On May 8, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, reported its financial results for the first quarter ended March 31, 2020 (Press release, Synlogic, MAY 8, 2020, View Source [SID1234557347]).

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"In the first quarter, we continued to advance our programs and enhance our Synthetic Biotic platform capabilities in synthetic biology, manufacturing and development. While the coronavirus pandemic necessitated we update a number of our development plans, I am very grateful to our dedicated employees who have enabled us to continue to execute on our strategy under challenging circumstances," said Aoife Brennan, M.B., Ch.B., Synlogic’s president and chief executive officer. "We look forward to showcasing our deep platform capabilities and expanding program portfolio at our upcoming virtual R&D event on May 27th, which will include more details on one of our newest and rapidly advancing programs in enteric hyperoxaluria."

2020 Priorities

Pipeline

Initiation of a Phase 2 clinical trial to evaluate a solid formulation of SYNB1618 in patients with phenylketonuria (PKU). SYNB1618 is an orally administered Synthetic Biotic medicine that is being developed as a treatment for PKU.
Synlogic intends to continue to work with sites to complete preparatory work, however as a result of the coronavirus pandemic it does not expect to be able to enroll subjects into its Phase 2 clinical trial of SYNB1618 until it is safe for patients to enter clinical trial sites.
The Phase 2 trial is designed to evaluate safety and tolerability of a solid formulation of SYNB1618 as well as its potential to lower blood phenylalanine levels in PKU patients. In addition, the study is expected to provide valuable information to validate predictive pharmacodynamic and preclinical modeling.
Evaluation of data from the monotherapy arm of the Phase 1 clinical study of SYNB1891 in patients with advanced solid tumors or lymphoma. SYNB1891 is an intra-tumorally administered Synthetic Biotic medicine engineered to produce cyclic di-AMP, an agonist of the STING pathway, that is designed to serve as a dual innate activator of the immune system as a potential treatment for solid tumors or lymphoma. SYNB1891 is being evaluated as a monotherapy in an ongoing Phase 1 open-label, multicenter, dose escalation clinical trial (NCT04167137) in patients with advanced solid tumors or lymphoma.
While this clinical trial and most clinical sites have remained open and enrolled patients have continued on study, Synlogic expects slower enrollment of new patients as a result of the coronavirus pandemic, which has the potential to impact the availability of data in 2020.
Continued development of patient and commercialization-appropriate drug presentations of SYNB1618.
Synlogic has developed and manufactured a solid formulation of its Synthetic Biotic SYNB1618 that it plans to use in future clinical trials and continues to evaluate and develop different drug presentations (e.g. capsule, pressed pill, sachet) for eventual commercialization.
Advancement of new Synthetic Biotic programs in metabolic diseases with high unmet medical need.
Synlogic is conducting preclinical studies of Synthetic Biotic medicines to treat enteric hyperoxaluria, an acquired metabolic disorder in which patients develop recurrent kidney stones due to elevated urinary oxalate levels and are at an increased risk of kidney failure. Synlogic expects to move a clinical candidate in its enteric hyperoxaluria program, into IND-enabling studies in 2020.
In addition, Synlogic is also developing Synthetic Biotic medicines for the treatment of other metabolic diseases, including maple syrup urine disease (MSUD), a rare inherited metabolic disease caused by defective enzymes that metabolize branched chain amino acids (BCAAs) which are components of protein.
Publication and presentation of data demonstrating the breadth and potential of its Synthetic Biotic platform.
On March 8th, Nature Communications published a Perspective authored by Synlogic scientists entitled, "Developing a new class of engineered live bacterial therapeutics to treat human diseases." The paper, which highlights the considerations for the design and development of engineered live bacteria, including Synthetic Biotic medicines, is available on the Synlogic website on the Presentations and Publications page of the Investors and Media section.
On May 27th, 2020 Synlogic will host its first virtual R&D event
The webcast event will highlight the Company’s progress in developing its platform capabilities and pipeline of rare metabolic disease programs for internal prosecution and programs in immunomodulation for potential partnering. Dr. David Goldfarb, Professor of Medicine and Physiology at NYU School of Medicine, Clinical Chief of the Nephrology Division at NYU Langone Health, and Chief of the Nephrology Section and Director of the Hemodialysis Unit at the New York VA Medical Center in Manhattan will also present an overview of enteric hyperoxaluria. A link to the live webcast will be available on Synlogic’s website on the News & Events page of the Investors and Media section.
First Quarter 2020 Financial Results
As of March 31, 2020, Synlogic had cash, cash equivalents, and short-term investments of $114.2 million.

For the three months ended March 31, 2020, Synlogic reported a consolidated net loss of $15.8 million, or $0.46 per share, compared to a net loss of $12.9 million, or $0.51 per share, for the corresponding period in 2019.

Research and development expenses were $12.7 million for the three months ended March 31, 2020 compared to $10.4 million for the corresponding period in 2019. The increase in expenses was primarily due to use of synthetic biology services provided under Synlogic’s collaboration with Ginkgo and increased clinical activities associated with the SYNB1618 bridging study and the SYNB1891 Phase 1 clinical trial.

General and administrative expenses for the three months ended March 31, 2020 were $3.8 million compared to $3.7 million for the corresponding period in 2019.

Revenue was $0.1 million for the three months ended March 31, 2020 compared to $0.3 million for the three months ended March 31, 2019. Revenue is associated with services performed under the Synlogic’s collaboration with AbbVie to develop a Synthetic Biotic medicine for the treatment of IBD.

Conference Call & Webcast Information
Synlogic will host a conference call and live webcast today at 8:00 a.m. ET today, Friday, May 8, 2020. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Alternatively, investors may listen to the call by dialing +1 (844) 815-2882 from locations in the United States or +1 (213) 660-0926 from outside the United States. The conference ID number is 6869043. For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors and Media section of the Synlogic website.

On May 8, 2020 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel immunotherapy treatments for cancer and autoimmune disease, reported the grant of a new patent (number 6691054) entitled "Combined Preparations for the Treatment of Cancer" by the Japanese Patent Office (Press release, Immutep, MAY 8, 2020, View Source [SID1234557346]).

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This Japanese patent follows the grant of the corresponding European and Australian patents (announced 23 May 2019 and 21 June 2019, respectively) and protects Immutep’s intellectual property relating to combined therapeutic preparations comprising its lead active immunotherapy candidate eftilagimod alpha ("efti" or "IMP321") and a chemotherapy agent. The chemotherapy agent is oxaliplatin, carboplatin, or topotecan.

The new patent highlights the broad potential of efti as an immunostimulant and provides patent protection in Japan for a range of novel and highly relevant chemo-immunotherapies featuring efti that may be pursued in the future.

The patent expiry date is 19 December 2034.

On May 8, 2020 Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress"), an innovative biopharmaceutical company, reported that Oncogenuity, Inc. ("Oncogenuity"), a new Fortress partner company, has entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers (Press release, Fortress Biotech, MAY 8, 2020, View Source [SID1234557345]). The proprietary platform produces oligomers, now known as "ONCOlogues," that are capable of binding gene sequences 1,000 times more effectively than complementary native DNA. The technology comes from the labs of Gary Schwartz, M.D., Division Chief, Hematology/Oncology, and Jeffrey Rothman, M.D., Ph.D., Assistant Professor of Medicine.

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ONCOlogues are sensitive to a single base pair mismatch, resistant to degradation and use a proprietary delivery sequence to enter cells. ONCOlogues’ selectivity enables Oncogenuity to target genetically driven cancers caused by mutations without impacting wild-type ("WT") DNA sequences, potentially limiting off-target toxicity. In addition, this allows ONCOlogues to target mutations that have historically been considered "un-druggable."

Oncogenuity has established proof-of-concept in a pre-clinical setting for various cancer types. The company’s most advanced program is targeting the KRAS mutation G12D, which was previously considered un-druggable and plays a significant role in various cancer types with substantial unmet need, including pancreatic and colorectal. Given the platform’s ability to target any mutation, Oncogenuity will continue to evaluate other mutations simultaneously. The company anticipates additional data publications in the coming 12 months.

Additionally, Oncogenuity is exploring the platform’s potential to treat coronaviruses. Coronaviruses have single-stranded RNA genomes, making them strong targets for ONCOlogues. The company is studying replacement sequences, which could help combat COVID-19 and provide proof-of-concept as a treatment for coronaviruses. These ongoing experiments would validate ONCOlogues as a possible treatment for COVID-19, as well as potentially expedite the discovery of treatments for future coronavirus outbreaks.

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are excited to work with the excellent scientists and physicians at Columbia University again. Our last joint effort with Columbia University led to the formation of our partner company, Caelum Biosciences, Inc. ("Caelum"). Since formation, Caelum has raised approximately $60 million in development funding from a number of sources, with additional amounts available upon the satisfaction of certain milestones and will be initiating two registration clinical trials in the next several weeks. Building upon our success with Caelum, we are grateful to Columbia University for entrusting us to develop this highly innovative technology using oligonucleotides to target genetically driven cancers and coronaviruses. Using a targeted genetic approach to treat cancer has become essential to limiting toxicity and treating patients effectively. This technology has the potential to target mutations that have previously been considered un-druggable. Oncogenuity will aggressively pursue the development of ONCOlogues to ultimately provide patients with new, safe and effective treatment options."

Scientific Co-Founder Jeffrey Rothman, M.D., Ph.D., said, "Through rigorous statistical, mechanical and molecular modeling, combined with gene sequence data, we are able to create sequence-specific, targeted therapeutics against oncogenes, which are the cause of and specific to tumor cells. Until now, achieving this goal had been considered nearly impossible. However, with these novel design features, we now have the ability to target cancer while potentially avoiding side effects, which are the main cause of dose-limitation, by design. There is much potential because we are able to target multiple genes and therefore, multiple cancers. Moreover, due to their single-strand format, application toward viral targets such as in COVID are even more facile given their easier accessibility. We are excited and determined to pursue this endeavor with Fortress Biotech and very much welcome their continued support."

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.

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

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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.