Your Daily Pharma Scoop: Conatus Data, Solid Biosciences' SGT-001, Ultragenyx's Crysvita Approved

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About: Conatus Pharmaceuticals (CNAT)
by: Avisol Capital Partners
Summary

Conatus reported promising data with IDN-7314.

Solid Biosciences received FDA’s Full Clinical Hold letter related to the suspended IGNITE DMD study.

Ultragenyx announced that the FDA has approved Crysvita.

Analysis focus: Conatus

Today we will discuss Conatus (NASDAQ:CNAT), which fell more than 4% in regular trading even as the company reported promising data with IDN-7314.

IDN-7314 is a pan-caspase inhibitor. CNAT today announced results from in vitro and in vivo studies with the investigational candidate. The studies were sponsored by CNAT and conducted in collaboration with the Department of Pathobiology & Diagnostic Investigation and the Institute for Integrative Toxicologyat Michigan State University. Results from the studies showed reductions of hepatic tissue factor-driven coagulation.

IDN-7314 has already been granted an Orphan Drug designation by the FDA and the EMA for the treatment of primary sclerosing cholangitis (NASDAQ:PSC).

Conatus shares have struggled in recent weeks after the setback with its lead candidate emricasan in one of the four studies in NASH. Earlier this month, the company had announced top-line results from the exploratory phase 2b POLT-HCV-SVR proof-of-concept study in liver transplant patients with fibrosis or cirrhosis. The trial failed to meet its primary endpoint, however, we believe that the sharp pullback was unjustified. This is mainly because the failure of the trial does not impact other studies as the patient population in POLT-HCV-SVR study is different compared to other three phase 2b studies. The most important data readout will be from the ENCORE-LF study in the second half of 2019. The study is evaluating 210 patients with decompensated NASH cirrhosis. While the NASH treatment development pipeline is extremely crowded, CNAT is focusing on an area where there is no competition. If the trial succeeds, the upside in CNAT could be significant.

Later this year, CNAT will also report data from the ENCORE-PH study, which is evaluating 240 patients with NASH cirrhosis and severe portal hypertension. Top-line results from this study are expected in the second half of 2018. There is also the ENCORE-NF study, which is evaluating patients with NASH fibrosis. With multiple catalysts coming up, we believe this is a good time to enter CNAT.

Stocks in News: Analysis of SLDB, CYTR

Solid Biosciences (NASDAQ:SLDB) announced the receipt of FDA’s Full Clinical Hold letter related to the suspended IGNITE DMD study of gene therapy SGT-001.

Analysis: In the letter, the FDA requested additional information before the clinical trial can be resumed. This includes an assessment of the underlying etiology of the serious adverse event (patient was hospitalized), the patient's clinical status and laboratory parameters and any additional measures to address patient safety. The FDA has also notified the company in the letter that it has satisfactorily addressed the manufacturing-related questions that led to the partial clinical hold on the high dose of SGT-001 used in IGNITE DMD. With this, the partial clinical hold has now been resolved, enabling the company to use a single lot or dose administration and limiting the number of vials of product required to treat each patient. Solid Biosciences expects to formally respond to the FDA in the coming weeks.

CytRx (NASDAQ:CYTR) announced encouraging preclinical data with its Linker Activated Drug Release (LADR) drug candidates in a range of cancers.

Analysis: The company reported that LADR-7, LADR-8, LADR-9 and LADR-10, enable drug compounds to be molecularly bound to the protein albumin in the bloodstream which allows its controlled release at the tumor site. Furthermore, in preclinical testing, LADR-7 produced a 93% response rate in a melanoma model, an 86% response rate in ovarian cancer and a 100% response rate in non-small cell lung cancer (NSCLC). LADR-8 showed a 67% response rate in ovarian cancer but only 27% in NSCLC.

In other news

Ultragenyx Pharmaceuticals (NASDAQ:RARE) announced that the FDA approved its Crysvita (burosumab) for the treatment of patients with a rare musculoskeletal disorder called X-linked hypophosphatemia (XLH).

Amgen (NASDAQ:AMGN) announced that its Aimovig showed a significant treatment effect in a phase 3b study LIBERTY in patients who had failed two-to-four preventative treatments. The results, which were presented at the Annual Meeting of the American Academy of Neurology in Los Angeles, showed that over a 12-week treatment period, 30.3% of patients receiving monthly subcutaneous injections of Aimovig experienced at least a 50% reduction in migraine days compared to 13.7% for placebo (p<0.002).

Catabasis (NASDAQ:CATB) announced that it will restructure its operations, cutting its workforce by 42% and saving $3.3 million in annualized personnel costs. The company will record a charge of $1 million this quarter related to the costs of restructuring.

Aeglea Bio (NASDAQ:AGLE) has launched a public offering of 4.5 million shares of its common stock. The company is yet to announce the price and terms of the offering.

Bellicum (NASDAQ:BLCM) priced an upsized 8 million share offering at $7.50 per share. The gross proceeds from the offering totaled $60 million. Underwriters to the offering have been granted an option to acquire an additional 1.2 million shares to cover for over-allotment, if any.

Provectus Biopharmaceuticals (PVCT) announced that the Japan Patent Office has granted and the European Patent Office allowed its patent application for the combination of PV-10 with systemic immunomodulatory therapy.

Calithera Biosciences (NASDAQ:CALA) announced that the FDA has granted its CB-839 in combination with CABOMETYX a Fast Track review for the treatment of patients with metastatic renal cell carcinoma who have received one or two prior lines of therapy, including at least one vascular endothelial growth factor tyrosine kinase inhibitor or a combination of Opdivo and Yervoy.

Fibrocell Science (NASDAQ:FCSC) announced that it will initiate a comprehensive review of strategic alternatives aimed at boosting shareholder value.

INSYS Therapeutics (NASDAQ:INSY) announced that it plans to advance the inhaled delivery of dronabinol for the potential treatment of cancer-related anorexia and Alzheimer's disease.

Paratek Pharmaceuticals (NASDAQ:PRTK) announced that it has commenced a $125 million debt offering of convertible senior subordinated notes due 2024. Price, yield and terms of the offering have not yet been announced.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.