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Thursday, August 8, 2019

Gossamer Bio Announces Second Quarter 2019 Financial Results

– Five active clinical trials across four clinical programs, with data readouts for all four programs expected in 2020 –

– Company to host conference call today at 4:30 p.m. ET –

Gossamer Bio , Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, today announced its financial results for the quarter ended June 30, 2019 and provided a corporate update.

“Over the last several months, we have made significant progress advancing our diversified development portfolio, with five clinical trials now active,” said Sheila Gujrathi, M.D., Co-Founder and Chief Executive Officer of Gossamer. “We are poised for a steady cadence of data readouts throughout 2020. Supported by a strong balance sheet and our experienced and growing team, we are well positioned to advance toward our goal of becoming an industry leader in immunology, inflammation and oncology.”

Pipeline Updates

GB001: Oral DP2 Antagonist for Asthma and Allergic Disease

Enrollment in the Phase 2b LEDA study in moderate-to-severe eosinophilic asthma is on track, with an interim analysis expected in the first half of 2020. Full results from the LEDA study are expected in the second half of 2020.
Patient enrollment in the TITAN Phase 2 proof-of-concept study in chronic rhinosinusitis, with and without nasal polyps, commenced in the second quarter. Topline data from the TITAN study are expected in the second half of 2020.

GB002: Inhaled PDGFR Inhibitor for Pulmonary Arterial Hypertension (PAH)

During the second quarter, the European Medicines Agency granted orphan medicinal product designation to GB002 for the treatment of PAH.
Sites have been initiated for a Phase 1b translational study in patients with PAH, with patient enrollment expected to begin in the third quarter. Results from the Phase 1b study are expected in the first half of 2020.

GB004: Oral HIF-1α Stabilizer for Inflammatory Bowel Disease

Patient enrollment in a Phase 1b study of active mild-to-moderate ulcerative colitis (UC) began during the second quarter, and the Company expects topline results from the study in the first half of 2020.

GB1275: Oral CD11b Modulator for Oncology Indications

Patient screening in a Phase 1/2 study in selected solid tumors is now underway, with patient enrollment expected to begin in the third quarter of 2019. Following monotherapy dose escalation, we will explore combinations with anti-PD-1 therapy and chemotherapy. Initial data from the Phase 1/2 study is expected in the second half of 2020.
Preclinical data supporting GB1275 were published in the July 3, 2019 edition of Science Translational Medicine by researchers at the Washington University School of Medicine in St. Louis and Rush University.

Financial Results for Quarter Ended June 30, 2019

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of June 30, 2019, were $464.0 million. The Company expects current cash, cash equivalents and marketable securities, and access to its debt facility will be sufficient to fund its operating and capital expenditures into the second half of 2021.

Conference Call and Webcast

Gossamer’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Thursday, Aug. 8, 2019, to discuss its second quarter 2019 financial results and provide a corporate update.

The live audio webcast may be accessed through the Events/Presentations page in the Investors section of the Company’s website at www.gossamerbio.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 1393207
Domestic Dial-in Number: (866) 221-1654
International Dial-in Number: (470) 495-9466
Live Webcast: https://edge.media-server.com/mmc/p/o3xj5mjx

A replay of the audio webcast will be available for 30 days on the Investors section of the Company’s website, www.gossamerbio.com.

https://www.biospace.com/article/releases/gossamer-bio-announces-second-quarter-2019-financial-results/

Akcea, Ionis Gear Up for Second Attempt at Getting Waylivra FDA Approval

Boston-based Akcea Therapeutics and Ionis Pharmaceuticals are gearing up for another attempt at gaining regulatory approval for Waylivra, (volanesorsen), a treatment for patients with familial chylomicronemia syndrome (FCS).

One year ago, the U.S. Food and Drug Administration stunned the two companies when it rejected Waylivra, even after strong support from an advisory committee. There were some concerns over the drug’s safety profile, which may have been the reason for the FDA’s initial rejection. Now though, with final data from the Phase III APPROACH study published in the New England Journal of Medicine, Akcea and its parent company Ionis are ready to try again for approval in the U.S. Waylivra was approved for use in the European Union earlier this year. The study data published in the article “Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome” showed that mean triglyceride levels decreased 77% in Waylivra-treated patients versus an 18% increase in patients in the placebo group. Waylivra is designed to reduce the production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a cnentral role in the regulation of plasma triglycerides in FCS patients. In the Phase III study, the levels of apoC-III also decreased by an average of 84 percent from baseline at three months. The most common adverse events noted in the APPROACH trial were injection site reactions and platelet declines, Akcea said.

Brett P. Monia, chief operating officer of Ionis Pharmaceuticals, said the data published in the New England Journal of Medicine “clearly demonstrates Waylivra’s safety and efficacy profile for patients with FCS.” Although no timeline was provided, BioSpace was told that Akcea and Ionis are working to confirm a path forward for Waylivra in the U.S. and Canada.

“We are very pleased that The New England Journal of Medicine recognizes the significance of the APPROACH data and the potential of Waylivra to address an area of significant unmet need in the treatment of FCS,” Louis O’Dea, chief medical officer of Akcea Therapeutics said in a statement.

FCS is a rare disorder where patients have extremely high levels of triglycerides in their blood. That high level causes a range of symptoms including potentially fatal attacks of acute pancreatitis. With limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90 percent triglycerides. FCS patients are at risk of chronic complications due to permanent organ damage. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. It is estimated that there are 3,000 to 5,000 people living with FCS worldwide, Akcea noted.

Marcello Arca, head of the Lipid and Atherosclerosis Unit of the University Hospital Policlinico Umberto I and a lead investigator, said the APPROACH study results show that Waylivra lowers triglyceride levels below the threshold for risk of triglyceride-induced acute pancreatitis in the majority of patients. Arca called Waylivra a promising treatment that can “significantly reduce the burden of FCS for patients and their families.”

O’Dea noted that with the approval of Waylivra in Europe, the therapy is the only approved FCS treatment. The company plans to launch the drug in Germany, then expand across the European Union.

Earlier this month, Akcea and Ionis announced positive top-line results for the BROADEN study of Waylivra for the treatment of patients with familial partial lipodystrophy, or FPL. The study met its primary endpoint that showed a statistically significant reduction in triglyceride levels in patients with FPL who were treated with Waylivra compared to placebo. In addition to achieving the primary endpoint, the study achieved a key secondary endpoint of a statistically significant reduction in liver fat, Akcea said.

https://www.biospace.com/article/akcea-and-ionis-eye-a-second-chance-for-fda-approval-of-waylivra/

CMS Releases New Rules Overseeing CAR-T Immuno-Oncology Therapy

Up to this point, Medicare’s regional administrators made the decision on whether the government’s healthcare payer for older populations would cover the innovation CAR-T therapies. However, yesterday, the Centers for Medicare and Medicaid Services (CMS) announced the agency would cover CAR-T so patients would have “consistent and predictable access” to the therapy.

Medicare will pay for CAR-T if they are administered in facilities that follow the U.S. Food and Drug Administration (FDA)’s special safety rules, called risk evaluation and mitigation strategies (REMS). It will also reimburse for the therapy when it’s used to treat illnesses that aren’t approved by the FDA.

There are two CAR-T therapies on the market, Gilead Sciences’ Yescarta and Novartis’ Kymriah. Yescarta is approved for non-Hodgkin lymphoma and Kymriah for acute lymphoblastic leukemia.

When the therapies were approved, the FDA required both companies to set up REMS programs, because of the potential for significant side effects, primarily cytokine release syndrome and neurotoxicities. Those REMS programs required hospitals and clinics that provide CAR-T to be specially certified and have on-site access to treatments to handle the side effects, if necessary.

In a 2018 BioSpace interview with David Maloney, medical director of the Bezos Family Immunotherapy Clinic and director of cellular immunotherapy at Fred Hutchinson Cancer Research Center, Maloney said, “These types of therapies are totally different than any other cancer therapies. They are essentially living cells that actually replicate and grow in the patient as they attack the cancer. It’s exactly the opposite of chemotherapy, which goes in and causes damage, and the damage is slowly resolved over time. CAR-T cells are infused in a relatively small number of cells that actively grow as they engage the target tumor’s cells and kill them. It is a type of living therapy that can expand to eradicate all of the tumor.”

Part of the reluctance on payment is tied to the costs, which run around $375,000 to $475,000, depending on whether the therapies are for patients with advanced lymphoma or pediatric leukemia. And if the patients require hospitalization, which is variable, it can cost hundreds of thousands of dollars more.

The therapies, despite being revolutionary for certain types of cancers, haven’t gained as much traction as expected. This is typically related to confusion over CMS’s rules and reluctance on the part of payers to pay for it.

“Today’s coverage decision provides consistent and predictable patient access nationwide,” stated Seema Verma, CMS Administrator. “CMS will work closely with our sister agencies to monitor outcomes for Medicare patients receiving this in innovative therapy going forward.”

STAT notes that CMS’s decision to write the national coverage determination has been controversial. “The two drug makers already selling CAR-T treatments have argued the national coverage policy is entirely unnecessary, because the contractors that administer Medicare’s hospital claims were already paying for the treatment. Others, including doctors groups, criticized a number of policies in the draft proposal.”

The agency tried to address the concerns in its final rules. It walks back earlier restrictions on CAR-T only being administered in hospitals, which will allow standalone oncology clinics to provide the therapy. Another, perhaps more puzzling change, is CMS gave up on requiring hospitals collect data on how the patients respond to the treatment. Instead, CMS will depend on patient information collected by the FDA and the National Cancer Institute (NCI). The FDA already requires Novartis and Gilead to follow patients for several years and report outcomes. It will be stored in a database supported by NCI.

The Medicare changes don’t guarantee hospitals will break even on CAR-T, however. CMS has struggled to decide on how much to reimburse and Verma told STAT in an earlier interview that it could be years before it is resolved.

Last Friday, CMS indicated it would increase reimbursement for CAR-T, but hospitals indicate those reimbursements are too low and are losing money on Medicare patients.

https://www.biospace.com/article/medicare-to-cover-car-t-immuno-oncology-therapies/

How to Gain Access to Cancer Drugs Not Yet Approved by the FDA

In June 2019, the Food and Drug Administration (FDA) launched the federal government’s latest initiative to make it easier for Americans who have serious illnesses to gain access to experimental drugs.

Dubbed Project Facilitate, it’s a call center that oncologists can use to request help from the FDA in getting these drugs for their cancer patients from the companies developing them. Project Facilitate comes just one year after President Trump signed the “right to try” law, designed to give terminally ill patients the ability to use drugs before they’re approved by the FDA.

Despite these efforts on the part of the government, though, there’s still one big hurdle standing between patients and unapproved drugs: pharmaceutical companies can still decline requests for them. Even the FDA acknowledges that can be a challenge.

“I favor clinical trials more than anything, because that’s how we learn whether drugs will truly be efficacious for patients.”

Project Facilitate is designed to make the process of requesting experimental drugs “as streamlined and efficient as possible,” says FDA press officer Amanda Turney, but the company developing the product “has the right to approve or disapprove the physician’s request.”

Doctor Says: First Try Clinical Trials

That’s just one reason many physicians who treat terminally ill patients recommend they try to get into clinical trials for investigational products before attempting to secure those drugs directly from the manufacturers.

“I favor clinical trials more than anything, because that’s how we learn whether drugs will truly be efficacious for patients,” says Dr. David Hong, director of investigational cancer therapeutics at the MD Anderson Cancer Center in Houston.

When patients try drugs outside of closely monitored trials, “there can be unseen consequences,” Hong says, such as side effects that physicians don’t know how to manage because of a lack of experience with the medications.

Patients with any disease can search for clinical trials online at ClinicalTrials.gov. Several cancer-focused nonprofits, such as BreastCancerTrials.org (in collaboration with the Susan G. Komen Foundation), offer free online tools that match patients to clinical trials based on the specifics of their diagnoses, as does the website EmergingMed.

Hong recommends cancer patients first request help identifying appropriate clinical trials from the oncologist managing their treatment plan. But it’s also important to get a second opinion, ideally from a large cancer center that runs several clinical trials. Why?

“There are thousands of new drugs in development,” Hong says, and there are so many clinical trials starting up all the time that your oncologist may not have the most up-to-date information.

Newly diagnosed cancer patients should also request “next-generation” gene sequencing of their tumors, suggests Dr. Leonard Lichtenfeld, interim chief medical and scientific officer of the American Cancer Society. These tests can identify hundreds of genetic irregularities, such as mutations, that may be targetable with experimental therapies — if not now, then perhaps sometime in the future.

“Say you have a genetic analysis done and there’s no trial available” of a drug targeted at your genetic results, Lichtenfeld says. “Six months later, you may find that a trial exists where there was none before.”

Working With Drug Companies

Another good reason to insist on next-generation gene sequencing is that pharmaceutical companies will often go out of their way to get targeted drugs to patients with rare cancers. That was the case when a small company called Loxo Oncology started developing what is now Vitrakvi (larotrectinib), a drug that targets tumors with a rare abnormality called NTRK gene fusion.

For several years, Loxo (now owned by Eli Lilly) worked with oncologists all over the world to enroll people with the abnormality in the clinical trials — even patients who lived nowhere near the main sites where the drug was being tested. The drug was approved by the FDA last November based on clinical trial data showing that 75% of patients in the trials had a partial or complete response to it.

Not all companies are so accommodating, however, which is why resources like Project Facilitate could be useful.

The program, which is in the pilot phase, includes a call center that will be staffed by oncology experts at the FDA. These experts will help physicians fill out the paperwork that companies require when requests for experimental drugs are made. The FDA staff member assigned to a request will act as a liaison between the physician and drug company.

Whenever a request is denied, the FDA liaison will follow up to find out why the drug maker said no. The program is an extension of Expanded Access, otherwise known as “compassionate use,” which lets patients with life-threatening diseases file requests online for investigational products.

Expanding Access to Drug Trials

It’s important for patients to understand that if they are ultimately able to get an unapproved drug outside of a clinical trial, their health insurer may refuse to pay for it. But insurers will cover the routine costs associated with participating in clinical trials of experimental products, and drug makers, generally, will supply medicines at no charge to patients.

In a perfect world, every patient who wants to try an experimental drug would be able to get into a clinical trial. That’s a long way off, Lichtenfeld says, but initiatives like Project Facilitate could help lessen the barriers.

“Only a small percentage of cancer patients even have the opportunity to participate in clinical trials,” he says. “We need to find patients wherever they are and figure out the best way to get them into the trials. The FDA making these resources available will be helpful.”

How to Gain Access to Cancer Drugs Not Yet Approved by the FDA

Amarin hit on FDA action date extension for Vascepa cardio benefit claim

An FDA advisory committee meeting is tentatively set for Thursday, November 14, to review and discuss Amarin’s (NASDAQ:AMRN) supplemental marketing application seeking a cardiovascular (CV) benefit claim for Vascepa (icosapent ethyl).

The FDA’s current action date of September 28 will most certainly be reset.

Management will host a conference call at 7:00 pm ET to discuss the matter.

Shares down 23% after hours.

https://seekingalpha.com/news/3489920-amarin-23-percent-hours-extension-fda-action-date-vascepa-cv-benefit-claim