FDA Approves Supernus Novel Non-Stimulant Treatment for ADHD

 Shares of Supernus Pharmaceuticals were climbing in premarket trading this morning after Friday’s announcement the U.S. Food and Drug Administration (FDA) approved the company’s non-stimulant treatment for attention-deficit hyperactivity disorder (ADHD) in pediatric patients 6 to 17 years of age.

Jack A, Khattar, president and chief executive officer of Rockville, Md.-based Supernus, said the company believes Qelbree (viloxazine extended-release capsules) is a unique alternative for the treatment of ADHD, which is often treated with medications that are considered a controlled substance.

“Qelbree provides prescribing physicians and patients living with ADHD a therapy that is not a controlled substance with proven efficacy and a tolerable safety profile. We are grateful to the patients, families and their care givers who participated in and supported our research,” Khattar said in a statement.

Qelbree, formerly known as SPN-812, is a serotonin norepinephrine modulating agent, a novel non-stimulant for the treatment of ADHD. The active ingredient in SPN-812, viloxazine hydrochloride, has an extensive safety record in Europe, where it was previously marketed for many years as an antidepressant, the company said.

The approval of Qelbree is supported by data from an extensive development program consisting of four Phase III clinical trials that studied more than 1000 pediatric patients from the age of 6 to 17 years.

Qelbree does come with a black-box warning. Qelbree may increase suicidal thoughts and actions in some children with ADHD, especially within the first few months of treatment or when the dose is changed. Qelbree should not be taken by patients that also take certain anti-depression medicines, especially those called a monoamine oxidase inhibitor or MAOI, or certain asthma medicines.

Supernus resubmitted its New Drug Application to the FDA in February after receiving a Complete Response Letter from the regulatory agency last year. In the CRL, the FDA raised concern about the company’s in-house laboratory that conducts analytical testing, which had recently moved to a new location. In the NDA resubmission Supernus removed reference to its in-house laboratory and addressed other contents of the CRL. Following the receipt of the CRL, Supernus and the FDA held a Type A meeting in January 2021 to discuss the CRL and the requirements for the NDA resubmission. Once the NDA was accepted, the FDA reclassified the NDA as a Class I review to expedite the evaluation of the medication.

Between the time the company received the CRL and resubmitted its NDA, Supernus announced positive topline Phase III results for Qelbree that showed efficacy on both hyperactivity/impulsivity and inattention subscales with statistical significance in adult patients. The data from this study will be used to support an additional indication for Qelbree. Supernus plans to file a supplemental New Drug Application to the FDA in the second half of 2021.

https://www.biospace.com/article/maryland-based-supernus-pharmaceuticals-wins-fda-approval-for-novel-non-stimulant-adhd-treatment/

Emergent BioSolutions Gets HHS Modified Order to Expand Manufacturing for J&J Vax

  Emergent BioSolutions Inc. (NYSE:EBS) today announced that the Company continues to be on track with its manufacturing agreements related to COVID-19 vaccines and confirmed that there are no changes to its financial guidance for 2021. In addition, the Company received a contract modification to increase the original task order by $23 million from Biomedical Advanced Research and Development Authority (BARDA), which is part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) at the U.S. Department of Health & Human Services (HHS).

The $23 million will be used for the purchase of biologics manufacturing equipment specific to Johnson & Johnson’s COVID-19 vaccine for the potential expansion of manufacturing of that bulk drug substance into a third suite of Emergent’s Baltimore Bayview facility. In addition, Emergent expects to align with the U.S. government and AstraZeneca on a mutually agreed ramp down of manufacturing for AstraZeneca’s COVID-19 vaccine bulk drug substance.

“Emergent is actively working with the U.S. government to fulfill its policy objectives consistent with the Center for Innovation in Advanced Development and Manufacturing (CIADM) that was put in place under the Obama-Biden Administration in 2012,” said Robert G. Kramer, president and chief executive officer of Emergent. “This unique public-private partnership has been a key element in our ability to quickly scale up to produce COVID-19 vaccines at a current rate of more than one billion dose-equivalents annually.”

Emergent continues to own the facility and perform its contracts consistent with its obligations to all of its customers and in compliance with the regulatory standards promulgated by the FDA and all other applicable regulatory authorities.

https://finance.yahoo.com/news/emergent-biosolutions-track-respect-covid-020900595.html

Acadia's Nuplazid Receives Regulatory Rejection for Dementia-Related Psychosis

 The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) over Acadia Pharmaceuticals’ supplemental New Drug Application (sNDA) for Nuplazid for hallucinations and delusions associated with dementia-related psychosis. The drug, pimavanserin, is the first and only drug approved for hallucinations and delusions associated with Parkinson’s disease psychosis.

The FDA’s CRL noted a lack of statistical significance in some of the dementia subgroups, claiming not enough patients with specific less common dementia subtypes failed to show effectiveness. The company points out that they had previous agreements with the Division of Psychiatry over the study design for the pivotal Phase III HARMONY study that targeted a broad DRP patient population analyzed as a single group.

The HARMONY study hit its prespecified primary and secondary endpoints. They further noted that statistical separation by dementia subgroups and patient minimums for different subgroups were not in the prespecified requirements.

There has been speculation this year that the FDA has been getting tougher on approvals, and this rejection would seem to support that argument.

The Acadia press release hints that the company is unhappy with this decision. It had been forewarned. Only a few weeks ago executives shocked investors when they said the agency wasn’t willing to negotiate on the label.

 “Acadia stands behind the robustly positive results from the pivotal Phase III HARMONY study and the prospectively agreed trial design and criteria for establishing efficacy in DRP. Over the entire course of the review, the Division did not raise any concerns regarding the agreed upon study design, including the issues raised in the CRL,” said Steve Davis, Acadia’s chief executive officer. “We will immediately request a Type A meeting to work with the FDA to address the CRL and determine an expeditious path forward for the approval of pimavanserin in DRP.”

Paul Matteis, analyst with Stifel, wrote in a note to investors, “Pimavanserin’s efficacy in the relapse prevention trial was most robust in the Parkinson’s sub-population, and one could argue that this is the primary reason why the trial was stopped at the interim. We had figured this detail could be overlooked since the data for ADP was still pretty good (14 relapses placebo, 8 drug), but data in some of the other rarer subgroups is less consistent, and perhaps if one is not a believer in the ‘acute’ Phase II trial the same numbers issue with the Phase III become more prominent. Bottom line: our view is that Acadia will likely need to run one or even two more trials here, and while pimavanserin has always seemed to be a very safe drug, it’s efficacy is at the low end of the range for atypical antipsychotics, which makes any future trial (especially one that needs to demonstrate clear acute efficacy in a placebo-controlled manner) fairly risky, in our opinion.”

The FDA has said in its CRL that the company’s Phase II Alzheimer’s disease psychosis study -019, which was used for supportive data in the sNDA, was not adequate and well controlled. Acadia, on the other hand, argues that “these observations impact neither the positive results on the study’s primary endpoint, nor the study’s overall conclusions of efficacy.”

https://www.biospace.com/article/fda-gives-a-thumbs-down-on-acadia-s-nuplazid-for-dementia-related-psychosis/

Cidara, Janssen to Develop, Commercialize AVCs for Flu Prevention, Treatment

  Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company developing long-acting therapeutics designed to transform the standard of care for patients facing serious fungal or viral infections, today announced that it has entered into an exclusive worldwide license and collaboration agreement with Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize Cidara’s Cloudbreak^® antiviral conjugates (AVCs) for the prevention and treatment of seasonal and pandemic influenza. This agreement was facilitated by Johnson & Johnson Innovation.

Under the collaboration, Cidara will be responsible for the development and manufacturing of the first influenza AVC, CD388, into the clinic and through Phase 2 clinical development, and Janssen will be responsible for late-stage development, manufacturing, registration and global commercialization. Cidara will receive an upfront payment of $27 million and Janssen will fund all future research, development, manufacturing and commercialization for CD388. In addition to the upfront payment, Cidara is eligible to receive up to an aggregate of $753 million in budgeted R&D funding and in development, regulatory and commercial milestones, plus tiered royalties on worldwide sales in the mid to high single digits. Cidara has the option to co-detail CD388 in the U.S.

Cidara management will hold a conference call and webcast today at 8:30 am ET / 5:30 am PT to discuss its worldwide license and collaboration agreement with Janssen. The dial-in number for the conference call is 1-877-407-0789 (U.S./Canada) or 1-201-689-8562 (international). The conference ID for all callers is 13718338. The live webcast and replay may be accessed by visiting Cidara’s website at https://ir.cidara.com/presentations.

https://www.biospace.com/article/releases/cidara-therapeutics-announces-agreement-with-janssen-to-develop-and-commercialize-avcs-for-the-prevention-and-treatment-of-influenza-/

Vir Biotechnology: VIR-7831 Shows Neutralizing Activity Against SARS-CoV-2 California Variant

 Vir Biotechnology, Inc. (VIR) reported new preclinical research showing the ability of VIR-7831, the company's investigational SARS-CoV-2 monoclonal antibody, to maintain its neutralizing activity against a mutation in the receptor binding domain of SARS-CoV-2, called L452R, which is found in the California variant.

The company noted that the study results also showed the L452R mutation reduced both the neutralization potency of plasma from vaccinated and convalescent donors and the neutralization activity of 14 RBD-specific and 10 N-terminal domain-specific monoclonal antibodies.

George Scangos, CEO of Vir Biotechnology, said: "We believe VIR-7831 could significantly impact both the trajectory of the pandemic and the outcomes of patients facing the more dire consequences of COVID-19. We look forward to continuing to work with global regulatory authorities to bring VIR-7831 to patients as quickly as possible."

https://www.nasdaq.com/articles/vir-biotechnology%3A-vir-7831-shows-neutralizing-activity-against-sars-cov-2-california

Exelixis: FDA Accepts Investigational New Drug Application for XB002 in Solid Tumors

 Exelixis, Inc. (Nasdaq: EXEL) today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug Application (IND) to evaluate the safety, tolerability, pharmacokinetics and preliminary antitumor activity of XB002 in patients with advanced solid tumors. As a next-generation tissue factor-targeting antibody-drug conjugate (ADC), XB002 has the potential for an improved therapeutic index and may provide a favorable safety profile compared with earlier-generation tissue factor-targeting ADCs.

“The acceptance of our Investigational New Drug Application for XB002 gets us one step closer to our first biologic entering the clinic and learning more about its potential to help patients with difficult-to-treat cancers,” said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. “Considering XB002’s promising preclinical data and potential differentiation from other tissue factor-targeting antibody-drug conjugates, we look forward to initiating our phase 1 trial in patients with advanced solid tumors.”

XB002 (formerly ICON-2) is an ADC composed of a human monoclonal antibody against tissue factor that is conjugated to a cytotoxic agent. After binding to tissue factor on tumor cells, XB002 is internalized, and the cytotoxic agent is released, resulting in targeted tumor cell death. XB002 is a rationally designed next-generation ADC that leverages proprietary linker-payload technology.

Preclinical data demonstrated that XB002 binds to tissue factor without affecting the coagulation cascade, in contrast with prior therapies in this class. The data also demonstrated encouraging activity of XB002 in multiple solid tumor cancer models and improved tolerability compared with other tissue factor-targeting ADCs. XB002 has shown significant tumor growth inhibition and, in some cases, complete regression. The rational design and preclinical profile of this novel tissue factor-targeting ADC suggest that, if born out in clinical evaluation, XB002 could have an improved therapeutic index and favorable safety profile compared with earlier tissue factor-targeting ADCs.

https://www.argus-press.com/news/national/article_64de30d4-3ddf-5a2b-ac4f-205f9104e49f.html

Ionis Pharma Starts Late-stage Study Of ION363 To Treat ALS

  Ionis Pharmaceuticals, Inc. (IONS) has begun the late-stage study of its investigational drug, ION363 in patients with amyotrophic lateral sclerosis (ALS),the company said in a release.

ALS is a rare, rapidly progressing and fatal neurodegenerative disorder.

Phase III study of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma gene (FUS) is a global, multi-center study in up to 64 patients.

"There is an urgent need for novel treatments for all forms of ALS, a devastating disease that affects far too many patients and their families. Advancement of ION363 to a pivotal trial is the latest example of the power of Ionis' antisense technology to potentially target the root causes of neurological diseases," said C. Frank Bennett, Ph.D., Ionis' chief scientific officer and franchise leader for neurological programs.

https://www.nasdaq.com/articles/ionis-pharma-starts-late-stage-study-of-ion363-to-treat-als-quick-facts-2021-04-05