 12, 2020, from 11:45 a.m.-12 p.m. ET

"Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia"
This presentation will focus on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele to apply CRISPR/Cas9 technology to develop engineered T cell therapies to address intractable cancers, such as AML. Researchers will also present data showing a significant decrease in AML tumor burden in mice treated with the WT1-specific TCRs, which are incorporated into Intellia’s lead NTLA-5001 candidate for the treatment of AML.

Presenter: Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy
Abstract number: 78
Session: CAR T and Other Engineered T Cells Targeting Hematological Malignancies
Presentation date/time: Tuesday, May 12, 2020, from 3:45-4 p.m. ET

"CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema"
Intellia will present data updates on its potential hereditary angioedema (HAE) therapy, which uses the company’s modular lipid nanoparticle (LNP)-based CRISPR/Cas9 delivery system to knock out the prekallikrein B1 (KLKB1) gene and reduce kallikrein activity. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. This presentation will include results from the company’s ongoing collaboration with researchers at Regeneron and also will build on initial data released at Keystone Symposia’s Engineering the Genome Conference in February. Intellia expects to nominate a development candidate for HAE in the first half of 2020.

Presenter: Jessica Seitzer, director, Genomics
Abstract number: 1320
Session: Gene Regulation and Delivery Technologies
Presentation date/time: Friday, May 15, 2020, from 10:30-10:45 a.m. ET

Poster Presentations

"Validation of CRISPR/Cas9 Off-Target Discovery Profiles from In Silico Prediction, Cell-Based and Biochemical-Based Assays with Targeted Off-Target Sequencing"
This presentation will highlight Intellia’s approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Nishit Patel, Ph.D., senior associate scientist, Informatics, Computational Biology and Genomics
Abstract number: 203
Session date/time: Tuesday, May 12, 2020, from 5:30-6:30 p.m. ET

"In Vivo Model Development for Genome-Edited T Cell Therapeutics"
Researchers will present improved murine models used to assess safety, efficacy and persistence of therapeutic modalities in cell-based therapies. Together with biotechnology company Taconic Biosciences, Intellia developed two in vivo mouse models, which allow for enhanced monitoring of graft-versus-host disease and human natural killer cytotoxicity.

Presenter: Yong Zhang, Ph.D., principal scientist, Cell Therapy
Abstract number: 1174
Session date/time: Thursday, May 14, 2020, from 5:30-6:30 p.m. ET

Workshop and Symposia

"Building a Modular CRISPR/Cas9 Platform for Human Therapeutic Applications"
Intellia will participate in the ASGCT (Free ASGCT Whitepaper) Translational Science Genome Editing Pre-Meeting Workshop, which will include an overview on important factors to consider in building a modular CRISPR/Cas9 platform for human therapeutic applications.

Presenter: Jessica Seitzer, director, Genomics
Session: Gene Editing Workshop
Presentation date/time: Monday, May 11, 2020, from 4:10-4:30 p.m. ET

Industry Sponsored Symposia

Intellia will participate in an industry lunchtime panel discussion during ASGCT (Free ASGCT Whitepaper).

Panelist: Laura Sepp-Lorenzino, Ph.D., chief scientific officer
Panel date/time: Wednesday, May 13, 2020, from 12-1:30 p.m. ET