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bluebird bio to Present Updated Results From Pivotal Phase 2 KarMMa Study of Ide-cel in Relapsed and Refractory Multiple Myeloma During ASCO20 Virtual Scientific Program

On May 8, 2020 bluebird bio, Inc. (Nasdaq: BLUE) reported that updated results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of idecabtagene vicleucel (ide-cel; bb2121), an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy, in heavily pre-treated patients with relapsed and refractory multiple myeloma (RRMM) will be presented, in partnership with Bristol Myers Squibb, during the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 (ASCO20) Virtual Scientific Program beginning on May 29 (Press release, bluebird bio, MAY 8, 2020, View Source [SID1234557343]).

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Topline results from the KarMMa study were previously disclosed in December 2019. An additional presentation at ASCO (Free ASCO Whitepaper)20 will include results from a real-world, non-interventional, retrospective study evaluating treatment patterns in heavily pre-treated patients with RRMM compared to outcomes from the KarMMa study.

On Wednesday, May 13 at 5:00 PM ET, accepted abstracts will be available on the ASCO (Free ASCO Whitepaper) conference website. At that time, the embargo for the data included in the May 29 presentations will lift. The companies plan to issue a data press release at the time of the embargo lift. Video and slide presentations will be available on demand on the ASCO (Free ASCO Whitepaper) conference website beginning Friday, May 29 at 8:00 AM ET and will remain available for 180 days.

Oral Presentation:

Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in patients with relapsed and refractory multiple myeloma (RRMM): initial KarMMa results

Presenting Author: Nikhil C. Munshi, MD, The LeBow Institute for Myeloma Therapeutics and Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA
Session Title: Hematologic Malignancies- Plasma Cell Dyscrasia
Date & Time: Abstract #8503, Friday, May 29, 2020, 8:00 AM ET

Poster Presentation

KarMMa-RW: A study of real-world treatment patterns in heavily pretreated patients with relapsed and refractory multiple myeloma (RRMM) and comparison of outcomes to KarMMa

Presenting Author: Sundar Jagannath, MD, Mount Sinai Hospital, New York, NY, USA
Session Title: Hematologic Malignancies- Plasma Cell Dyscrasia
Date & Time: Abstract #8525, Friday, May 29, 2020, 8:00 AM ET

About idecabtagene vicleucel (ide-cel; bb2121)

On March 31, 2020, bluebird bio and Bristol Myers Squibb announced the submission of a Biologics License Application for ide-cel to the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. Ide-cel is the first chimeric antigen receptor (CAR) T cell therapy submitted for regulatory review to target B-cell maturation antigen (BCMA) and for multiple myeloma.

Ide-cel is a BCMA-directed genetically modified autologous CAR T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 α hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3-ζ chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

Bristol Myers Squibb and bluebird bio’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.

Ide-cel is not approved for any indication in any geography.

About KarMMa

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adult patients with relapsed and refractory multiple myeloma (RRMM) in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other efficacy endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

InDex Pharmaceuticals Holding AB (publ) interim report January – March 2020

On May 7, 2020 InDex Pharmaceuticals Holding AB (publ) interim report January – March 2020 (Press release, InDex Pharmaceuticals, MAY 7, 2020, View Source [SID1234562659])

FDA and EMA endorse the advancement of cobitolimod into phase III studies

"The positive responses from the regulators is an important external validation of our study results and cobitolimod’s potential," says Peter Zerhouni, CEO of InDex Pharmaceuticals.

Period January – March 2020

Net sales amounted to SEK 0.0 (0.0) million
Operating loss amounted to SEK –24.0 (–17.2) million
Result after tax amounted to SEK –24.0 (–17.2) million, corresponding to SEK –0.27 per share (–0.25) before and after dilution
Cash flow from operating activities amounted to SEK –21.9 (–18.4) million
Cash and cash equivalents at the end of the period amounted to SEK 104.6 (64.4) million
Number of employees at the end of the period was 7 (7)
Number of shares at the end of the period was 88,781,275
All comparative amounts in brackets refer to the outcome during the corresponding period 2019.

Significant events during January – March 2020

InDex in-depth analysis of the CONDUCT study confirmed the successful top line results and supports the strategy going forward
InDex published mechanism of action data for cobitolimod in scientific journal
InDex received grant from Vinnova for pre-clinical development of DIMS compounds in inflammation
Significant events after the reporting period

InDex received positive responses from FDA and EMA regarding phase III development of cobitolimod for the treatment of moderate to severe ulcerative colitis
The annual general meeting in InDex Pharmaceuticals Holding AB was held on April 20, 2020
CEO statement

Following the phase IIb study CONDUCT meeting its primary endpoint in August 2019, dialogues were initiated with FDA and EMA respectively, for the further development of cobitolimod. These processes have now been completed and both authorities endorse the advancement of cobitolimod into phase III studies in patients with moderate to severe left-sided ulcerative colitis. The positive responses from the regulators is an important external validation of our study results and cobitolimod’s potential.

This regulatory feedback gives flexibility for different designs of the phase III program, for example, to conduct studies sequentially and potentially to include a higher dose in addition to the highest dose regimen tested in the phase IIb study (2×250 mg). We continue to evaluate the most advantageous study design based on, among other things, development risk, commercial potential, time to market and cost.

The covid-19 pandemic affects the healthcare systems and the investor sentiment globally, which must be taken into account in our strategic planning. Before we can start phase III, the healthcare situation must have normalised, and the necessary funding be secured. In addition to assessing the conditions for InDex conducting the phase III program, we continue to engage with potential partners, presenting the now complete business development package.

An important component is the qualitative market research conducted by an independent market research company with senior gastroenterologists active in ulcerative colitis and payers, such as experts from pricing authorities and insurance companies, in France, the UK, Germany and the USA during the first quarter of 2020. Both target groups recognise the unmet need for new safe and effective treatments for moderate to severe ulcerative colitis.

Cobitolimod’s Target Product Profile, based on the phase IIb data, tested well as a novel treatment and the efficacy/safety ratio is considered unsurpassable. Gastroenterologists in the study are likely to prescribe cobitolimod to a significant proportion of their patients with moderate to severe ulcerative colitis in a future commercialization, and the study supports pricing in line with modern treatment options. All in all, the results from this market research support our assessment that the annual sales of cobitolimod at a successful commercialization could reach more than USD 1 billion.

Regarding the development of additional drug candidates from the DIMS platform, we have received a new grant of SEK 2 million from Vinnova to evaluate selected substances in inflammatory disease models outside the area of inflammatory bowel disease.

After an extended period with minimal sales of the diagnostic test DiBiCol, and due to upcoming changes in regulations, we have decided to discontinue that business.

On April 20, a covid-19 adapted annual general meeting was held. Instead of a CEO presentation at the annual general meeting, I gave a webcasted company update the same day on InDex’s phase III preparations. The webcast is available on InDex’s homepage.

During the first quarter of 2020 we have been able to complete several important steps on the path towards phase III and I look forward to the continued work in which the mutually dependent activities: decision on study design and securing funding, will be the highest priority in the coming months.

Anavex Life Sciences Reports Fiscal 2020 Second Quarter Financial Results And Provides Business Updates

On May 7, 2020 Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported financial results for its fiscal 2020 second quarter (Press release, Anavex Life Sciences, MAY 7, 2020, View Source [SID1234561603]).

"We are pleased to report that we are approaching the conclusion of the double-blind Phase 2 Parkinson’s disease dementia trial with ANAVEX2-73 (blarcamesine) and we expect to report top line results by mid-2020," said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. "Both the Alzheimer’s disease Phase 2b/3 trial and clinical Rett syndrome programs are also proceeding as planned. Our focus on Precision Medicine studies for neurological disorders remains persistent."

Program Updates:

Enrollment for the two randomized, double-blind, placebo-controlled Phase 2 ANAVEX2-73 (blarcamesine) U.S. Phase 2 Rett syndrome trial[1] and the AVATAR Rett syndrome trial[2] each have surpassed the 50% mark and continue to enroll.
Despite the outbreak of COVID-19, all clinical trial programs continue to proceed. While Rett syndrome protocols have always by default allowed at-home visits, Anavex implemented contingency plans as recommended by local regulatory authorities for the Phase 2b/3 ANAVEX2-73 (blarcamesine) Alzheimer’s disease (AD) study[3] and the Phase 2 study in Parkinson’s Disease Dementia (PDD) study[4] to ensure remote or virtual assessments for all active patients and all respective extension studies. Because ANAVEX2-73 (blarcamesine) is an oral formulation, study participants are able to receive shipments of their study medication in a controlled and compliant fashion, and direct-to-patient delivery is occurring in multiple countries.
In January 2020, Anavex announced achieving the enrollment target for the ANAVEX2-73 (blarcamesine) Phase 2 study in Parkinson’s disease dementia (PDD). Topline results from this randomized, double-blind, placebo-controlled study are expected by mid-2020.
In February 2020, Anavex announced a publication in the peer-reviewed Journal of Neuroimmunology titled "Sigma-1 Receptor Agonists as Potential Protective Therapies in Multiple Sclerosis" featuring preclinical data of ANAVEX2-73 (blarcamesine) relevant to multiple sclerosis.
In April 2020, Anavex announced a publication in the peer-reviewed journal Alzheimer’s & Dementia: Translational Research & Clinical Interventions,entitled, "A precision medicine framework using Artificial Intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer’s disease therapy: Analysis of the Blarcamesine (ANAVEX2-73) Phase 2a clinical study" highlights the relevance of phenotypic and genotypic precision medicine analyses of Whole Exome Sequencing (WES) and gene expression (RNAseq) data in drug development and in particular the potential to identify patients’ genetic variants and gene expression changes that may predict increased chances of success of Alzheimer’s disease treatments.
Financial Highlights:

Cash and cash equivalents of $26.6 million at March 31, 2020, compared to $22.2 million at fiscal year ended September 30, 2019.
Research and development expenses of $6.1 million for both the current quarter as well as the comparable quarter in 2019.
General and administrative expenses of $1.7 million for the quarter as compared to $2.1 million for the comparable quarter in 2019.
Net loss of $7.2 million, or $0.12 per share for the quarter, compared to net loss of $7.2 million, or $0.15 per share in the comparable quarter of 2019.
The financial information for the fiscal quarter ended March 31, 2020 should be read in conjunction with the Company’s consolidated interim financial statements, which will appear on EDGAR, www.sec.gov and will be available on the Anavex website at www.anavex.com.

Conference Call / Webcast Information

Anavex will host a conference call and webcast today at 4:30 p.m. ET.

The live webcast of the conference call can be accessed online at View Source

To join the conference call, live via telephone, interested parties within the U.S. should dial, toll-free, 1 (866) 939-3921 and international callers should dial 1 (678) 302-3550. Please use confirmation number 49673201, followed by the pound sign (#).

A replay of the conference call will also be available on www.anavex.com.

Ayala Pharmaceuticals Announces Pricing of Initial Public Offering

On May 7, 2020 Ayala Pharmaceuticals, Inc. (NASDAQ: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, reported the pricing of its initial public offering of 3,666,667 shares of common stock at a public offering price of $15.00 per share, before underwriting discounts and commissions (Press release, Ayala Pharmaceuticals, MAY 7, 2020, View Source [SID1234558078]). All of the common stock is being offered by Ayala. The offering is expected to close on May 12, 2020, subject to customary closing conditions. In addition, Ayala has granted the underwriters a 30-day option to purchase up to an additional 550,000 shares of its common stock at the public offering price, less underwriting discounts and commissions. The gross proceeds of the offering are expected to be $55.0 million, excluding any exercise of the underwriters’ option.

Ayala’s common stock is expected to begin trading on the NASDAQ Global Market under the ticker symbol "AYLA" on May 8, 2020.

Citigroup and Jefferies are acting as the lead book-running managers for the offering. Oppenheimer & Co. and Raymond James are acting as the co-managers for the offering.

Merrimack Reports First Quarter 2020 Financial Results

On May 7, 2020 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) [("Merrimack" or the "Company")] reported its first quarter 2020 financial results for the period ended March 31, 2020 (Press release, Merrimack, MAY 7, 2020, View Source [SID1234557415]).

"We are pleased to report that we have significantly reduced our operating expenses and also sold certain of our remaining intellectual property assets during the first quarter of 2020," said Gary Crocker, Chairman of Merrimack’s Board of Directors. "As a result of reduced general and administrative costs and the proceeds from the previously announced asset sale we reported a profit of $249,000 for the first quarter. We are currently focused on making further reductions to legal, accounting and management consulting expenses which we plan to realize during the second half of 2020."

First Quarter 2020 Financial Results

Merrimack reported net income of $0.25 million for the first quarter ended March 31, 2020, or $0.02 per basic and diluted share on a fully diluted basis, compared to a net loss of $10.5 million, or $0.78 per basic and diluted share on a fully diluted basis, for the same period in 2019.

Merrimack reported a gain on the sale of assets for the first quarter ended March 31, 2020 of $2.1 million, attributable to the sale of certain of our preclinical nanoliposome programs.

No research and development expenses were recognized for the first quarter ended March 31, 2020 compared to $6.4 million for the same period in 2019.

General and administrative expenses for the first quarter ended March 31, 2020 were $1.9 million, compared to $3.7 million for the same period in 2019.

As of March 31, 2020, Merrimack had cash and cash equivalents and investments of $18.0 million, compared to $16.6 million as of December 31, 2019.

As of March 31, 2020, Merrimack had 13.4 million shares of common stock outstanding.