On May 8, 2020 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Crinetics Pharmaceuticals, MAY 8, 2020, View Source [SID1234557376]).

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"So far, 2020 has proven to be a transformational time for Crinetics, highlighted by the positive interim Phase 2 results for paltusotine, our lead product candidate for the treatment of acromegaly," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "The interim data we reported showed that acromegaly patients switching from injectable depot therapy to once daily oral paltusotine maintained IGF-1 levels previously achieved with commercially available depot injections of somatostatin receptor ligands. We plan to report topline data from our ongoing Phase 2 trials in the fourth quarter of 2020 and advance paltusotine into Phase 3 development for patients with acromegaly in the first half of 2021 following conversations with regulatory agencies. Additionally, our successful follow-on offering in April strengthened our balance sheet and provided us with sufficient resources to advance our pipeline through key milestones, including our planned Phase 3 trial in acromegaly, a Phase 2 trial of paltusotine in carcinoid syndrome associated with neuroendocrine tumors (NETs), as well as Phase 1 trials to demonstrate proof-of-concept and PK/PD with our ACTH antagonist and SST5 agonist programs."

"We are both grateful and inspired by the dedication of staff and patients at our clinical trial sites around the world who have worked through the global COVID-19 pandemic to continue their participation in these important studies," said Alan Krasner, M.D., Chief Medical Officer or Crinetics. "Their commitment suggests a strong desire by the global acromegaly patient community and their healthcare providers to seek new therapies to manage their disease."

First Quarter and Subsequent Highlights

Reported positive interim results for the ACROBAT Edge Phase 2 trial of paltusotine in acromegaly patients. In April 2020, Crinetics reported interim results from its ongoing ACROBAT Edge Phase 2 trial. Results as of the February 23, 2020 data cutoff showed that acromegaly patients switching from injectable depot therapy to once daily oral paltusotine maintained IGF-1 levels previously achieved with commercially available depot injections of somatostatin receptor ligands. Interim results from an exploratory analysis of the first 13 patients who entered the Edge trial on octreotide or lanreotide depot monotherapy (group 1) showed that patient IGF-1 levels were maintained after switching to once daily oral paltusotine when compared to IGF-1 levels achieved with prior depot therapy [mean change from baseline = -0.015 x ULN (95% CI = -0.123, +0.092)]. Ten of the 11 (91%) patients in group 1 who completed paltusotine treatment maintained IGF-1 levels within 15% of their respective baseline levels at week 13. No patient required "rescue therapy" with prior injected peptide acromegaly therapy after switching to paltusotine. As a result of these data, new enrollment in the ACROBAT Evolve trial has been discontinued. The patients already enrolled in both the Edge and Evolve Phase 2 trials will continue in the studies and topline data is expected to be reported in the fourth quarter.

Provided a corporate update on the pipeline. In April 2020, Crinetics also provided an update on its other development programs, including an announcement that Phase 1 data for CRN01941 in healthy volunteers showed that the compound did not represent an improvement over paltusotine. Therefore, the company has discontinued development of CRN01941 in order to focus resources on paltusotine for both acromegaly and NETs. Crinetics believes that the acceleration and increased efficiency offered by focusing on paltusotine provides the best path forward for its SST2 franchise. Additionally, first-in-human enabling activities are ongoing for both the oral nonpeptide ACTH antagonist for the treatment of Cushing’s disease and congenital adrenal hyperplasia, and the oral nonpeptide SST5 agonist for the treatment of hyperinsulinism. The start of Phase 1 clinical trials for

these programs is planned for late 2020 or early 2021 and, if successful, the company anticipates PK/PD data from these human proof-of-concept studies in the first half of 2021.

Successful public offering strengthens cash position. In April 2020, Crinetics completed a public offering in which the company sold an aggregate of 8,222,500 shares of common stock at a price to the public of $14.00 per share. Net proceeds from the public offering after deducting underwriting discounts, commissions and offering expenses, were approximately $107.9 million.

COVID-19 Impact

During these challenging times, Crinetics remains committed to the health and safety of our employees, as well as the clinical sites and patients involved in its clinical trials. The company is closely monitoring public health guidance as it aims to continue to treat patients in its ongoing and planned clinical trials, which, so far, have remained on track. The overall impact of the COVID-19 pandemic on Crinetics’ business, its ability to obtain clinical material, and to conduct preclinical research and clinical trials in a timely manner is currently unknown. So far, the pandemic has not had a significant negative impact on the company’s ability to conduct business activities, but that could change, and the company is continuously monitoring the impact of the outbreak of the virus.

First Quarter 2020 Financial Results

Research and development expenses were $13.9 million for the three months ended March 31, 2020, compared to $7.3 million for the same period in 2019. The increases were primarily attributable to development and manufacturing activities for paltusotine as well as the advancement of the company’s preclinical programs and higher personnel costs.

General and administrative expenses were $4.0 million for the three months ended March 31, 2020, compared to $3.2 million for the same period in 2019. The increases were primarily due to personnel costs to support the company’s growth.
e months ended March 31, 2020 was $17.4 million, compared to a net loss of $9.0 million for the three months ended March 31, 2019.

Cash, cash equivalents and investments totaled $112.8 million as of March 31, 2020, compared with $118.4 million as of December 31, 2019. The cash balance at the end of March does not include the $107.9 million net proceeds from the public equity offering completed in April.

As of April 30, 2020, the company had 32,845,572 common shares outstanding.

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.