Telix Pharmaceuticals Limited Acquires TheraPharm GmbH, Broadening Reach to Hematologic Cancers and Transplant Medicine

MELBOURNE, Australia and BAAR, Switzerland, Nov. 29, 2020 (GLOBE NEWSWIRE) — Telix Pharmaceuticals Limited (ASX: TLX, ‘Telix’, the ‘Company’) announces it has entered into an agreement with Scintec Diagnostics GmbH (‘Scintec’) to acquire TheraPharm GmbH (‘TheraPharm’), a Swiss-German biotechnology company developing innovative diagnostic and therapeutic solutions in the field of hematology.

The acquisition of TheraPharm provides Telix with access to a portfolio of patents, technologies, production systems, clinical data and know-how in relation to the use of Molecularly Targeted Radiation (MTR) in hematology and immunology. TheraPharm is developing antibody MTR technology against CD66, a cell surface target highly expressed by neutrophils (a type of white blood cell) and tumor-infiltrating lymphocytes. As such, the technology has potentially very broad applications in the diagnosis and treatment of hematologic diseases (e.g. blood cancers), lymphoproliferative disorders and immune-mediated diseases (e.g. lupus, and multiple sclerosis). Of particular interest is the demonstrated use of the technology to safely and effectively perform bone marrow conditioning (BMC) prior to bone marrow stem cell transplant.

Telix CEO, Dr. Christian Behrenbruch stated, “Telix is committed to extending and improving the lives of patients with serious diseases. As such, the acquisition of TheraPharm and its MTR assets are uniquely aligned to Telix’s mission and technical strengths in antibody engineering and radiochemistry. TheraPharm’s technology has a significant role to play in BMC and stem cell transplantation across a broad range of blood cancers and rare diseases. The current approach to BMC employs highly toxic drugs that have a poor morbidity and mortality profile, and for which many patients are ineligible. MTR offers an excellent safety profile that may greatly expand the number of patients able to undergo life prolonging stem cell transplantation while greatly reducing the hospitalisation burden and cost associated with such procedures.”

TheraPharm co-founder and Managing Director, Dr. Klaus Bosslet added, “Over the past 5 years, TheraPharm, in collaboration with Dr. Kim Orchard from the University of Southampton (UK), has made excellent progress developing 90Y-besilesomab for the treatment of hematologic cancers and several related conditions including multiple myeloma, leukemia and amyloidosis. This unique asset is a logical addition to Telix’s portfolio, offering a potentially rapid development path to a first commercial indication for the treatment of patients with SALA, while at the same time having potentially broad applications for stem cell transplantation in patients with more common cancers of the blood, including multiple myeloma and leukemia. We look forward to joining the Telix team in order to expedite the development of products for this under-served field.”

Full transaction details, including financial terms, can be found via the Telix website and ASX portal here.

About Hematopoietic Stem Cell Transplant (HSCT)

Bone marrow conditioning (BMC) followed by hematopoietic stem cell transplantation (HSCT) is presently performed to treat patients with hematologic malignancies (blood cancers), with the objective of extending patient survival or achieving cure. HSCT is also performed for a broad range of non-cancer conditions. HSCT is preferentially performed in countries of high income (Europe >30,000, Americas >20,000, worldwide >65,000 p.a., respectively)

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TFF Pharmaceuticals, Inc. and Augmenta Bioworks, Inc. Enter Into a Worldwide Joint Development Agreement for COVID-19 Monoclonal Antibody Therapies

Companies to collaborate in first-of-its-kind uses of Thin Film Freezing technology applied to monoclonal antibodies

TFF Pharmaceuticals, Inc. (NASDAQ: TFFP), a clinical-stage biopharmaceutical company, and Augmenta Bioworks, Inc., a biotechnology company enabling breakthroughs in medicine through immune profiling, today jointly announce that both companies have entered into a worldwide Joint Development and Collaboration Agreement to develop novel commercial products incorporating Augmenta’s human-derived monoclonal antibodies (mAbs) for potential COVID-19 therapeutics.

Under the terms of the Agreement, both companies will collaborate in a Joint Development Project to develop one or more commercial therapeutics based on, derived from, and/or incorporating Augmenta’s human monoclonal antibodies to potentially treat patients with COVID-19. These products will be developed utilizing TFF Pharmaceuticals’ Thin-Film Freezing technology to manufacture dry powder formulations of these specific mAbs for inhalation delivery directly to the lungs of patients. The Agreement also includes the development of formulations suitable for parenteral administration, where the Thin Film Freezing dry powder formulations can be reconstituted, potentially mitigating the impacts of cold-chain storage and handling. TFF Pharmaceuticals will also have the option to develop two additional Augmenta mAbs for indications other than COVID-19.

Augmenta Bioworks and TFF Pharmaceuticals will allocate patent license rights to their respective technologies to allow each company to jointly commercialize the products developed under the Joint Development Project. The companies have agreed to a 50-50 split of all costs and expenses to further the Joint Development Project and both companies have agreed to the same 50-50 split of all revenues, cash payments and/or future cash payments related to the sale and/or license of the products resulting from the Joint Development Project to a third party.

“This important agreement represents the culmination of many months of work by our scientific team, as we work towards the development of a never-before-achieved formulation of monoclonal antibodies into a dry powder therapeutic,” said Glenn Mattes, CEO, of TFF Pharmaceuticals, Inc. “It is a testament to the remarkable flexibility and capability of our Thin Film Freezing platform and we are eager to develop these potentially breakthrough mAb therapies internally, along with our other programs in Invasive Pulmonary Aspergillosis, solid organ transplant anti-rejection, and botanicals.”

“Confirmed discovery of novel anti-SARS-Cov-2 antibodies in 8 days was an achievement made possible by years of technology development, and a clear indication of the power and potential of our platform,” said Christopher Emig, Ph.D., CEO and Co-Founder of Augmenta Bioworks, Inc. “We are excited to enter this partnership to bring our COVID-19 treatment into clinical development, and are looking forward to the world’s first effective, affordable and scalable antibody therapeutic to mitigate the devastating effects of this disease.”

“We believe the interest in monoclonal-antibody therapeutics for the treatment of COVID-19 is extremely high, with the promise that they will harness the immune system’s natural response to viral invaders,” said Robert O. Williams III, Ph.D., Division Head of the University of Texas at Austin’s Division of Molecular Pharmaceutics and Drug Delivery and inventor of TFF Pharmaceuticals’ Thin Film Freezing technology.

“The challenge

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Telix Pharmaceuticals and China Grand Pharma Announce Strategic Licence and Commercial Partnership for Greater China Market

MELBOURNE, Australia and HONG KONG, Nov. 02, 2020 (GLOBE NEWSWIRE) — Telix Pharmaceuticals Limited (ASX: TLX, ‘Telix’, the ‘Company’) announces it has entered into a strategic licence and commercial partnership with China Grand Pharmaceutical and Healthcare Holdings Limited (‘China Grand Pharma’) for Telix’s portfolio of Molecularly-Targeted Radiation (‘MTR’) products.

Telix has appointed China Grand Pharma as its exclusive partner for the Greater China market (‘Territory’)1 and grants China Grand Pharma exclusive development and commercialisation rights to Telix’s portfolio of prostate, renal and brain (glioblastoma) cancer imaging and therapeutic MTR products in the Territory.

Leveraging off China Grand Pharma’s capabilities and infrastructure in China, Telix will enter a significant oncology market, and by partnering with Telix, China Grand Pharma will build on its pipeline of innovative products for Greater China, as well as its strategy in Nuclear Medicine.

The material terms of the partnership include:

Therapeutic Products

  • US$25M (~AU$35M) up-front non-refundable prepayment to Telix, to be credited against future regulatory and commercial milestone payments.

  • Up to US$225M (~AU$315M) in regulatory and commercial milestone payments to Telix, across Telix’s existing therapeutic products portfolio.

  • Program-related investment estimated at up to US$65M (~AU$90M) for clinical costs associated with the development of the therapeutic products in the Territory, to align with Telix’s global clinical development programs.

  • Royalties on therapeutic product sales in the Territory, in addition to milestone payments.

Imaging Products

  • Exclusive commercial partnership (sales, marketing, distribution) for Telix’s core imaging product portfolio:

    • TLX250-CDx (89Zr-Girentuximab) for renal cancer, and;

    • TLX591-CDx (68Ga-PSMA), TLX599-CDx (99Tc-PSMA) for prostate cancer.

Strategic Equity Investment

Additionally, China Grand Pharma will make a simultaneous one-time strategic equity investment of US$25M (~AU$35M) in Telix. The investment is in the form of a private placement to China Grand Pharma of 20,947,181 fully paid ordinary Telix shares representing a post-issue holding by China Grand Pharma of 7.62%. Shares will be issued at a price of AU$1.69, based on the 10-day volume-weighted average price (‘VWAP’) for Telix shares up to and including 28th October 2020. Shares will be issued no later than November 06 2020, following receipt of the placement proceeds. Shares issued to China Grand Pharma are subject to a holding lock and will not be able to be traded for a period of 12 months from the date of issue. In addition, China Grand Pharma is subject to a standstill provision and is unable to trade in Telix shares for a period of 12 months.

Telix Pharmaceuticals CEO, Dr. Chris Behrenbruch stated, “Telix’s mission is to be a leading global oncology company and China is an important future market for our products. We are pleased to be working with China Grand Pharma to deliver our diagnostic imaging and therapeutic products to cancer patients in China. Considering the successful acquisition of Sirtex Medical Limited with joint venture private equity partner CDH Genetech Limited2 and subsequent approval of a New Drug Application filing for SIR-Spheres® by the National Medical Products Administration (‘NMPA’) of the

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Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trials of Setmelanotide in The Lancet Diabetes & Endocrinology

Largest studies in POMC and LEPR deficiency obesities demonstrate that treatment with setmelanotide reduced body weight and hunger

BOSTON, Oct. 30, 2020 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from two pivotal Phase 3 studies evaluating setmelanotide in proopiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity were published in The Lancet Diabetes & Endocrinology. As previously reported, data from the studies demonstrate that treatment with setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, led to statistically significant and clinically meaningful reductions of weight and hunger.

“Results from Rhythm’s pivotal Phase 3 studies, which are the largest studies to date in POMC and LEPR deficiency obesities, provide evidence regarding the safety and efficacy of setmelanotide and we believe they validate its potential long-term use as a novel treatment for severe obesity and hyperphagia,” said co-author Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin, Germany. “It is important to recognize the signs of these rare genetic disorders because we may soon have a targeted treatment option available for the first time for obesity disorders caused by impairments of the MC4R pathway.”

Rhythm initially reported positive topline data from the Phase 3 studies in August 2019 and subsequently presented updated data in a late-breaking research forum during the 37th Annual Meeting of The Obesity Society at ObesityWeek® 2019.

Eight of 10 participants with POMC deficiency obesity (80%; P<0.0001 compared with historical data) and five of 11 participants with LEPR deficiency obesity (45%; P=0.0001 compared with historical data) achieved at least 10 percent weight loss at approximately one year. The mean percent change in “most hunger” score in participants aged 12 years and older was -27.1 percent (n=7; P=0.0005) in POMC deficiency obesity and -43.7 percent (n=7; P<0.0001) in LEPR deficiency obesity. Consistent with prior clinical experience, setmelanotide was generally well-tolerated in both trials. The most common adverse events were injection site reaction, skin hyperpigmentation, and nausea.

“These results are significant because, as we know from natural history data, individuals living with POMC or LEPR deficiency obesity consistently experience substantial weight gain each year beginning in early childhood, and we would not expect any of these patients to be able to achieve 10 percent weight loss over the course of a year without continued treatment,” said co-author Karine Clément, professor of nutrition at Pitié-Salpêtrière hospital and Sorbonne University in Paris. “These data and the significant unmet need to address the obesity and hyperphagia caused by rare genetic disorders of obesity underscore the importance of testing for genetic variants that may impair MC4R activation and lead to severe obesity.”

In May 2020, Rhythm announced that the U.S. Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, granted Priority Review of the NDA and assigned a Prescription Drug

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Nexus Pharmaceuticals Receives FDA Approval for Succinylcholine Chloride Injection, USP

Nexus Pharmaceuticals announced it has received U.S. Food and Drug Administration (FDA) approval for Succinylcholine Chloride Injection, USP in 200mg/10mL Multiple-Dose Vials. It is an AP-rated generic to QUELICIN™¥.

“Nexus Pharmaceuticals is very pleased to announce the approval of Succinylcholine Chloride. This critical need drug has seen an increased demand due to the COVID-19 pandemic and Nexus will be ready to meet the challenge. This approval further cements Nexus’ commitment to alleviating the drug shortage crisis with sterile injectable products produced exclusively in the US and Europe,” said Mariam S. Darsot, CEO & President of Nexus Pharmaceuticals.

Nexus Pharmaceuticals’ Succinylcholine Chloride Injection will be available in cartons of 25 vials.

About Succinylcholine Chloride Injection, USP

Succinylcholine Chloride Injection, USP is a sterile, nonpyrogenic solution to be used as an ultra-short-acting, depolarizing, skeletal muscle relaxant. Succinylcholine is indicated as an adjunct to general anesthesia, to facilitate tracheal intubation, and to provide skeletal muscle relaxation during surgery or mechanical ventilation.

For prescribing information, please see the following link.

About Nexus Pharmaceuticals Inc.

Nexus Pharmaceuticals Inc., a US-based healthcare company, specializes in innovative processes to make difficult-to-manufacture specialty and generic drugs that are easier to use, less labor intensive, and more streamlined in practice. Nexus ensures that its high-quality FDA-approved drugs fulfill a critical unmet medical need and delivers dependable life-saving treatment options when and where they’re needed most.

¥QUELICIN is a registered trademark of Hospira, Inc., a wholly-owned subsidiary of Pfizer Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201029006301/en/

Contacts

Diana Lebiecki
847-996-3790
[email protected]

Source Article

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RespireRx Pharmaceuticals Inc. Announces Appointment of Dr. James Cook and Dr. Jeffrey Witkin as Research Fellows

Dr. James Cook

“As the original designer of the GABAkines licensed by RespireRx, I am very excited to be working on the research team being assembled by RespireRx to realize their therapeutic potential,” said Dr. Cook
“As the original designer of the GABAkines licensed by RespireRx, I am very excited to be working on the research team being assembled by RespireRx to realize their therapeutic potential,” said Dr. Cook
“As the original designer of the GABAkines licensed by RespireRx, I am very excited to be working on the research team being assembled by RespireRx to realize their therapeutic potential,” said Dr. Cook

Dr. Jeffrey Witkin

“It is an honor to be afforded this singular opportunity to join such a capable and driven team in further progressing the expanding portfolio of highly promising neuromodulator compounds. The broad-ranging efficacy of our GABAkine candidate, KRM-II-81, in preclinical models and in human epileptic tissue, already provides compelling evidence to substantiate this compound as a next-generation antiepileptic drug. I am enthusiastically looking forward to becoming an integral part of RespireRx’ bright future,” said Dr. Witkin.
“It is an honor to be afforded this singular opportunity to join such a capable and driven team in further progressing the expanding portfolio of highly promising neuromodulator compounds. The broad-ranging efficacy of our GABAkine candidate, KRM-II-81, in preclinical models and in human epileptic tissue, already provides compelling evidence to substantiate this compound as a next-generation antiepileptic drug. I am enthusiastically looking forward to becoming an integral part of RespireRx’ bright future,” said Dr. Witkin.
“It is an honor to be afforded this singular opportunity to join such a capable and driven team in further progressing the expanding portfolio of highly promising neuromodulator compounds. The broad-ranging efficacy of our GABAkine candidate, KRM-II-81, in preclinical models and in human epileptic tissue, already provides compelling evidence to substantiate this compound as a next-generation antiepileptic drug. I am enthusiastically looking forward to becoming an integral part of RespireRx’ bright future,” said Dr. Witkin.

Glen Rock, N.J., Oct. 27, 2020 (GLOBE NEWSWIRE) — RespireRx Pharmaceuticals Inc. (OTCQB: RSPI) (“RespireRx” or the “Company”), a leader in the discovery and development of innovative and revolutionary treatments to combat diseases caused by disruption of neuronal signaling, is pleased to announce the appointment on October 16, 2020, of Dr. James Cook and Dr. Jeffrey Witkin as Research Fellows.

Tim Jones, President and Chief Executive Officer said, ‘‘We are thrilled to formally welcome Dr. Cook and Dr. Witkin to the RespireRx team. In their capacities as Research Fellows, they will immediately assume roles as integral core members of our research team, reporting to Dr. Arnold Lippa, and participate as members of our Scientific Advisory Board. Their intrinsic depth of knowledge, collective academic and industry achievements in the field of neuroscience is unrivaled. Their expertise in the development of novel and innovative glutamate and GABA-A receptor neuromodulators, across a broad scope of patient -critical disorders will prove extremely valuable as we continue to grow the business; in parallel, we are actively expanding our portfolio of products, broadening our technical and regulatory asset base and in turn strengthening our already longstanding collaboration with the University of Wisconsin-Milwaukee Research Foundation ‘UWMRF’.’

Dr. James Cook is a Distinguished Professor of Chemistry at the University Wisconsin-Milwaukee where he co-leads a group of scientists who have synthesized and tested a broad series of novel drugs that display GABA-A receptor subtype selectivity and pharmacological specificity. He is a leading expert in GABA-A receptor drug targeting with more than 40 years’ experience in organic and medicinal chemistry and more than 500 scientific publications and 60 patents.

Dr. Jeffrey Witkin, is

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Aridis Pharmaceuticals to Present at the ROTH Capital Partners 2020 MedTech Innovation Forum on a COVID-19 Panel

SAN JOSE, Calif., Oct. 27, 2020 /PRNewswire/ — Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, today announced the Company will present at the ROTH Capital Partners 2020 MedTech Innovation Forum on Wednesday, October 28, 2020. Dr. Hasan Jafri, Chief Medical Officer of Aridis Pharmaceuticals, will be a speaker on a panel entitled “Direct Antivirals and Other Agents Against SARS-CoV2 Virus.”

Panel: Direct Antivirals and Other Agents Against SARS-CoV2 Virus
Date: Wednesday, October 28, 2020
Time: 10:30AM-11:50AM ET

Dr. Jafri will present a summary of the Company’s recently published preclinical data of its COVID-19 inhaled mAb (AR-711). He will address the preclinical performance of AR-711, the advantages of direct lung delivery using nebulized aerosols, and the COVID-19 clinical program.

About AR-711

AR-711 is a fully human immunoglobulin 1, or IgG1, monoclonal antibody discovered from screening the antibody secreting B-cells of convalescent COVID-19 patients. AR-711 exhibits high affinity for SARS-CoV-2 spike protein, approximately 10-fold or higher than mAb candidates currently in late stage clinical testing. AR-711 was previously shown to be effective in prophylactic as well as therapeutic treatment modes in a SARS-CoV-2 viral challenge study. AR-711 is currently being developed as an inhaled, self-administered treatment for non-hospitalized patients suffering from mild to moderate COVID-19. AR-711 is also one the two mAbs in the company’s AR-701 mAb cocktail, which is a separate program being developed as an intravenous treatment of moderate to severe, hospitalized COVID-19 patients.

About Aridis Pharmaceuticals, Inc.

Aridis Pharmaceuticals, Inc. discovers and develops anti-infectives to be used as add-on treatments to standard-of-care antibiotics. The Company is utilizing its proprietary ʎPEXTM and MabIgX® technology platforms to rapidly identify rare, potent antibody-producing B-cells from patients who have successfully overcome an infection, and to rapidly manufacture monoclonal antibody (mAbs) for therapeutic treatment of critical infections. These mAbs are already of human origin and functionally optimized for high potency by the donor’s immune system; hence, they technically do not require genetic engineering or further optimization to achieve full functionality.

The Company has generated multiple clinical stage mAbs targeting bacteria that cause life-threatening infections such as ventilator associated pneumonia (VAP) and hospital acquired pneumonia (HAP), in addition to preclinical stage antiviral mAbs. The use of mAbs as anti-infective treatments represents an innovative therapeutic approach that harnesses the human immune system to fight infections and is designed to overcome the deficiencies associated with the current standard of care which is broad spectrum antibiotics. Such deficiencies include, but are not limited to, increasing drug resistance, short duration of efficacy, disruption of the normal flora of the human microbiome and lack of differentiation among current treatments. The mAb portfolio is complemented by a non-antibiotic novel mechanism small molecule anti-infective candidate being developed to treat lung infections in cystic fibrosis patients. The Company’s pipeline is highlighted below:

Aridis’ Pipeline

AR-301 (VAP). AR-301 is a fully human IgG1 mAb currently in Phase 3 clinical development targeting gram-positive 

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Crinetics Pharmaceuticals Announces Phase 2 ACROBAT Edge Study with Paltusotine in Acromegaly Met Primary Endpoint

ACROBAT Edge Results Showed Maintenance of IGF-1 Suppression with Paltusotine After Switching from Depot Somatostatin Receptor Ligand Monotherapy  

Paltusotine was observed to be well tolerated among the 60 participants in the ACROBAT Edge and Evolve Studies

On Track to Initiate Phase 3 Paltusotine Program in 1H 2021

SAN DIEGO, Oct. 26, 2020 (GLOBE NEWSWIRE) —  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, today announced positive topline results from the company’s Phase 2 ACROBAT Edge and ACROBAT Evolve studies of paltusotine (formerly CRN00808), the company’s lead candidate for the treatment of acromegaly. The Company will hold a conference call at 8:00 a.m. Eastern Time today to discuss these results. In addition, a Key Opinion Leader (KOL) call will be held on November 20th to discuss these results in the context of the current standard of care with clinical experts.

The prespecified primary endpoint in Edge was achieved, showing that once daily oral paltusotine maintained insulin-like growth factor-1  (IGF-1) levels at Week 13 in acromegaly patients who were switched from an injected somatostatin receptor ligand  (SRL) depot of either octreotide or lanreotide monotherapy  [change in IGF-1 = -0.034  (-0.107, 0.107), median (IQR)]. There were 25 patients enrolled in this prespecified primary analysis population (Group 1). During the four-week washout period after the 13-week treatment period, Group 1 patients showed a meaningful  (>20%) and prompt  (within two weeks) rise in IGF-1 levels from baseline, which characterized the magnitude of therapeutic activity of oral paltusotine in acromegaly patients. Edge also enrolled an additional 22 patients into four different exploratory populations  (Groups 2-5). 

Paltusotine was generally well tolerated among the 60 ACROBAT participants (including both Edge and Evolve), which is consistent with prior clinical findings in healthy volunteers. There were no discontinuations due to drug-related adverse events, no safety signals seen in clinical laboratory analyses, no treatment-related serious adverse events (SAEs), and no patients required rescue treatments with standard acromegaly medications during treatment. The most common treatment-emergent adverse events (>10%) included: headache, arthralgia, fatigue, peripheral swelling, paresthesia and hyperhidrosis.

“These results from Edge and Evolve support the potential to effectively switch acromegaly patients from their current depot injections to a once-daily oral treatment while maintaining hormonal control,” stated Alan Krasner M.D., Chief Medical Officer of Crinetics. “The heterogeneous nature of the ACROBAT patient population is representative of the real-world population, where acromegaly patients are prescribed a variety of treatments in an effort to control their IGF-1 levels, often unsuccessfully. The potential for once daily oral paltusotine to offer similar disease control provided by first-line injected depot SRLs gives us confidence to move into a Phase 3 program to further evaluate the efficacy and safety of paltusotine.”

Scott Struthers, Ph.D., founder and Chief Executive Officer of Crinetics, added, “Overall, we believe these findings support our thesis that once daily oral paltusotine has the potential to replace injected peptide depots for acromegaly therapy. We

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Corbus Pharmaceuticals Announces Presentation of Data from its Phase 2b Study in Cystic Fibrosis at NACFC 2020

  • As previously reported, lenabasum did not meet its primary efficacy endpoint in the study

  • Lenabasum was well tolerated with no new safety findings

  • Exploratory post-hoc analyses revealed unexpectedly low pulmonary exacerbation (PEx) rates in subjects from five eastern European countries (21% of total subjects) who received placebo; PEx rates in these subjects were 85% lower than in subjects from other countries

  • Post-hoc analyses in subjects with similar FEV1% predicted at baseline and similar treatment with CFTR-modulators suggested evidence of clinical benefit of lenabasum

  • Company to continue discussions and data analyses with CF experts

Norwood, MA, Oct. 23, 2020 (GLOBE NEWSWIRE) — Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) (“Corbus” or the “Company”), a clinical-stage drug development company pioneering transformative medicines that target the endocannabinoid system, today announced the presentation of the preliminary data from its 28 week Phase 2b study of lenabasum in patients with cystic fibrosis (CF) at the North American Cystic Fibrosis Conference (NACFC) held online, Oct 7-23, 2020. The data are being presented in poster number 817 titled: “CB2 Agonist, Lenabasum, for the Treatment of Pulmonary Exacerbations in Cystic Fibrosis.” These data can be found at the following link: NACFC Poster #817

James Chmiel, M.D., MPH, Principal Investigator of CF-002 and Division Chief for Pediatric Pulmonology, Allergy and Sleep Medicine and Professor of Pediatrics at Indiana University School of Medicine said, “This was the first interventional study in CF to select for patients who have high rates of pulmonary exacerbations and one of the largest CF studies to date. The negative impact of recurrent pulmonary exacerbations on the health and quality of life of people with CF cannot be overstated, nor can the need to find non-immunosuppressive treatments to control the lung inflammation that causes these events.” Dr. Chmiel commented further, “While I am disappointed that the study did not achieve its primary endpoint, I am encouraged by findings of potential reduction in exacerbation rates in subjects with similar lung function and treatment with CFTR-modulators. I look forward to further analyses of the data, because they may confirm the initial clinical rationale to continue development of lenabasum for treatment of pulmonary exacerbations in people with CF.”

Summary of findings:

  • Disposition, baseline demographics, and baseline disease characteristics and treatments were as expected (Tables 1 and 2 as presented in the poster). Enrolled subjects had significantly impaired mean baseline FEV1 and between 2-7 PEx in the prior year, despite background treatments reported to reduce PEx rates.

  • Lenabasum did not meet its primary efficacy endpoint of reducing the rate of PEx. PEx rates per subject per 28 weeks were as follows: 0.85, 0.75 and 0.90 for placebo, 5 mg, and 20 mg doses, respectively (Table 3).

  • Mean baseline FEV1 % predicted was similar across treatment groups, approximately 60% predicted. No significant differences among treatment groups were seen in change in FEV1% predicted (Table 4).

  • Lenabasum was safely administered and well tolerated in this study, with no new safety findings. Dizziness, fatigue, and dry mouth were among adverse events that

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Terns Pharmaceuticals to Present Clinical Data on Improved TERN-101 Tablet Formulation at the Paris NASH Digital Experience

Terns Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on developing best-in-class single-agent and combination therapies to treat non-alcoholic steatohepatitis (NASH) and other liver diseases, announced today the presentation of new clinical data on an improved tablet formulation of its liver-distributed farnesoid X receptor (FXR) agonist TERN-101 at the Paris NASH Digital Experience, taking place virtually October 22-23, 2020. The presentation, entitled “Comparative pharmacokinetics of two oral formulations of nonsteroidal farnesoid X receptor agonist TERN-101 in healthy volunteers,” demonstrated that an amorphous tablet formulation of TERN-101, which is currently being evaluated in the Phase 2a LIFT study in NASH patients, achieved faster absorption and higher plasma levels compared to a previous crystalline capsule formulation of TERN-101. The study results also demonstrate that the new formulation reduces pharmacokinetic variability and that TERN-101 can be administered without regard to food.

“In Terns’ Phase 1 studies, TERN-101 has demonstrated potent FXR activation in the liver and is well tolerated, with no observation of pruritus and no difference from placebo in serum lipid effects. We’ve now developed an improved formulation that optimizes absorption and minimizes PK variability,” said Erin Quirk, M.D., President and Chief Medical Officer of Terns. “This new TERN-101 formulation is now being studied in our 12-week Phase 2a LIFT study in NASH patients, and we look forward to seeing the results in mid-2021.”

Full details from the presentation can be found at www.ternspharma.com/scientific-publications.

About TERN-101 and Farnesoid X Receptor (FXR) Agonism

TERN-101 is a potent, non-steroidal FXR agonist, with enhanced liver distribution being developed for the treatment of NASH. FXR is a nuclear receptor that is highly expressed in the liver and small intestine. FXR agonism has demonstrated improvement over placebo in regression of histological liver fibrosis without progression of NASH in a late-stage study, demonstrating the potential for FXR agonists to be a new treatment modality for NASH. TERN-101 has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of NASH.

About NASH

Non-alcoholic steatohepatitis (NASH) is a severe form of non-alcoholic fatty liver disease (NAFLD), which is caused by the accumulation of excess fat in the liver. NASH is associated with chronic liver inflammation and liver cell injury, and it can lead to fibrosis, cirrhosis, and eventually liver cancer or liver failure. Global rates of NAFLD and NASH are increasing rapidly, in tandem with rising rates of obesity. There is currently no approved medication for the treatment of NASH.

About Terns Pharmaceuticals

Terns Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing best-in-class single-agent and combination therapies to treat non-alcoholic steatohepatitis (NASH) and other liver diseases. The company’s liver-selective FXR-agonist, TERN-101, is currently conducting a multi-center, randomized, double-blind, placebo-controlled Phase 2a clinical trial designed to evaluate efficacy, safety, and pharmacokinetics in 96 presumed NASH patients who receive placebo or TERN-101 at various dose levels for 12 weeks. Terns recently announced positive Phase 1 clinical data for its highly selective SSAO inhibitor, TERN-201, demonstrating potent and sustained target engagement. In addition,

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