RiluzoleTabs USP 50mg 60ctNEW HAVEN: Biohaven Pharmaceutical Holding Company Ltd. [NYSE:BHVN] announced positive results of its bioequivalence study with BHV-0223, what it calls an “innovative sublingual [can be taken without swallowing] formulation of riluzole.”

The study was designed to demonstrate “pharmacokinetic equivalence” of the company’s drug sublingual BHV-0223 compared to the reference listed drug Rilutek (riluzole), which is currently the standard of care treatment for patients with Amyotrophic Lateral Sclerosis (ALS).

Topline results confirmed that sublingual BHV-0223 (40 mg) achieved bioequivalent exposures relative to Rilutek (50 mg). In the study, 138 healthy volunteers were administered BHV-0223 and Rilutek under fasted conditions.

Opinion and Analysis

By Mitchell Young

Alexion Board Chair
David Brennan
Activist Investor,
Paul Elliott Singer

NEWHAVEN: Things are looking better at Alexion Pharmaceuticals [NASDAQ: Alxn] as the New Year begins. The collapse of the company’s stock from $149 to $105 has abated with a rise back to $125 a share as the year started.

The company announced on January 4th that it has made peace with activist investor Elliot Management. Elliot reportedly made a sizable investment in the company and according to a report in the New York Times wanted to see the company do more to increase the stock price including the possible sale of the company [see Activist Investor Targets Alexion].

Elliot also questioned the level of bioscience expertise on the board, a criticism we raised about the company’s board as well in early November 2017 in Alexion Sent On Market Tailspin Since Relocation, Reorganization Announcements.

Biostage 1st patientHARTFORD: Connecticut Children's Medical Center has made an investment in a small Massachusetts based biotech company Biostage, Inc. [OTCQB: BSTG/BSTGD]. The Center’s investment was part of a $4.2 million private placement. The current company’s market value is approximately $2.5 million. The hospital has reportedly made a $100,000 investment in the placement.

CCMC has several locations in Connecticut and at the Shriners Hospital and Baystate Medical [hospital] in Springfield, Mass.

Biostage says it is developing “bioengineered organ implants to treat cancers and other life-threatening conditions of the esophagus, bronchus and trachea.” According to the company, the Children’s Medical Center was joined by a “group of investors from China.”

In October the company reduced its workforce by 71% [17 employees], in response to problems with another investment that didn’t materialize.

Screen Shot 2018 01 04 at 9.48.54 PMNEW HAVEN: The privately owned biotech Arvinas LLC, based on research by Yale’s Craig Crews, entered a license agreement and research collaboration with Pfizer Inc. [NYSE: PFE] potentially worth more than $830 million. The agreement is for the discovery and development of drug candidates using Arvinas' proprietary PROTAC (PROteolysis TArgeting Chimeras) Platform. The technology developed by Crews is what the company calls, “a novel technology used to create small molecule therapeutics aimed at degrading disease-causing cellular proteins.”

Crews was recognized by Business New Haven in 2014 as the Researcher of the Year, Healthcare Hero. Company Chairman Tim Shannon  led the founding investment for the company, he was recognized as a 2014 Healthcare Hero, Person of Merit as well.

defrancesco melintaNEW HAVEN: Melinta Therapeutics, Inc. [Nasdaq: MLNT], a commercial-stage company discovering, developing, and commercializing novel antibiotics to treat serious bacterial infections, today announced the appointment of Lisa DeFrancesco as senior vice president of investor relations (IR) reporting to Dan Wechsler, president and chief executive officer.

DeFrancesco is a seasoned healthcare industry executive, with 18 years of experience in investor relations, finance and communications. She joins Melinta from Allergan [NYSE: AGN] where she served most recently as vice president of IR.

biotech and life sciencesNEW HAVEN: The US Federal Drug Administration FDA has awarded Achillion Pharmaceuticals, Inc. [Nasdaq:ACHN], orphan drug designation for it development drug ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G).

According to the company, C3G is a “devastating renal disease for which there is no approved therapy.” Adding, “there are estimated to be approximately 4,000 C3G patients in the United States, approximately 4,000 in Europe, and more than 1,000 patients in Japan.

The FDA Orphan Drug Designation program provides incentives for the development of potentially promising drugs to treat, diagnose or prevent orphan diseases and disorders that affect fewer than 200,000 people in the U.S. This designation may provide, under specified conditions, for a seven-year marketing exclusivity period, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.

On November 14, 2017, Achillion announced interim data from the first two patients in an on-going Phase 2 study in C3G patients demonstrated that the current formulation of ACH-4471 achieved “complement alternative pathway inhibition resulting in greater than 50% reduction in proteinuria over the 14-day treatment period and a favorable tolerability profile.”

“We are pleased that the FDA has granted orphan drug designation to ACH-4471 for treatment of C3G, and we look forward to additional data from the ongoing extended release formulation study. We are keenly aware of the unmet need for patients and we are committed to advancing ACH-4471 for C3G as we believe we have an opportunity to develop a potentially disease-modifying therapy, based on the unique mechanism of action, for ACH-4471,” commented Milind Deshpande, Ph.D., President and CEO.

akariBy Mitchell Young

NEW YORK and LONDON: Akari Therapeutics, Plc [NASDAQ:AKTX], a biopharmaceutical company in clinical development of a drug competitive to Alexion Pharmaceuticals [NYSE: ALXN] flagship drug Soliris, announced that the Phase II COBALT trial of CoversinTM in paroxysmal nocturnal hemoglobinuria [PNH], met its primary goals.

Akari is a public company but tiny when compared to Alexion. Akari’s market capitalization is approximately $68 million, Alexion $24 billion.

Clinical trials for drugs competing with Alexion’s Soliris typically have very small patient enrollments. Akari’s trial consisted of a total of only 9 patients. New Haven’ Achillion Pharmaceuticals [ Nasdaq: ACHN] also has a drug in development to treat [PNH], and has met its goals in a Phase II trial as well.

“We are encouraged by the results from the Phase II trial, especially the lower mean LDH value observed in the last three patients enrolled into the trial treated with 45 mg daily compared to the patients treated with 30 mg daily,” commented Dr. David Solomon, Chief Executive Officer of Akari Therapeutics.

Solomon added, “we are on track to progress into Phase III clinical trials in the first quarter of 2018 with the revised dosing regimen of 45 mg, as discussed with the FDA.”

According to the company, “the Phase II data suggest that Coversin is a potential alternative to existing therapy for patients with PNH, and could allow independence from intravenous infusions through self-administration.”

Soliris is administered via infusion, Alexion announced a licensing deal with Halozyme Therapeutics, Inc. [Nasdaq:HALO] of San , technology that will allow Alexions Soliris replacement drug ALXN1210 [ in clinical trials] to also be available to patients by self-administration without infusion, [see licensing].

Akaris expects it Phase III trial will include patients that have not been treated with Soliris as well as patients that have been on the Alexion drug. Alexion’s Soliris generates more than $3 billion annually in sales and at more than $440,000 per patient per year is considered the most expensive drug on the market today.

In reporting last week the New York Times disclosed  that activist investor Elliot Management is seeking changes to Alexion’s board and a revised strategic plan, [see Activist Investor Targets Alexion].