Search This Blog

Friday, November 2, 2018

Biotech Investors: Mark Your Calendar For These November FDA Dates


October was a volatile month for the markets, and biotech stocks were no exception. The iShares NASDAQ Biotechnology Index IBB had slipped about 17 percent in the period. This compares to S&P 500’s 7 percent retreat.
As we enter the last leg of the year, here are a few PDUFA catalysts in November that could help determine as to where biotech stocks are headed.
PDUFA dates are deadlines for the FDA to review new drugs. The FDA is normally given 10 months to review new drugs. If a drug is selected for priority review, the FDA is allotted six months to review the drug. These time frames begin on the date that an NDA is accepted by the FDA as complete.

Can FDA Spring In a Surprise And Offer A Reprieve to Trevena Shares?

  • Company: Trevena Inc TRVN
  • Type of Application: NDA
  • Candidate: Oliceridine 1 milligram/milliliter injection
  • Indication: management of moderate-to-severe acute pain in adult patients for whom an intravenous opioid is warranted
  • Date: Nov. 2
FDA’s Anesthetic and Analgesic Drug Products Advisory Committee, which met Oct. 11 to discuss the NDA, ruled against approving the drug by an 8-7 margin. Trevena’s shares have plummeted about 70 percent since Oct. 8 in reaction the release of the FDA Committee’s briefing document.

AcelRx Awaits Positive Verdict After FDA Panel Backed Its Pain Drug

  • Company: AcelRx Pharmaceuticals Inc ACRX
  • Type of Application: NDA
  • Candidate: Dsuvia (sufentanil sublingual tablets)
  • Indication: Management of moderate-to-severe acute pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate
  • Date: Nov. 3
The Anesthetic and Analgesic Drug Products Advisory Committee, which met Oct. 12, voted 10-3 in favor of recommending the approval of the pipeline candidate.

Will Theratechnologis Get Nod For A More Concentrated Fat-Fighting Regimen In HIV Patients?

  • Company: Theratechnologies Inc THERF
  • Type of Application: sBLA
  • Candidate: Single vial formulation of Egrifta
  • Indication: Reducing abdominal fat in HIV- patients
  • Date: Nov. 3
Egrifta was initially approved by the FDA in Nov. 2010 to reduce visceral adipose tissue – deep belly fat surrounding the liver, stomach and other abdominal organs – in HIV patients, who have experienced lipodystrophy as a side effect of antiretroviral therapy.
The company is now seeking an expansion in label to include a single-vial formulation based on bioequivalence studies. The single-vial formulation is four times more concentrated than the currently commercialized formulation.

Coherus Seeks FDA Approval For Neulasta Biosimilar

  • Company: Coherus Biosciences Inc CHRS
  • Type of Application: BLA
  • Candidate: CHS-1701, a Neulasta biosimilar
  • Indication: Reduce Indidence of Neutropenia
  • Date: Nov. 3
Following the issue of a complete response letter in June 2017, Coherus made a resubmission, with the resubmitted application accepted for review in May.
Neulasta is Amgen, Inc. AMGN‘s white-cell boosting therapeutic used to decrease the incidence of neutropenia (abnormally low neutrophil count), which can lead to patients contracting potentially life threatening infections following chemotherapy.

Merck’s Wonder Cancer Drug Now For Liver Cancer?

  • Company: Merck & Co., Inc. MRK
  • Type of Application: sBLA
  • Candidate: Keytruda
  • Indication: Previously treated patients with advanced hepatocellular carcinoma
  • Date: Nov. 9
Merck is seeking FDA approval for Keytruda for a new indication, namely hepatocellular carcinoma – a form of liver cancer. The application is based on the Phase 2 KEYNOTE-224 study.

Novartis Generic Unit’s Humira Biosimilar Up Before FDA

  • Company: Novartis AG NVS
  • Type of Application: BLA
  • Candidate: Adalimumab biosimilar
  • Indication: Inflammatory diseases such as rheumatoid arthritis, plaque psoriasis, Crohn’s disease and ulcerative colitis
  • Date: Nov. 16 (estimated)
The BLA filed by Novartis’ Sandoz generic unit was accepted Jan. 16, 2018. Given the 10-month standard review period, the PDUFA date is estimated to be Nov. 16. The rights to Humira, the original Adalimumab, are now owned by AbbVie Inc ABBV.

Mallinckrodt’s Pain Medication Under FDA Scanner

  • Company: Mallinckrodt PLC MNK
  • Type of Application: NDA
  • Candidate: MNK-812 (abuse-deterrent formulation of immediate-release, single-entity oxycodone tablets)
  • Indication: Manage pain severe enough to require an opioid analgesic
  • Date: Nov. 16
An Adcom meeting for the candidate is scheduled for Nov. 14.

Loxo Seeks Approval For Novel Solid Tumor-Targeting Drug

  • Company: Loxo Oncology Inc LOXO
  • Type of Application: NDA
  • Candidate: Larotrectinib (LOXO-101)
  • Indication: Treating adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion.
  • Date: Nov. 26
The NDA was granted priority review status. This drug has a unique mechanism of attacking the tumor’s genetics, rather than its site of origin in the body.

Catalyst Pharma Looks For A Catalyst In Rare Autoimmune Disorder Drug Approval

  • Company: Catalyst Pharmaceuticals Inc CPRX
  • Type of Application: NDA
  • Candidate: Firdapse
  • Indication: Lambert-Eaton myasthenic syndrome (LEMS)
  • Date: Nov. 28
Following the original filing, the FDA issued a refuse-to-file letter in February 2016. Catalyst resubmitted the application, which was subsequently given priority review status. The PDUFA date is set for Nov. 28.
Firdapse, according to the company, is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS, a rare autoimmune disorder.

FDA To Rule On Pfizer’s Lung Cancer Drug

  • Company: Pfizer Inc. PFE
  • Type of Application: NDA
  • Candidate: Lorlatinib
  • Indication: metastatic non-small cell lung cancer, or NSCLC
  • Date: October
Lorlatinib is an anaplastic lymphoma kinase, or ALK, tyrosine kinase inhibitor, or TKI for the treatment of patients with ALK-positive metastatic NSCLC previously treated with one or more ALK TKIs.
Following FDA’s decision to extend the review period by three months, the initial PDUFA date of August was pushed backward to October.

Adcom Review

A joint committee of Psychopharmacologic Drugs Advisory Committee and Drug Safety and Risk Management Advisory Committee will discuss Nov. 1 efficacy, safety and risk-benefit profile ofAlkermes Plc ALKS‘s NDA for buprenorphine and samidorphan sublingual tablets, being evaluated as an adjunctive treatment of major depressive disorder.
The same joint committee will discuss Nov. 2 the efficacy, safety, and benefit-risk profile ofSAGE Therapeutics Inc SAGE‘s brexanolone 5 mg/mL intravenous injection, tested for postpartum depression.
Mallinckrodt’s generic SpecGx Inc’s NDA for an immediate-release oral tablet formulation of oxycodone – MNK-812 – will be discussed by the same joint committee Nov. 14. This pipeline asset is intended to manage pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate.
The joint committee will also determine whether the sponsor company demonstrated that the abuse-deterrent properties of the proposed product are sufficient to include this information in the product label, and whether the product should be approved.
Posted by at No comments:

Lake Street affirms $60 target on Tandem Diabetes after Q3 results


Tandem Diabetes’ “strong momentum” continued during Q3 as revenue growth accelerated from 43.7% in Q1 to 60.0% in Q2 and 71.3% during Q3, Lake Street analyst Brooks O’Neil tells investors in a post-earnings research note. The analyst sees the strong results continuing into Q4 and affirms a buy rating on the shares with a $60 price target.
https://thefly.com/landingPageNews.php?id=2816635
Posted by at No comments:

Amazon launches exclusive diabetes and hypertension devices


Amazon has launched an exclusive range of medical devices to help people manage their diabetes and hypertension at home.
The Choice devices, produced by the healthcare consultancy, Arcadia Group – which is not affiliated with the British fashion brand of the same name –include blood glucose monitors, blood pressure monitors and test strips.
Choice estimates that diabetes and hypertension affect up to 130 million Americans and hopes that its devices will help those afflicted to benefit from daily monitoring and progress tracking over time.
Conventional models and devices enabled with Bluetooth connectivity features will be available to buy, with pricing said to offer better value over devices that can be bought in pharmacies, according to the company.
Bob Guest, CEO of Arcadia Group, said: “The Choice brand is all about accessible wellness. Consumers no longer need to drive to a store to stand in-line and purchase their medical devices and supplies.
“Now, in the privacy of their home, consumers can review, compare and purchase the products of their choice. No insurance is required. Therefore, customers have the freedom of choice. They will no longer be told by their insurance company what brand they can buy. Choice is freedom.”
Guest went on to describe the products as “best in class and very affordable”. He said the company intends to incorporate voice-driven measurement interpretation as well as individualize wellness recommendations via Amazon’s Echo home speaker devices.
“This is all possible with Alexa, and will provide patients with a wellness experience not available until now,” he said.
Amazon is expanding its health-related products to capture more of the market. In June this year, the online retailer announced it is buying PillPack for $1 billion.
The move is expected to disrupt the pharmacy market by delivering pre-sorted medicines and refills to customers’ doors.
PillPack describes itself as “a full-service pharmacy that delivers a better, simpler experience for people managing multiple medications”. The firm liaises with doctors and also provides customer support.
Posted by at No comments:

NeuroPointDX, looking for earlier diagnosis, launches autism blood test


NeuroPointDX has launched what it says is the first objective blood test set up to help diagnose children with autism spectrum disorder earlier, which could help with quicker treatments for some.
The so-called NPDX AA test works by identifying metabolic subtypes associated in young children with autism spectrum disorder (ASD).
This blood plasma‐based test can screen kids as young as 18 months; if positive, they can then be referred to a neurodevelopmental specialist, which could lead to treatment or help with developing cognitive and adaptive skills, while also focusing on reducing ASD symptoms, which can impact communication and other areas.

The test works by detecting amine imbalances in the blood plasma of children with “very precise thresholds,” according to the firm, and was developed from patient samples out of the Children’s Autism Metabolome Project (a.k.a. CAMP), a 1,100‐subject study and the largest one to date focused on the metabolism of children with ASD.
These imbalances, which can be picked up in around of 30% of children with ASD, are not identified by other currently available metabolic tests.
ASD is made up of a varied set of neurodevelopmental disorders that comes from differences in underlying genetic, metabolic and environmental factors, resulting in a spectrum of cognitive, behavioral and biological profiles.
No reliable, objective biomarkers have existed to aid in the diagnosis of ASD, which is currently diagnosed based on the behavioral characteristics shown by an affected child. While a diagnosis is possible in children as young as 24 months, the average age of ASD diagnosis in the U.S. is, according to NeuroPointDX, more than four years old, so it hopes to get an edge on those already on the market.
“Early diagnosis is important because intensive behavioral therapy has been shown to improve the symptoms of autism; the benefit of such intervention is greater the earlier it is started,” the company explains.

Elizabeth Donley, president and CEO of NeuroPointDX, said: “This test provides specific information about the precise cutoffs for each metabolite measured, based on data from the large, rigorous CAMP study. We have taken great care to identify these thresholds for optimal accuracy and clinical utility, using data from the CAMP study. The result provides a biological basis for further evaluation by a neurodevelopmental specialist. Moreover, in conjunction with advice from a physician, they may point to a treatment strategy for some children.”
And there’s plans for more: “We are continuing to mine data from the CAMP study to identify and validate additional metabolic subtypes in children with ASD,” added Bob Burrier, CCO and VP of Research & Development at NeuroPointDX.
“Our aim is to further build on the utility of this test to identify a greater percentage of children with additional metabolic subtypes. The metabolic biomarkers we have identified will serve as targets for new therapies ranging from pharmaceuticals to dietary supplements, bringing precision medicine to the diagnosis and treatment of this neurodevelopmental disorder.”
NeuroPointDX says it has plans to bring a second test panel to market in 2019. The company is also seeking to identify potential treatments for ASD based on differences in the metabolism of these children.
But getting your hands on this test won’t be easy: The NPDX AA is only available on a “limited basis” for now through NeuroPointDX’s early access program, which “will be expanded over time,” with the company adding that its test is not currently covered by insurance.
Posted by at No comments:

Mallinckrodt Has Data on H.P. Acthar in Rheumatoid Arthritis


Mallinckrodt plc(NYSE: MNK), a leading global specialty pharmaceutical company, is reporting interim data1 at the midway point of enrollment in the ongoing Phase 4, multicenter study assessing the efficacy and safety of H.P. Acthar Gel in patients with persistently active rheumatoid arthritis (RA) despite receiving disease-modifying anti-rheumatic drugs (DMARDs) and corticosteroids. The data will be reported in a poster presentation on Tuesday, Oct. 23 at the 2018 American College of Rheumatology/Association of Rheumatology Health Professionals (ACR/ARHP) Annual Meeting (Oct. 19-24) in Chicago, IL (see also Mallinckrodt plc).
Earlier at this year’s 2018 ACR/ARHP Annual Meeting, Mallinckrodt presented pre-clinical findings2 on the immune modulatory effect of H.P. Acthar Gel in a rat adjuvant-induced arthritis (AIA) model and the effect on osteoclast-mediated bone resorption compared to an immunosuppressive steroid.
Posted by at No comments:

Lilly buys Siga’s Priority Review voucher gained via smallpox approval


Eli Lilly and Co. (NYSE:LLY) acquired a Priority Review voucher from Siga Technologies Inc. (NASDAQ:SIGA) for $80 million. The voucher, which Siga obtained in July upon approval of TPOXX tecovirimat to treat smallpox, was the first FDA awarded under its Material Threat Medical Countermeasure voucher program.
TPOXX, an oral small molecule antiviral, is the first drug FDA has approved to treat smallpox (see “Siga Gets Priority Review Voucher For First Approved Smallpox Drug”).
Priority Review voucher prices have ranged from $67.5 million to $350 million since the first one was issued in 2009. Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151) and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) sold one in July for $80.6 million (see “Voucher Equilibrium” and “Kyowa, Ultragenyx Sell Priority Review Voucher For $80.6M”).
The medical countermeasure program was created under the 21st Century Cures Act, and awards vouchers for “medical products intended to diagnose, prevent or treat diseases or conditions associated with threats and emerging infectious diseases.”
Posted by at No comments:

Moleculin Biotech: Announces Significant Milestone Achieved in Glioblastoma Trial


Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, today announced positive progress in the Phase 1 clinical trial of its immuno-stimulating STAT3 inhibitor, WP1066, with initial results showing bioavailability of the drug in patients.
Although this data is preliminary, it represents a significant milestone for the development of WP1066, commented Dr. Donald Picker, Moleculins Chief Science Officer. In the first two cohorts of the Phase 1 study, we are already seeing measurable levels of the drug in the patients plasma resulting from oral administration. Knowing we can deliver drug this way opens the door for further development and expanded clinical activity.
Walter Klemp, Moleculins Chairman and CEO added, We believe WP1066 is a first-in-class compound capable of stimulating a natural immune response in animal models while directly attacking tumors by modulating transcriptional activity and repressing what we call oncogenic transcription factors. Chief among these is STAT3, considered a master regulator of tumor progression. While activity in animal models has been very promising, one of the goals of this trial was to determine the potential for bioavailability in humans. The initial positive indications of this clinical trial increase our confidence that WP1066 has the potential to become an important drug in the treatment of certain cancers. The initial demonstration of human bioavilability is an important milestone.
Posted by at No comments:
Subscribe to: Posts (Atom)