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The present disclosure relates to pantothenate derivatives for the treatment of neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders.

The present disclosure relates to pantothenate derivatives for the treatment of neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders.

Perlstein Lab, or PLab as it is also known, continues to hone its automated drug discovery platform and presents its latest iteration Wednesday March 23rd at Y Combinator Demo Day. The platform they created helps the bio startup focus on finding cures for rare diseases rather than common ones. This novel approach is different from most Big Pharma labs and is thus one of their differentiators. What is an “automated drug discovery platform?” I asked this to PLab founder and CEO Ethan Perlstein and he summarized for me. Hold onto your braincells, we’re about to get a little technical. First of all, it is important to know that, according to Perlstein, there are about 7000 diseases classified as rare and 95% of these have no FDA approved cure. This is PLab’s area of focus. This is a much different model than Big Pharma which usually focuses on more common illnesses where the research ROI stands to be much greater. Of these 7000 rare diseases, about half are caused by a single broken gene. These are usually inherited diseases and affect children. PLab focuses on single broken gene diseases initially because they are obviously less complex and easier to approach. Using a molecular apparatus known as a CRISPR—which Perlstein likened to a command line editor for animal genomes—PLab alters the genome of test animals (yeasts, flies, worms, fish and sometimes mice) to mimic the broken gene disorder…to essentially, make them ill. Because many of these animal are small, even microscopic, PLab can have large numbers of them to test against. Then, using an automated platform, they apply thousands of chemical compounds to these animals to see which compounds are effective at reversing or bypassing the loss of the broken gene’s function. In this way they can rapidly and effectively test for cures. Using these complex animal types is key to effectiveness according to Perlstein, and differentiates their lab in many ways from Big Pharma, who often focus on cellular tests in petri dishes rather than live organisms. I did have to ask Perlstein what he thought the general public might think about their animal testing. He responded by saying that they prioritize by the least complex animals first—yeasts, worms, flies and fish—an only move up to mice if necessary. However, he indicated that even when testing on mice becomes necessary, they are not doing anything differently from many Pharma companies out there that also test on mice. The second future phase they have planned is to add a predictive learning model to this automated platform that will become faster over time and generate many more insights more quickly and efficiently. So that’s an extremely simplified version of how their technology works. How they plan to enter the market revolves around two main business models which could be summarized as Lead Gen and Accelerator. In one model, they plan to operate as sort of a scout or lead generation partner to larger pharmaceutical companies. For example, when the ROI makes it much harder for a big pharma company to dive into research on a rare disease, PLab could use their intellectual property to be the research partner and to efficiently determine a reasonable disease to target. Then they hand off the results to the larger company to conduct clinical trials. They are currently discussing partnerships now. To bolster this, PLab has calculated a market size of $1 million per person afflicted. A second, more radical model, involves ultra-rare diseases. These are diseases that afflict less than one person in a million. These are diseases that larger pharmaceutical companies definitely don’t focus on.  However, many of the families of the afflicted are already raising funds on their own to contribute to research. In this business model, PLab will work directly with these patient families. Each disease would spin off as a separate research-based startup company focused on that disease. PLab would take a cut of the business they help accelerate. PLab was already quite busy before joining Y Combinator and had already raised a total of $2.5 million from several investors including Retrophin and their infamous ex-CEO Martin Shkreli. However they have severed all ties with Skreli and are not linked with him any longer. PLab has also raised another $1 million convertible note via Slow Ventures. UPDATE: Slow Ventures only invested $500,000 on the $1 million convertible note. The other $500,000 on the note is a mix of angels and existing investors.

The present disclosure relates to pantothenate derivatives for the treatment of neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders.

News Article | December 17, 2015
Site: motherboard.vice.com

Investigators revealed more information Thursday afternoon about the arrest of Martin Shkreli, the millionaire pharmaceutical CEO who drew public rage this year for acquiring an HIV treatment drug and then jacking up the price. Shkreli was arrested earlier Thursday—along with his former lawyer Evan Greebel—and charged with seven counts of fraud for allegedly operating what investigators dubbed a “Ponzi-like scheme” through his hedge fund companies. “These charges in today’s indictment highlight the brazenness and the breadth of Shkreli’s schemes and the outrageous web of lies and deceit weaved by both defendants: Shkreli and Greebel,” said US Attorney Robert Capers at a press conference in Brooklyn. Earlier this year, Shkreli ignited a public debate over the private pharmaceutical industry when his company, Turing Pharmaceuticals, bought Daraprim: a drug used in HIV treatment. Daraprim had long cost $13.50 a pill—not a bargain, but within the realm of affordability. Turing increased the price to $750 a pop, leading people to call 32-year-old Shkreli everything from a “garbage monster” to “the most hated man in America. But long before he started drawing public rage for his drug price hike, Shkreli ran a couple of hedge funds, and it’s his management of these funds—not his pharmaceutical business—that led to his recent charges. With the help of visual aids, Capers and representatives from the FBI and the SEC provided more details to the allegations laid out in the 30-page criminal indictment that was unsealed ahead of Shkreli’s arraignment. Here’s the gist of what the feds are alleging: Shkreli owned a hedge fund called MSMB Capital that he started back in 2009. Investors fed money into that fund (as investors tend to do with hedge funds), but the feds allege Shkreli lost or blew through nearly all of it in the first year. “Mr. Shkreli spent, misappropriated, or lost through poor trading decisions all but $331 of the $660,000 or so of [initial] investor funds raised,” said Andrew Ceresney, the director of enforcement for the SEC. Over the next few years, investigators allege Shkreli raised about $3 million through MSMB Capital, but lost pretty much all of it “He didn’t tell his investor he lost their money,” Capers said. “Instead Shkreli started a new hedge fund called MSMB Healthcare, which he founded in 2011. Here again he lied to the investors in the same way: he concealed from them the prior truth about his performance with MSMB Capital and the fact that he had lost $3 million.” With his new hedge fund, Shkreli raised about $5 million, according to investigators, and kept telling investors in both groups that their money was growing. Shkreli allegedly used the money raised from MSMB Healthcare to pay off debts he had for the poor trades with MSMB Capital, as well as to provide seed money for Retrophin, the pharmaceutical company he started in 2012. Chart provided by the Department of Justice illustrating Shkreli's alleged scheme. Prosecutors allege Shkreli then secretly used profits from Retrophin to pay back the shareholders he owed in his two hedge funds, and that he got Greebel to help him cover it all up from Retrophin’s board of directors. The allegations laid out mean Shkreli could face up to 20 years in prison. The whole alleged scheme is a pretty sticky web—and one that has, of course, not yet been proven in court—and investigators illuminated some of the grey areas at the press conference. But there are still a lot of lingering questions. What happened to all the money Shkreli allegedly lost or spent? Was anyone else involved—like the anonymous individuals dubbed “corrupt employee 1” and “corrupt employee 2” in the indictment? Did lingering debts contribute to his decision to increase the cost of that HIV drug 50-fold? On these important details, investigators were being tight-lipped, partly because they’re still investigating Shkreli. “We’re following the investigation wherever it takes us. We won’t comment on what we’re investigating or what avenues we’re taking,” Capers said, but noted: “It may or may not lead to other arrests.” At Shkreli’s arraignment, the room filled quickly, and spectators and members of the press without a seat were ordered to leave and wait outside the room. Shkreli came in wearing a black T-shirt and jeans. Both Shkreli and Greebel pled not guilty, and agreed to $5 million and $1 million bond amounts, respectively. Both had already surrendered their passports to the FBI. The judge read as a condition of the bond agreement that Shkreli and Greebel were to have no contact with current or former Retrophin employees, but as some of those individuals currently work at Turing Pharmaceuticals, the judge was willing to grant an exception in those cases—but conversations have to be restricted to Turing-related business. Both defendants asked for a time exemption between now and January 20th, their next court date. (Because the right to a speedy trial sets a 70-day maximum period before defendants have to be brought to trial, some defendants opt to delay the start of this 70-day countdown to have more time to mount a defense.) The exemption was granted. Outside the courthouse, a scrum of reporters and photographers waited in the pouring rain for Shkreli to emerge. Once he did, it was a mad rush to get to Shkreli, who did not answer any questions lobbed at him and wore sunglasses.

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