Samumed stuns yet again as anti-aging pipeline draws $438M at $12B valuation

Samumed came out with a bang a couple years ago, when it boldly announced a couple of anti-aging programs and a $12 billion valuation. The San Diego company, which operated in stealth through much of its first decade, had no late-stage programs and no products on the market at the time, causing quite the hubbub when it claimed unicorn status.

Today, that company has added $438 million of equity backing to its coffers, closing a mega-round that adds to its previously raised $212 million. And yet again, the company announced its pre-money valuation for the latest round at a whopping $12 billion. Even in these go-go days of biotech, the dollars are eye-popping.

The figure will certainly raise eyebrows, but that’s nothing new for Osman Kibar, Samumed’s passionate and colorful CEO. I met with Kibar back in 2016, touring the rather quiet halls of Samumed’s headquarters. His enthusiasm for the company’s investigational osteoarthritis drug left an impression on me, as he fervently sketched graphs on a whiteboard to help me understand the WNT pathway.

“We’re sitting on a goldmine, and this is only the tip of the iceberg,” Kibar said at the time. “If our strategy was to sell, no one could afford us.”

Founded in 2008, Samumed now has eight programs in its pipeline: two Phase II drugs, five in Phase I, and one preclinical. Its late-stage programs are in osteoarthritis and androgenetic alopecia (hair loss).

The company’s Phase II trial in OA enrolled 455 patients and went for a year. This patient population includes over 20 million Americans who suffer from cartilage wearing away in their joints and the resulting pain associated with movement. There’s no real treatment besides pain medication, which can exacerbate the problem (feeling less pain, patients tend to move around more and further wear down that cartilage). After receiving a single injection, patients showed cartilage growth at the medial joint. The company’s hair loss drug has shown promising progress, as well.

But in Samumed’s very brief press release on this new round of funding, the company did not mention where the money came from. That caught my attention, because when I first wrote about Samumed, I thought the company’s lead investors were odd choices: IKEA’s private venture firm, anonymous high-net-worth individuals and a single venture capital firm called Vickers Venture Partners. The biggest investors are a furniture company and a venture firm with no history of drug discovery investments. How could they recognize a valuable investment when they saw one?

I reached out to Kibar this morning to see who was in this new syndicate. Kibar replied with this:

Our existing investors participated about ~15-20% of this round, the rest are new. And we went with private capital again (family offices, high net worth individuals, and sovereign funds), no VC/PE (except Vickers).

Finian Tan, chairman at Vickers, at the time explained that Samumed was the first of many investments in drug discovery. Tan himself is no rookie in healthcare. He’s served as executive deputy chairman of Singapore’s National Science and Technology Board, as well as on the boards of The National Cancer Center of Singapore, venture firm Life Sciences Investment, and SingHealth, the biggest health care group in Singapore.

Though not a scientist himself, he apparently has a knack for eyeing good investments. Tan was an early investor of Baidu, known as the “Google of China” and currently valued at $80 billion. Tan said his venture firm made the biggest entry investment it’s ever made with Samumed due to the company’s uniquely high reward vs. risk profile. The last time he made a similar investment was Baidu itself, Tan said.

Samumed’s drug discovery platform could be “a breakthrough of huge proportions,” Tan said in an email. “The impact on humankind would be an order of magnitude we have not seen since (Alexander) Fleming’s discovery of antibiotics (in the 1920s).”

Time will tell.

Kibar said Samumed is targeting its first approval within the next few years.

“This round takes us there (including all our programs, clinical and preclinical pipeline), plus launch cost, plus some extra cushion,” Kibar wrote in an email.

https://bit.ly/2vml7mM

Boehringer Ingelheim lands option on cystic fibrosis gene therapy

Boehringer Ingelheim has secured the option to license a cystic fibrosis gene therapy. The agreement will see Boehringer work with British academics and Oxford BioMedica (OXB) to develop an inhaled, lentiviral vector-based gene therapy treatment for cystic fibrosis.

The U.K. Cystic Fibrosis Gene Therapy Consortium (GTC), one member of the collaboration, laid the groundwork for the deal 17 years ago when it began exploring the viability of treating cystic fibrosis with a gene therapy. Since then, the academic partners from Edinburgh, London and Oxford that make up the GTC have taken a lentiviral vector gene therapy to the cusp of clinical development.

Boehringer’s involvement will help to power the candidate through the rest of the preclinical studies. The GTC will continue to lead development and up the data package needed to move into humans, but the consortium will now benefit from Boehringer’s money and R&D expertise.

The GTC will also receive nonfinancial support from OXB, the gene and cell therapy specialist that makes the lentiviral vector used in Novartis’ CAR-T therapy Kymriah. OXB is contributing its lentiviral-based vector manufacturing knowhow and capabilities to the cystic fibrosis program.

Imperial Innovations, the technology transfer office for Imperial College London, played a leading role in setting up the project, which Boehringer thinks has the mix of capabilities needed to succeed.

“Bringing together our existing expertise as a leader for nearly a century in the discovery and development of therapies that have advanced patient care in respiratory diseases with the gene therapy knowledge of our partners, we aim to unlock unprecedented opportunities for patients with this devastating disease who are desperately waiting for better treatment options,” Clive Wood, Ph.D., senior corporate vice president, discovery research at Boehringer, said in a statement.

Together, the organizations will work to prepare the gene therapy for clinical development. At some point in the R&D process, Boehringer can exercise its option to land the global rights to the asset. Financial terms of the deal have not been disclosed.

https://bit.ly/2KsPbSo

Glenmark, Harbour to Develop Antibody for HER2-Positive Cancers for China

Glenmark Pharmaceuticals S.A. and Harbour Biomed announced today that they have entered into an exclusive license agreement for the Greater China territory to develop, manufacture and commercialize GBR 1302, Glenmark’s bispecific antibody targeting HER2 and CD3 for the treatment of HER2-positive cancers.

“We are very pleased to begin this strategic relationship with Harbour BioMed for the development and commercialization of our bispecific antibody, GBR 1302 in Greater China, where the predominance of certain HER-2 positive cancers presents a significant clinical need,” said Glenn Saldanha, Chairman and Managing Director of Glenmark. “GBR1302 is representative of Glenmark’s commitment to the discovery and development of innovative therapeutics for unmet medical need, and the opportunity to work collaboratively with Harbour BioMed on this program, which brings extensive local experience, is very important to Glenmark.”

Dr. Jingsong Wang, founder and CEO of Harbour BioMed said: “We are looking forward to collaborating with Glenmark Pharmaceuticals to develop and commercialize this promising, novel bispecific antibody in Greater China to meet the significant unmet medical needs of Chinese cancer patients. This collaboration is aligned with our strategy to leverage our clinical development expertise by in-licensing highly innovative clinical stage assets. GBR 1302 is complementary to the internal portfolio we are building through our industry leading transgenic mouse platforms for generating innovative antibody-based therapeutics.”

Under the terms of the agreement, Glenmark will receive an upfront payment and is eligible to receive payments for achieving pre-specified development, regulatory and commercialization milestones, as well as tiered royalties on net sales for any approved products from Harbour BioMed. The agreement is potentially worth more than $120 million in addition to royalties for Glenmark. Harbour BioMed will lead the clinical development and commercialization of GBR 1302, with the option to manufacture GBR 1302 for the Greater China market. The companies will collaborate on the generation of clinical data to support the registration of GBR 1302 in HER2-positive indications in their respective territories.

GBR 1302, Glenmark’s lead immuno-oncology candidate, works by stimulating the patient’s immune system against HER2 overexpressing tumor cells. GBR 1302 is currently in a first-in-human study to determine maximum tolerated dose (MTD) in an all-comers population of patients with a variety of HER2-positive cancers. Enrollment for the GBR 1302 clinical study is currently ongoing in the U.S. and Germany.

Kurt Stoeckli, President and Chief Scientific Officer for Glenmark added, “Harbour BioMed represents a company that is dedicated to the same principles as Glenmark in pursuing highly effective, precision-medicine based immunotherapeutics for the benefit of cancer patients and we look forward to working closely with them to advance meaningful treatment options.”

https://bit.ly/2nh9wkt

Elite Pharmaceuticals Receives FDA Approval for Generic Methadone

 Elite Pharmaceuticals, Inc. (“Elite” or the “Company”) (OTCBB: ELTP), a specialty pharmaceutical company developing abuse-deterrent opioids and niche generic products, today announced that it received approval from the U.S. Food and Drug Administration (FDA) for the Company’s abbreviated new drug application (ANDA) for methadone hydrochloride 5 mg and 10 mg tablets.  Methadone is indicated for the management of pain severe enough to require daily, around-the-clock long-term opioid treatment and for which alternative treatment options are inadequate.  Methadone can also be used for maintenance treatment of opioid addiction (heroin or other morphine-like drugs) in conjunction with appropriate social and medical services.

Glenmark Pharmaceuticals, Inc., Elite’s marketing alliance partner, will sell and distribute methadone for Elite for which Elite will receive manufacturing and license fees. Based on QuintilesIMS Health data, the annual retail sales for the brand and generic products were approximately $30 million.

“I am pleased to receive FDA approval for our methadone ANDA filing.  We expect methadone to be a key product for our marketing alliance with Glenmark Pharmaceuticals,” stated Nasrat Hakim, President and CEO of Elite. “This is our second product approval in the last 30 days. We have four additional ANDAs and an NDA for SequestOx™ currently filed with the FDA.”

https://bit.ly/2OiNKIB

Alzheon IDs Brain Molecule that Inhibits Formation of Alzheimer’s Proteins

_{3-D Rendering of beta-amyloid peptide, a hallmark pathology in Alzheimer’s disease}

Scientists with Framingham, Massachusetts-based Alzheon identified a substance in the human brain that inhibits the formation of beta-amyloid (Aβ), the primary driver of Alzheimer’s disease. The substance is 3-sulfopropanoic acid (3-SPA).

The company indicates that tramiprosate and its prodrug ALZ-801 are consistently metabolized in humans to a single major metabolite, 3-SPA. 3-SPA inhibits the formation of beta-amyloid oligomers, comparable to the effects of tramiprosate.

In their research, the company found that levels of 3-SPA were up to 12 times greater in Alzheimer’s patients who received oral tramiprosate than in drug-naïve patients or patients being treated with placebo. The data adds to what is already known about the mechanism of the company’s ALZ-801, which is a Phase III-ready drug candidate.

It also hints at the possibility of a protective endogenous anti-beta-amyloid oligomer pathway, which it’s dubbing an “Aβ oligomer brake pathway” within the human central nervous system. And, potentially, this pathway could prevent or delay the onset of Alzheimer’s disease, or offset or modulate the neurotoxic effects of abnormal beta-amyloid accumulation in the aging human brain. Which sounds like an awful lot of “ifs” and “maybes,” but at least is more information in support of the beta-amyloid theory of Alzheimer’s disease, which has come under a shadow from many failed Alzheimer’s drugs.

The beta-amyloid theory is based on the belief that the primary driver of Alzheimer’s disease is the accumulation of beta-amyloid plaques in the brain, which results in the memory loss and cognitive deficits seen in Alzheimer’s patients. However, there have been a number of high-level Phase III drug trial failures in antibodies that do prevent or clear beta-amyloid successfully, but do not result in improved cognition. Some believe this means the beta-amyloid theory is wrong, while most believe it means the brain damage has already been done and can’t be reversed, so clinical trials of drugs that prevent or clear beta-amyloid are taking place earlier and earlier in the disease.

“We are excited to contribute to a better understanding of the pathogenic and therapeutic mechanisms in Alzheimer’s disease,” said Martin Tolar, Alzheon’s founder, president and chief executive officer, in a statement. “The results from this publication suggest a potential protective role of endogenous 3-SPA in normal human brains, guarding against the formation of beta-amyloid oligomers that cause neurodegenerative disorders such as Alzheimer’s.”

Tolar goes on to say, “In addition, our results suggest a potential contribution of 3-SPA to the clinical efficacy of ALZ-801 and connect it more closely to the protective effects against neurotoxic amyloid oligomers. While targeting soluble amyloid aggregates is the only therapeutic approach to date that has shown a disease-modifying effect in Alzheimer’s patients, no drugs have been approved yet that can slow or stop the disease. This new discovery and mechanistic data strongly support our therapeutic approach and strengthen Alzheon’s commitment to confirm the efficacy of ALZ-801 in APOE4 carriers, a genetically-defined subset of Alzheimer’s patients.”

The research was published in the journal CNS Drug and is titled, “Discovery and Identification of An Endogenous Metabolite of Tramiprosate and its Prodrug ALZ-801 that Inhibits Beta Amyloid Oligomer Formation in Human Brain.”

https://bit.ly/2Kw2fGo

Pain Therapeutics CEO Lashes Out as FDA Rejects Company’s Remoxy

The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter—basically a rejection—to Austin, Texas-based Pain Therapeutics for Remoxy ER for pain.

Remoxy ER (oxycodone) Extended-Release Capsules CII, based on Pain Therapeutics’ ORADUR technology, is a long-acting formulation of oxycodone designed to discourage most methods of tampering linked to opioid abuse. On June 26, a joint meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee to the FDA voted 14 to 3 against approving the drug.

The FDA argued that the benefits of the drug did not outweigh the risks.

“This is a bizarre conclusion to reach, especially during a time of staggering human and economic toll created by opioid abuse and addiction,” said Remi Barbier, Pain Therapeutics’ president and chief executive officer, in a statement. “We have an innovative drug with a social purpose, and a staggering amount of data that easily supports best-in-class abuse deterrence versus OxyContin. We relied on the criteria of a fair, neutral and impartial regulatory review, as any sponsor would. Instead, I believe Remoxy received an ideological judgment call that is vague in nature but conclusive in its damaging effects.”

Seemingly in response to the rejection, Pain Therapeutics has launched a strategic reorganization. It plans to shift its focus from tamper-resistant opioid formulations to Alzheimer’s disease. Details are expected to be released in the upcoming weeks.

The company says it “believes its program in Alzheimer’s disease has game-changing characteristics. PTI-125, the lead drug candidate, is a small molecule that has a unique mechanism of action for treating Alzheimer’s disease.”

The drug wrapped a successful Phase I clinical trial, which was funded by a peer-reviewed research grant from the National Institute of Health (NIH). It also has another asset, a blood test for Alzheimer’s called PTI-125DX, which the company claims can detect or confirm whether an individual has Alzheimer’s, even years before symptoms show. It is also substantially funded by the NIH.

“Alzheimer’s disease is a therapeutic indication with a profound need for new treatments,” Barbier said in a statement. “A reorganization of the company represents a natural and timely evolution of the strength of our program in Alzheimer’s disease.”

If their diagnostic test is effective, and there are even a few details about what biomarkers the assay tests for, it could be a very important product. Otherwise, a positive Phase I product for Alzheimer’s disease is a longshot. Literally hundreds of drugs for Alzheimer’s that were positive in Phase I have failed in Phase II and III. Still, it’s what the company has to offer. There is also no indication if or how many jobs will be cut in the restructuring.

Company stocks dropped 6.6 percent in premarket trading at the news. Overall, shares have dropped 39.8 percent this year, compared to the S&P 500 SPX, which has gained 6.2 percent.

As of June 30, at the company’s second-quarter financial report, Pain Therapeutics has cash and cash equivalents of $9.6 million. At that time, the company indicated it had enough cash to continue operations for the next 12 months.

https://bit.ly/2M39b2F

FDA Action Alert: DURECT, Vertex, Arbutus & Alnylam, and Regeneron

Now that July is over, the U.S. Food and Drug Administration (FDA) appears to be back in full swing with an ample docket of drug decisions to make. There are four therapeutics with PDUFA dates this week involving six biopharma companies. Here’s a look.

Cupertino, California-based DURECT Corporation and Austin, Texas-based Pain Therapeutics are waiting for an approval from the FDA by August 7 for Remoxy ER for pain. Remoxy ER (oxycodone) Extended-Release Capsules CII, based on the company’s ORADUR technology, is a long-acting formulation of oxycodone designed to discourage most methods of tampering linked to opioid abuse. On July 30, the FDA approved its New Drug Application (NDA) for Perseris (risperidone) to treat schizophrenia.

The likelihood of approval for Remoxy is slim, however. On June 26, a joint meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee to the FDA voted 14 to 3 against approving the drug.

Vertex Corporation has a PDUFA date of August 7 as well, for its NDA for lumacaftor/ivacaftor combination therapy for cystic fibrosis in children ages 2 to 5 years of age. The company also has a supplemental NDA (sNDA) for ivacaftor in children ages 12 to less than 24 months with a PDUFA action date of August 15. On July 30, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended approval of the combination therapy for patients with two copies of the F508del mutation, or one copy of this CFTR gene variant plus one of 14 residual mutations.

“Our goal at Vertex is to find a cure for all people living with CF and we are moving rapidly towards treating up to 90 percent of patients,” said Reshma Kewalramani, executive vice president of global medicines development and medical affairs and chief medical officer at Vertex, in a statement. “Today’s announcement is a pivotal accomplishment along that journey.”

Arbutus Biopharma and Alnylam Pharmaceuticals, headquartered in Warminster, Pennsylvania and Cambridge, Massachusetts, respectively, have a PDUFA date of Saturday, August 11 for their patisiran. Patisiran is an investigational RNA interference therapy that inhibits hepatic synthesis of transthyretin. In its Phase III clinical trial in patients with hereditary transthyretin amyloidosis with polyneuropathy, the drug improved several clinical markers in the disease. Approval will also trigger a royalty entitlement “to Arbutus for the proprietary LNP technology licensed by Arbutus to Alnylam for patisiran.” The FDA had granted the drug Fast Track Designation, Breakthrough Therapy Designation, and an expanded Orphan Drug Designation for ATTR amyloidosis. The drug is also being evaluated by the European Medicines Agency (EMA).

Tarrytown, New York-based Regeneron Pharmaceuticals is awaiting a supplemental Biologics License Application (sBLA) target date for its eye drug, Eylea, of Saturday, August 11. The decision is regarding a 12-week dosing interval in wet age-related macular degeneration (AMD). Eylea is approved for wet AMD, diabetic macular edema (DME) and diabetic retinopathy (DR) in DME, and macular edema following retinal vein occlusion (RVO).

The sBLA was based on analysis of two-year data from VIEW 1 and VIEW 2 Phase III clinical trials that evaluated Eylea in wet AMD. The data found that 51 percent of patients had their Eylea dosing interval extended to every 12 weeks at the beginning of the second year of treatment and were able to maintain this every 12-week dosing interval and their best-corrected visual acuity (BCVA) improvements when they were evaluated at week 96.

https://bit.ly/2vJteJd