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Friday, August 2, 2019

Acorda down 44% after ugly Q2

Acorda Therapeutics (ACOR -43.7%) slumps on more than double normal volume after reporting Q2 results after the close yesterday.
Revenues were down 67% to $50.1M while earnings turned negative.
Inbrija sales were $3M, flat versus Q1 when the commercial launch started (late February). 1,900 patients have received their first prescriptions thus far. The company says it does not plan to provide sales guidance this year.
Ampyra sales were down 71% to $44.2M due to generic competition. Guidance for the year is sales greater than $140M (unch).

FDA clears digital app to help cancer patients self-manage their symptoms

Digital therapeutic developer Voluntis received a marketing authorization from the FDA for what it describes as the first software-as-a-medical-device cleared for use in all different types of cancer—a program previously dumped by Roche’s French pharma division.
The company’s Oleena mobile app and its algorithms are designed to provide users with real-time, adaptive recommendations to help them manage their own symptoms on a day-to-day basis.
Combined with remote patient monitoring and care team support, it aims to help them avoid the distressing yet common events and side effects that may lead to interruptions in their treatment—such as pain, diarrhea and nausea—as well as those that hinder daily function and quality of life. Voluntis also hopes it will help reduce unnecessary emergency room visits and hospitalizations.
The prescription app was branded a Class II medical device by the FDA; in addition to personalized coaching, it also supplies on-demand directions for initiation and dosing of supportive therapies based on a patient’s previously set care plan, according to Voluntis.

“Beyond monitoring their symptoms, we believe that empowering patients via the digital delivery of real-time and personalized therapeutic interventions offers the opportunity for significant clinical and economic outcomes,” CEO Pierre Leurent said in a statement.
Oleena is designed to help guide patients through a range of diagnoses as well as different anticancer treatments such as chemotherapy, immunotherapy and targeted therapies including PARP, PI3K and CDK4/6 inhibitors.

Voluntis previously signed a development deal with Roche Pharma France on a similar product focused on breast cancer, featuring a mobile app that analyzes symptoms and makes personalized care recommendations with the goal of improving adherence.
First launched in October 2015, the collaboration was formally ended earlier this year after Roche decided to not pursue further development of the app, known as Zemy, nor additional plans to extend its use to other solid tumors.

Freezing your T cells today for potential CAR-T therapies tomorrow

A company offering to freeze donors’ T cells and save them until the day they are needed—perhaps even years before the onset of cancer or disease—is getting off the ground with $1 million in funding and partnerships with some of the largest operators in blood sample management and cold chain logistics.
As a kind of T-cell insurance, Cell Vault is pitching a simple concept: Today’s cutting-edge chimeric antigen receptor T-cell (CAR-T) therapies require a starting point of donated T cells, which are genetically modified to better recognize, target and destroy tumor cells before being readministered to the patient.
Treatments like Novartis’ Kymriah and Gilead Sciences and Kite Pharma’s Yescarta have shown promise and been approved in different blood cancers, but these cell therapies are only used after other lines of treatment have failed.
As a result, many otherwise-eligible patients may not be able to provide enough viable T cells to support a CAR-T therapy following multiple regimens of chemotherapy and immune system-depleting treatments or due to the progression of the disease itself.
Cell Vault aims to sequester vials of blood cells and keep them on ice before patients reach that point, using the same processes employed in banking cord blood or freezing eggs for in vitro fertilization. This could be done at the time of diagnosis before beginning treatment or at any other point in a person’s life. The company plans to offer T-cell banking services at different rates and packages, ranging from annual payments to an 80-year plan that costs $100 per year.

According to the startup’s founder, Kevin Kirk, Cell Vault itself will focus mainly on marketing and driving adoption of its idea while the cryopreservation and biobanking work will be handled in partnership with Brooks Life Sciences.
“They handle all of the NIH’s stem cells, and all of Ancestry.com’s kits, for example,” Kirk told FierceMedTech in an interview. “They will produce, distribute and receive the kits, and then process them down to the cellular level.”
Elsewhere, mobile blood draw and sample collections will be completed by ExamOne, a Quest Diagnostics company. Cell Vault is also gathering medical advice from CAR-T specialists at the Medical College of Wisconsin.
The company plans to start in two or three major markets including New York and San Francisco—”longevity is a big deal for a lot of folks in San Francisco,” Kirk said—and it is slated to begin shipping kits in September.
But at the moment, it’s not clear how pharma companies, manufacturers or regulators may respond to the proposal—it’s not exactly known how years of being frozen may affect T cells’ potency as a final CAR-T product. However, Cell Vault does hope to explore whether starting the process with healthier cells could result in a stronger or easier-to-produce therapy down the road.
“CAR-T has only ever been done with T cells from people who have had cancer—it’s never been done with healthy cells before,” Kirk said. “No one has collected them ahead of time, before someone’s health has become compromised.”
In addition, the pursuit of so-called “off-the-shelf,” allogeneic CAR-T therapies may eliminate the need for patients to donate their own T cells in some cases.
“That was probably the biggest red flag or cause for concern that I found, as I was doing my due diligence,” Kirk said, though that technology may still be years away. “In the meantime—for the next five, seven, 10 years, however it plays out—if I can save one person from passing away because they had access to their own cells, I’m happy.”

Could FTC scrutiny of the Roche-Spark merger scuttle future pharma deals?

When Roche delayed its planned $4.8 billion purchase of gene therapy developer Spark Therapeutics for the fifth time earlier this week, citing the Federal Trade Commission’s (FTC’s) review of the deal, one burning question emerged among pharma watchers: What the heck is the FTC’s problem?
Shortly thereafter, another troubling question bubbled up: If the FTC is going to be this nitpicky about one giant pharma company’s effort to buy a relatively small player, could that end up scuttling future deals?
Bloomberg surveyed nine firms that specialize in mergers and acquisitions, and six of them said they’re worried the FTC has changed its approach to reviewing deals. A similar majority said the change—which has yet to be fully understood—could give future dealmakers second thoughts.
“You have to start wondering if this scrutiny will slow down or prohibit these kinds of deals, which would be a huge deal for the sector,” said Brad Loncar, CEO of Loncar Investments, in an interview with Bloomberg.

Roche has not provided any details about what’s holding up its Spark acquisition, but some analysts have speculated it may have something to do with Roche’s hemophilia A drug Hemlibra and two gene therapy products Spark is working on to treat the same disease. Hypothetically, Roche and Spark could combine products in reimbursement discussions with insurers to squeeze out BioMarin’s competing therapy in hemophilia A patients who don’t have factor VIII inhibitors, suggested Jefferies analyst Michael Yee in a note to clients.
The FTC’s monopoly fears have already dampened one big merger this year. In June, Bristol-Myers Squibb said that as part of its $74 billion merger with Celgene, it had agreed to sell off the blockbuster psoriasis drug Otezla. That prompted Jefferies to dash off a note to investors with a stark warning: “We believe this is a (potential) read-through that the FTC is being tougher on regulating competition.”
Now analysts are wondering how the FTC’s newfound scrutiny could affect 2019’s other megadeal, AbbVie’s planned $63 billion purchase of Allergan. AbbVie’s fast-growing IL-23 inhibitor, Skyrizi, is approved to treat psoriasis and in trials for inflammatory bowel disease. That could be a problem, considering Allergan is in late-stage development with its own IL-23 inhibitor, brazikumab, Credit Suisse analyst Vamil Divan pointed out in a recent note to investors.

Then there’s the question of whether the FTC’s tough-on-competition stance could discourage Big Pharma companies from taking out smaller players. A recent survey by RBC Capital Markets analysts turned up several likely acquisition targets, including uniQure, Biohaven and Blueprint Medicines. But uniQure is another gene therapy player that’s working on hemophilia, and that could raise questions among potential acquirers about whether a takeout would pass FTC muster.
Regulatory red tape is just one challenge that could slow the pace of biopharma M&A in the second half of the year. Earlier this week, analysts at Evaluate Vantage reported that the volume of mergers in the sector in the first half of the year plummeted from 83 to 62, and that licensing deals were down, too. The slowdown may have been prompted by a stock market run that has made potential targets unaffordable, they suggested, coupled with an uptick in venture investing in recent years, which has lessened the need for smaller companies to seek out Big Pharma partners.
As for the FTC, it hasn’t shed much light on its process for reviewing the Roche-Spark merger plans. Roche CEO Severin Schwan tried to sooth anxious investors during last week’s second-quarter earnings call, saying he was “very confident” the deal would close by the end of 2019. That may be small comfort, though, considering it was supposed to have been wrapped up by June 30.

Patient Immune Reaction to Checkpoint Inhibitors May Show Better 5-Yr Survival

Researchers with Johns Hopkins Kimmel Cancer Center in Baltimore recently published research that followed 270 cancer patients treated with Bristol-Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab). The patients were treated at 13 different medical centers in the U.S. for advanced melanoma, renal cell carcinoma or non-small-cell lung cancer (NSCLC) who had exhausted all other treatment options before receiving Opdivo. The research reported impressive improvements in five-year survival rates.
The study, which was published in JAMA Oncology, described the secondary analysis from the CA209-003 clinical trial. It found that five-year survival “was negatively associated with presence of bone or liver metastases and positively associated with Eastern Cooperative Oncology Group performance status of 0, objective response, degree of tumor burden reduction, and adverse event occurrence.”

What that means it there were dramatic increases in the proportion of patients that survived five years after receiving Opdivo. Patients with advanced melanoma, before the introduction of Opdivo, had a 5% chance of surviving three years, and those with NSCLC had a 6% chance of surviving five years. The study indicates a 34.2% five-year-survival rate for advanced melanoma patients and 15.6% five-year-survival rate for NSCLC patients after receiving Opdivo.
“The new study provides much-needed data on long-term clinical outcomes associated with nivolumab, a drug that blocks the activity of a molecule called PD-1, removing restraints on cancer-killing cells,” Suzanne Topalian, professor of surgery, associate director of the Bloomberg Kimmel Institute for Cancer Immunotherapy and the lead author of the study, told Forbes.
They also identified useful information about which patients were most likely to benefit from treatment with Opdivo.
All of the patients in the trial had received conventional therapies before entering the trial. However, 40.4% of the patients had been given three or more rounds of previous treatments.
“While all patients had been treated previously, the number of prior treatments did not make a statistical difference in terms of five-year survival in this study,” Topalian told Forbes.
The study found that patients with larger tumor burdens or whose cancer had spread to the bone or liver were less likely to survive five years after Opdivo treatment, nor were those with low lymphocyte counts.

Unexpectedly, the researchers found that patients who had serious side effects of the Opdivo treatment had significantly longer survival. About 10 to 20% of Opdivo patients experience serious side effects such as intestinal and lung inflammation. However, these patients seemed to respond better to the therapy as long as the side effects are managed well enough to not become life-threatening.
Topalian indicates that this may be a measure of immune response.
“Nivolumab activates immune responses,” Topalian told Forbes. “Although we would like those responses to be directed only at cancer cells, in some cases there is a spillover effect and we encounter immune responses against normal tissues. Based on the findings in this study, we can reassure our patients that if they develop these side effects, it may very well put them in a better response and long-term effect category. The real issue is whether we can devise treatments to manage these side effects that will not interfere at all with the anti-tumor immune response.”

RAPT Therapeutics Postpones $86 Million IPO

South San Francisco-based RAPT Therapeutics announced it is postponing its initial public offering (IPO) it filed earlier to raise $86 million via selling 5 million shares at $14 to $16 per share. No reasons were given for the postponement.
In May, the company changed its name from FLX Bio to RAPT Therapeutics to more accurately represent its focus on oral small molecule therapies. The company was founded in 2015 and since its founding has discovered and advanced two drug candidates that target CCR4.

FLX475 is the company’s lead oncology drug. It selectively inhibits the migration of regulatory T cells (Treg) into tumors. In a Phase I clinical trial in 104 healthy volunteers, the therapy was well tolerated and showed favorable drug-like properties and target engagement. It is currently in a Phase I/II trial as a monotherapy and in combination with Merck’s Keytruda (pembrolizumab) in patients with “charged” tumors. The company expects proof-of-concept data in the first half of 2020.
Its other lead product is RPT193, which selectively inhibits the migration of type 2 T helper cells (Th2) into tissues inflamed by allergic reactions. These types of Th2 are drivers of diseases like atopic dermatitis, asthma, chronic urticaria (skin rash), allergic conjunctivitis, rhinosinusitis and eosinophilic esophagitis. In preclinical models, RPT193 reduced inflammation comparable with leading injectable biologics.
On June 18, RAPT completed a $37 million Series C extension. The financing round included funds and accounts advised by T. Rowe Price Associates, and existing investors The Column Group, GV (formerly Google Ventures), Kleiner Perkins, Topspin Partners, and Celgene Corporation.
“We continue to advance our pipeline of oral small molecule therapeutics, with proof-of-concept results expected in the first half of 2020 from our FLX475 program targeting multiple cancers and in mid-2020 from our RPT193 program in atopic dermatitis,” stated Brian Wong, president and chief executive officer of RAPT, at the time. “We appreciate the belief in our vision to use our immunology-based drug discovery and development engine to bring new therapeutics to patients in need of safe and effective treatment options, both in cancer and in inflammatory disease.”
In May, RAPT entered into a research deal to use Personalis’ universal cancer immunogenomics platform, ImmunoID NeXT, to study therapy-related changes in advanced cancer patients taking part in a Phase I/II clinical trial of FLX475.

ImmunoID NeXT consolidates multiple biomarker assays, allowing for the analysis of about 20,000 genes in both DNA and RNA in a single sample. RAPT plans to compare pre- and post-treatment tumor biopsy samples to get a more comprehensive understanding of treatment-related changes in the tumors. RAPT also plans to use the technology platform to evaluate levels of several inflammation-related and immune cell type-related markers in its ongoing Phase II clinical trials of FLX475.
“Using the ImmunoID NeXT Platform for our FLX475 studies will help confirm its mechanism of action and demonstrate that inhibiting the CCR4 receptor with FLX475 blocks the migration of regulatory T-cells (Treg) into tumors,” Wong said at the time. “With this cutting-edge platform, we may be able to show that FLX475, by blocking Treg migration, decreases immune suppression and stimulates an immune response against cancer cells in the tumor microenvironment.”

Glaxo Nucala Receives EU Approval for Self-Administration by Asthma Patients

GlaxoSmithKline (LSE/NYSE: GSK) today announced that the European Commissionhas granted marketing authorisation for two new methods of administering Nucala (mepolizumab): a pre-filled pen and a pre-filled safety syringe. This is the only monthly* anti-IL5 biologic approved in Europe that people with severe eosinophilic asthma can take at home, after a healthcare professional decides it is appropriate.
Dr Hal Barron, Chief Scientific Officer and President, R&D, GSK, said: “Making Nucala available for patients to take in the convenience of their own home is an important advance that builds on its proven efficacy, reflecting our ongoing efforts to meet the needs of patients with complex diseases.”
Severe eosinophilic asthma can have a life-changing impact, with patients experiencing asthma symptoms that remain uncontrolled despite high-dose standard treatments. This can leave them struggling to breathe and at increased risk of a potentially fatal asthma attack./1,/2
The first European launches of the new administration options are expected to take place in August 2019. The original lyophilised version remains available, giving healthcare professionals a choice of three different administration options to best fit in with their patients’ lives.