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Acceleron’s Label Expansion, And Other News: The Good, Bad And Ugly Of Biopharma (NASDAQ:XLRN)

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Acceleron Granted FDA Approval for Luspatercept Label Expansion

Acceleron (XLRN) reported that the FDA has granted approval for the use of Reblozyl for treating anemia in certain adult patients with very low to intermediate risk myelodysplastic syndromes. These are the patients who did not respond to treatment with an erythropoiesis stimulating agent and require minimum two red blood cell units over eight weeks. Acceleron is collaborating with Bristol Myers Squibb for developing this drug candidate.

Acceleron noted that the drug is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia. Habib Dable, President and Chief Executive Officer, Acceleron said “We are enormously grateful to the patients, families and caregivers who participated in and supported the Reblozyl clinical trials, and to the researchers at Acceleron and beyond who, more than a decade ago, began this important quest to address patients’ chronic anemias.”

Acceleron had used the data from pivotal Phase 3 MEDALIST trial, which is a randomized and double blind trial. It is also placebo controlled and multi center study aimed at assessing the efficacy and safety of the drug candidate in treating patients with IPSS-R-defined very low, low and intermediate-risk non-del myelodysplastic syndromes with ring sideroblasts. All the patients enrolled in the trial were red blood cell transfusion-dependent and were either refractory or intolerant to prior erythropoiesis-stimulating agent therapy or were ESA naïve and unlikely to respond due to endogenous serum erythropoietin ≥200 U/L, and had no prior treatment with disease modifying agents.

This is the second indication approval for the drug. Luepatercept was initially approved in November 2019 for treating anemia in adults suffering from thalassemia who require regular RBC transfusions. The latest approval is important as it will serve patients with myelodysplastic syndromes who have very limited options in terms of treatment regimens.

This recent approval is a major relief for the firm which recently suffered a significant setback as it reported disappointing readout for its rare muscle disease drug candidate. ACE 083 was being tested for treating patients with Charcot Marie Tooth disease, which leads to muscle weakness and nerve damage. While the trial met the main goal by demonstrating a statistically significant increase in mean total muscle volume compared to placebo. However, there were no significant functional or quality of life improvements, prompting the company to discontinue further work on the drug. Acceleron stated that the decision was taken to channel the resources for other development purposes including developing drug candidates for pulmonary and hematology conditions.

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Earlier this year, Acceleron had also reported positive Phase 2 data for its lead drug candidate sotatercpet. The drug is being developed to treat patients suffering pulmonary arterial hypertension. While the company did not provide specific details it said that the drug met primary and key secondary endpoints. The study involved nearly 100 patients suffering from pulmonary arterial hypertension, which results in obstruction of lung arteries, causing high blood pressure. However, it needs to be noted that the development is still in initial stage and there is significant time period ahead for the drug to reach market.

Acceleron is one of the very few stocks which have bucked the trend. Despite ongoing pandemic scare, the stock is up over 50 percent this year so far.

Gilead Sciences Receives European Nod for Remdesivir Use

Gilead Sciences (GILD) announced that European authorities have allowed its remdesivir to be used on compassionate basis for treating COVID 19. Compassionate use involves the use of an unauthorized drug outside a clinical study in individual patients under strictly controlled environment. The European Medicines Agency stated that remdesivir has demonstrated active action against the new coronavirus in laboratory setup. However, the agency also noted that there is only limited data available about its effectiveness.

Remdesivir was earlier tested for treating Ebola but the drug candidate failed the test. The European Agency said, “The CHMP (EMA’s Committee for Medicinal Products for Human Use) encourages the company to make remdesivir available in a fair and transparent way to those member States wishing to take part in international clinical trials or treat patients in compassionate use programmes,” The WHO is in the process to launch a trial for assessing the potential of remdesivir as well as three other drug candidates for fighting coronavirus.

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It is expected that clinical data about the efficacy of the drug may arrive as early as by the end of this month. If remdesivir is successful in proving its utility, it may prove to be a massive win for Gilead. The company is currently struggling with steep drop in its revenue. The company posted $22.4 billion in its revenue for the year 2019, down from $30.4 billion in revenue it had reported for 2016.

Gilead’s remdesivir was granted an orphan status by the FDA on March 23rd. However, later the company appealed the agency to withdraw this designation. The company stated that it is capable of maintaining an expedited timeline even without this designation.

Sun BioPharma Suspends Enrolment in Clinical Programs

Sun BioPharma (OTCQB:SNBP) announced that it has put temporary pause on enrolment for its Phase clinical trial for assessing SBP 101 in treating pacreatic cancer patients. However, the company stated that the currently enrolled patients will keep receiving the treatment. Sun Bio cited disruption caused by Covid 19 and ensuing impact on its clinical trials conducted in the United States and Australia.

Sun BioPharma stated that it is following its peers with regard to instituting a recruitment delay for the current Phase 1 dose escalation and expansion study of SBP 101 in conjunction with nab-paclitaxel and gemcitabine in patients suffering from previously untreated metastatic pancreatic ductal adenocarcinoma. Michael T. Cullen, MD, MBA, Sun BioPharma’s President and CEO said, “Given the uncertainties associated with the COVID-19 pandemic including the risk to our supply chain and the limitation and redistribution of clinical resources at our investigative sites, we will pause recruitment through at least May 15th.” The company said that the timeline may be amended again keeping in view the prevailing circumstances.

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Sun BioPharma recently reported its financial number for FY 2019 where it reported its net loss for the entire year at $6.2 million, in comparison to net loss of $5.9 million it had reported for the previous year. Its general and administrative expenses for the year were at $2 million, down 6.4 percent on year over year basis. The company reported 31.7 percent increase in its annual research and development expenses to $2.3 million.

Sun BioPharma engages in developing treatments for urgent medical needs which have not yet been addresses. It is currently focusing on developing SBP 101 of treating pancreatic cancer.

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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.

Editor’s Note: This article covers one or more microcap stocks. Please be aware of the risks associated with these stocks.

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