Using cytokines therapeutically is like firing a shotgun at range – for sure you will hit your target, but off-target effects are likely, if not certain. To hit your target exclusively you have to get close. Delivering a proprietary version of the chronic inflammatory cytokine IL-10 encoded on a proprietary plasmid, injected locally, gets you really close. Using their non-viral, non-integrating vector, Xalud Therapeutics is exploring the use of IL-10 for osteoarthritis (FDA fast tracked), and ot...

An inch wide and a mile deep—that’s the sort of expertise CSO of Artios Pharma, Dr. Graeme Smith, brings his benchtop. Since his time at Cambridge, Smith has been laser focused on DDR mechanisms, first elucidating, and then translating those observations to create the PARP inhibitor drug, Lynparza. Developed at Kudos Pharma, and later, AstraZeneca, hear Smith recall his “aha” moment for this first-in-class blockbuster drug, as well as his descriptions of his two new DDR-targeting, clinical stage...

Hypoxia is the key. TEC-001 is a prodrug selectively activated in hypoxic environments, such as that induced by the long-known technique called trans-arterial embolization, a procedure that shuts off blood supply to tumors and thereby creates a low-oxygen, hypoxic environment. Delivered directly to the tumor bed through a tumor-feeding artery, hypoxia-activated TEC-001 then converts into free radicals that cause so-called immunogenic cell death (necrosis, as opposed to apoptosis) which enables i...

A car is a very efficient delivery system until you can’t find a place to park, or the spot you find is nowhere near where you wanted to go. For medicine, getting to the right place, and getting there in time is critical. With the iSPERSE platform you can reformulate known drug entities—compounds that have already been de-risked—for fast-acting pulmonary distribution using most any inhaler device. Listen to the POC work already in hand for migraine, and COPD as described by Pulmatrix CEO Ted Raa...

MDM2 is a key regulator of p53, the protein responsible for tumor suppression and apoptosis — an intractable, yet undruggable target. Previous attempts to drug MDM2 have been met with significant toxicities issues. The MDM2 oral inhibitor milademetan has the potential to address these issues and in doing so may open the door to treating multiple tumor types, as identified by standard molecular testing. Indeed, studies with milademetan have demonstrated promising preliminary results in liposarcom...

MDM2 is a key regulator of the holy grail of oncology, the tumor suppressor called p53—an intractable, as yet undruggable target. But you can drug MDM2, and a number of companies have, only to walk away due to unacceptable toxicity. The MDM2 inhibitor milademetan has solved that problem, and in doing so opened the door to treating multiple tumor types, as identified by standard molecular testing. Indeed, cancers as different as liposarcoma, and Merkle Cell are already responding to this drug. Le...

The only people that like infusion suites are those who own them. Payers don’t like them. Patients hate them. But for many drugs, due to their molecular nature there is simply no choice, and this has been the case with the class of drugs known as checkpoint inhibitors—until now. Now there is Envafolimab, a novel anti-PD-L1 antibody from Tracon Pharma with a low volume, subcutaneous administration that is already being tested in the clinic in multiple indications. Listen in as CEO Charles Theuer ...

By way of an exquisite toggling of activity, HADUVIO stimulates one opioid receptor (kappa) and blocks the activity of another (mu) while avoiding the engagement of the third (delta). The objective of this combination of receptor activity is to decrease chronic cough due to Idiopathic Pulmonary Fibrosis, or severe itch due to Prurigo Nodularis. Phase II data are available for both development programs, which CEO Jennifer Good has proudly shared—along with a story of how Trevi got its name…

If the pandemic taught us nothings else, we’ve learned the critical importance of having a dependable system for delivery. OncoNano offers two such systems (read: platforms). The first, ON-BOARD, is a polymeric micelle that delivers its therapeutic payload only in the acidic environment of a tumor bed. The second, OMNI, consists of a polymer that in itself stimulates the immune-activating STING pathway, and which can further be loaded with other STING agonists, or, potentially, other mechanistic...

Along with the first-ever approval by the FDA of a so-called checkpoint inhibitor, the anti-CTLA-4 drug Yervoy, in 2001, came vindication; the treatment modality of immuno-oncology (IO) had arrived. Anti-PD-1 drugs soon followed with even greater—far greater impact. But then came some failures, and the presumed bright future of other IO assets began to dim. Until now. The first LAG-3-targeting drug was recently approved, and as of ASCO 2022, Immutep’s LAG-3 asset is poised to replace IO/chemo in...

There’s no point in receiving a gift you can’t keep, be it a pony, or a therapeutic. For the latter to be effective it must be retained, yet the immune system can’t discriminate between friend or foe; there is only you, and not you, and not you is simply not tolerated. Enter ImmTOR®, a nanoparticle technology that aims to tolerize the immune system to the target of choice – a gene therapy, a biologic, or in cases of autoimmunity – you. With buy-ins from the likes of Takeda, Sarepta, Sobi and Ask...

There’s nothing new about transdermal delivery of drugs—patches, gadgets, and solvents abound—but each has physical limitations, and the drugs themselves may not be amenable to a particular approach. Enter a novel drug formulation platform by Dyve Biosciences. Delivered in what appears to be a cosmetic cream, Dyve has two POC clinical trials underway: Gout, to test local delivery of a drug; and cancer, testing the same drug that can only be delivered through the skin, and targeting the tumor mic...

In pharmacology, getting your drug to where it’s needed is everything. In immunology, to elicit an immune response that location is often the lymph node, and the point of delivery are antigen presenting cells. Amphiphile, from Elicio, can make that happen. Whether the cargo is DNA, a peptide, or a small molecule, once linked to the Amphiphile moiety, the construct is taken up by the lymphatic system. First asset in the clinic? A multiple antigen therapeutic for mKRAS.

Innovation can sometimes be counterintuitive, as in the case with Tyrvaya, a drug approved in 2021 for the signs and symptoms of dry eye disease that is a nasal spray. And how does that make sense? Well, it makes sense because Tyrvaya ®, a cholinergic agonist to treat the underlying, and sometimes debilitating disease. Hear Dr. Jeffrey Nau, ophthalmology expert and CEO of Oyster Point explain…

The allure of allogeneic “off-the-shelf” adaptive cell therapy (ACT) is obvious – by definition, the treatment is immediately available, and due to the nature of batch manufacture versus the one-off creations of an autologous ACT, it will very likely be cheaper. The question remaining is what cell type is best to use for clinical efficacy – T cells of the adaptive immune system, or natural killer (NK) cells of the innate system, OR, just maybe, a cell that does both – the CAR NKT cell therapy, f...

Having focused on endocrine disorders since grad school, first at the bench and later in the board room, Dr. Thierry Abribat founded and became the CEO of EU-based Amolyt Pharma because he wanted to have a “social impact” – he wanted to make a difference. With two clinical stage, biologic assets in hand (and an orphan drug designation) Abribat is hoping to address the serious unmet needs of patients with hypoparathyroidism and acromegaly.

Everyone is excited about KRAS, the “undruggable” target that now has a drug. However, that drug only hits one specific, rather rare KRAS mutation, leaving the vast majority of patients with KRAS mutant variants who, after exhausting standard of care (SOC), are left without any effective treatment options. So, what if you had a drug that hit just downstream in the KRAS pathway, and further, was synergistic with SOC – wouldn’t that serve all such patients? Yes. Yes, it would. That drug is called ...

Hear how Jay Mai, MD, PhD, went from his benchtop years at the NIH to a blockbuster launch at Celgene (think, Revlimid, after stints at Novartis and J&J along the way) – to spearhead the multi-asset juggernaut that is the oncology company, Antengene. From it’s “license and build” beginnings, to the advent of in-house programs (15 assets in all) Antengene is poised to hit multiple oncology targets, from a small molecule ERK 1/2 inhibitor, to a bispecific for PD-L1/ 4-1BB. And did I mention they h...

Proteins designed as drugs – the so-called biologics – sparked a medical revolution, but it has come at an extraordinary cost: less than 2% of Americans use these drugs, yet they account for 40% of total spend on prescription medications. Something’s got to give. Mark Emalfarb of Dyadic thinks that something is manufacturing, and his something is C1 – a protein manufacturing platform, and cell line that can produce biologics cheaper and faster than anything out there. Unconvinced? Give a listen.

Having the ability to activate a precise, robust and persistent immune response to kill cancer cells is what IMV is all about. Using its DPX delivery technology, IMV’s immunotherapies are easy and cost-effective to manufacture. With promising clinical data now available, their lead compound is well tolerated and effective in several cancer indications including solid cancers (ovarian and bladder cancers) and DLBCL (lymphoma). What else is cooking at IMV? Former professional chef, and IMV CEO, An...

There are approximately 30 million individuals in the U.S. currently living with one of at least 7,000 rare diseases. The National Organization for Rare Disorders estimates that more than 90% of these patients are still without an FDA-approved treatment. Dr. Jane Larkindale, VP of Clinical Science at PepGen, Inc. is looking to change that, and she has both the experience and passion to show you how.

A scar is proof you survived – a good thing. Uncontrolled scarring, called fibrosis, is a threat unto itself (think cirrhosis) or an impediment to clinical intervention (think macrophages, T-cells and cancer). Mechanisms that drive both involve the molecule, Gal-3, and an enzyme, LOXL2. The mission of Galecto is drugging these targets with small molecule, first-in-class inhibitors. Hear how Galecto’s CEO, Dr. Hans Schambye, made their mission his own.

In oncology, the field of neoantigen prediction is rife with proprietary mathematics for homing in on the best antigens for T cell activation. Only one neoantigen platform, ATLAS, from Genocea, tells you not only about what activates, but also what antigens/epitopes suppress that T cell activity – so called, “inhibigens”. With his platform recently validated in Cancer Discovery, Chip Clark, CEO of Genocea, explains the investment opportunity that is ATLAS.

Every cancer neoantigen prediction platform has lots and lots of math – the PIONEER platform, from the Danish company, Evaxion, has data. In a phase I/IIa study in metastatic melanoma the ORR was 67%, and two patients with stable disease converted to complete responders. Investors take note, because Merck sure did… Dr. Lars Wegner, CEO of Evaxion fills in the details.